Pediatrics

Apremilast (Otezla), an oral drug approved for adult psoriasis, appears to reduce psoriasis severity in some children with moderate to severe psoriasis not controlled by topical therapy, according to the results of a phase 3 trial.

“Unfortunately, there are limited treatment options for pediatric patients with moderate to severe plaque psoriasis” who do not respond to or cannot use topical therapy, said study investigator Anna Belloni Fortina, MD, speaking at the annual meeting of the European Academy of Dermatology and Venereology.

“In this randomized, placebo-controlled trial, oral apremilast demonstrated effectiveness and was well tolerated,” added Dr. Belloni Fortina, of Azienda Ospedale Università Padova (Italy). “I underline oral because for children, oral administration is better than the injection treatment.”
 

Key findings

Dubbed the SPROUT study, the trial set a primary endpoint of the percentage of children with a Physician’s Global Assessment (sPGA) response after 16 weeks of treatment or placebo. The sPGA is a 5-point scale ranging from 0 (clear) to 4 (severe). The study enrolled children with an sPGA greater than or equal to 3. Response was defined as a sPGA score of 0 or 1, indicating clear or almost clear skin, with at least a 2-point reduction from baseline values.

At week 16, the primary endpoint was met by 33% of 163 children treated with apremilast versus 11% of 82 children who had been given a placebo, a treatment difference of 21.7% (95% confidence interval, 11.2%-32.1%).

A greater proportion of children treated with apremilast also achieved a major secondary endpoint, a 75% or greater reduction in the Psoriasis Area and Severity Index (PASI-75) (45.4% vs. 16.1%), a treatment difference of 29.4% (95% CI, 17.8%-40.9%).
 

Results unaffected by weight and age

Regarding apremilast, “it’s important to underline that patients were dosed according to their weight,” Dr. Belloni Fortina said.

A dose of 20 mg twice daily was given to children who weighed between 20 kg and less than 50 kg, and a 30-mg twice-daily dose was given to those who weighed greater than or equal to 50 kg.

When the data were analyzed according to weight, proportionately more children on apremilast saw a sPGA response: 47.4% versus 21.8% in the lower weight and dose range and 19.2% versus 1.6% in the higher weight and dose range.

As for PASI-75, a greater proportion of children on apremilast also responded in both the lower and upper weight ranges, a respective 52.4% and 38.7% of patients, compared with 21.4% and 11% of those treated with placebo.

Data were also evaluated according to age, with a younger (aged 6-11 years) and older (age 12-17 years) group. The mean age of children was 12 years overall. Results showed a similar pattern for weight: The psoriasis of more children treated with apremilast was reduced by both measures, sPGA response, and PASI-75.
 

Safety of apremilast in children

“The overall safety profile during the placebo-controlled phase was comparable with the known safety profile of apremilast,” Dr. Belloni Fontina reported. “No new safety signals were identified.”

The rate of any adverse event was substantially higher in children given the active treatment, however, at 65% versus 41.3% for placebo.

Rates of severe and serious adverse events were low, at around 1.3%, and similar between the groups.

There was also a low rate of withdrawal because of side effects, although this was higher in the apremilast group (3.1% vs. 1.3%).

The primary reason for withdrawal of apremilast treatment were the most commonly reported adverse events: gastrointestinal disorders, including diarrhea, nausea, upper and lower abdominal pain, and vomiting. Headache, pyrexia, and nasopharyngitis were also reported.

Despite being common, most treatment-related adverse effects resolved within 3 days, Dr. Belloni Fontina said.
 

Expect further data

Further data from the trial are to be expected, because only the 16-week primary endpoint results have been released so far. The trial also included a 36-week extension phase, during which all children who had originally been randomly assigned to placebo were now eligible to be treated with apremilast, and all those who were originally given the active treatment were able to continue. This extension treatment period means that data will be available for a full year of treatment, and there will also be a further 2-week observational follow-up at the end of the trial.

The study was funded by Amgen. Dr. Belloni Fontina reported acting as an investigator and advisory board member for and receiving honoraria from Amgen, Galderma, Leo Pharma, and Pfizer. She also reported speaking on behalf of Pierre-Fabre and Galderma.

A version of this article first appeared on Medscape.com.

Apremilast (Otezla), an oral drug approved for adult psoriasis, appears to reduce psoriasis severity in some children with moderate to severe psoriasis not controlled by topical therapy, according to the results of a phase 3 trial.

“Unfortunately, there are limited treatment options for pediatric patients with moderate to severe plaque psoriasis” who do not respond to or cannot use topical therapy, said study investigator Anna Belloni Fortina, MD, speaking at the annual meeting of the European Academy of Dermatology and Venereology.

“In this randomized, placebo-controlled trial, oral apremilast demonstrated effectiveness and was well tolerated,” added Dr. Belloni Fortina, of Azienda Ospedale Università Padova (Italy). “I underline oral because for children, oral administration is better than the injection treatment.”
 

Key findings

Dubbed the SPROUT study, the trial set a primary endpoint of the percentage of children with a Physician’s Global Assessment (sPGA) response after 16 weeks of treatment or placebo. The sPGA is a 5-point scale ranging from 0 (clear) to 4 (severe). The study enrolled children with an sPGA greater than or equal to 3. Response was defined as a sPGA score of 0 or 1, indicating clear or almost clear skin, with at least a 2-point reduction from baseline values.

At week 16, the primary endpoint was met by 33% of 163 children treated with apremilast versus 11% of 82 children who had been given a placebo, a treatment difference of 21.7% (95% confidence interval, 11.2%-32.1%).

A greater proportion of children treated with apremilast also achieved a major secondary endpoint, a 75% or greater reduction in the Psoriasis Area and Severity Index (PASI-75) (45.4% vs. 16.1%), a treatment difference of 29.4% (95% CI, 17.8%-40.9%).
 

Results unaffected by weight and age

Regarding apremilast, “it’s important to underline that patients were dosed according to their weight,” Dr. Belloni Fortina said.

A dose of 20 mg twice daily was given to children who weighed between 20 kg and less than 50 kg, and a 30-mg twice-daily dose was given to those who weighed greater than or equal to 50 kg.

When the data were analyzed according to weight, proportionately more children on apremilast saw a sPGA response: 47.4% versus 21.8% in the lower weight and dose range and 19.2% versus 1.6% in the higher weight and dose range.

As for PASI-75, a greater proportion of children on apremilast also responded in both the lower and upper weight ranges, a respective 52.4% and 38.7% of patients, compared with 21.4% and 11% of those treated with placebo.

Data were also evaluated according to age, with a younger (aged 6-11 years) and older (age 12-17 years) group. The mean age of children was 12 years overall. Results showed a similar pattern for weight: The psoriasis of more children treated with apremilast was reduced by both measures, sPGA response, and PASI-75.
 

Safety of apremilast in children

“The overall safety profile during the placebo-controlled phase was comparable with the known safety profile of apremilast,” Dr. Belloni Fontina reported. “No new safety signals were identified.”

The rate of any adverse event was substantially higher in children given the active treatment, however, at 65% versus 41.3% for placebo.

Rates of severe and serious adverse events were low, at around 1.3%, and similar between the groups.

There was also a low rate of withdrawal because of side effects, although this was higher in the apremilast group (3.1% vs. 1.3%).

The primary reason for withdrawal of apremilast treatment were the most commonly reported adverse events: gastrointestinal disorders, including diarrhea, nausea, upper and lower abdominal pain, and vomiting. Headache, pyrexia, and nasopharyngitis were also reported.

Despite being common, most treatment-related adverse effects resolved within 3 days, Dr. Belloni Fontina said.
 

Expect further data

Further data from the trial are to be expected, because only the 16-week primary endpoint results have been released so far. The trial also included a 36-week extension phase, during which all children who had originally been randomly assigned to placebo were now eligible to be treated with apremilast, and all those who were originally given the active treatment were able to continue. This extension treatment period means that data will be available for a full year of treatment, and there will also be a further 2-week observational follow-up at the end of the trial.

The study was funded by Amgen. Dr. Belloni Fontina reported acting as an investigator and advisory board member for and receiving honoraria from Amgen, Galderma, Leo Pharma, and Pfizer. She also reported speaking on behalf of Pierre-Fabre and Galderma.

A version of this article first appeared on Medscape.com.

Apremilast (Otezla), an oral drug approved for adult psoriasis, appears to reduce psoriasis severity in some children with moderate to severe psoriasis not controlled by topical therapy, according to the results of a phase 3 trial.

“Unfortunately, there are limited treatment options for pediatric patients with moderate to severe plaque psoriasis” who do not respond to or cannot use topical therapy, said study investigator Anna Belloni Fortina, MD, speaking at the annual meeting of the European Academy of Dermatology and Venereology.

“In this randomized, placebo-controlled trial, oral apremilast demonstrated effectiveness and was well tolerated,” added Dr. Belloni Fortina, of Azienda Ospedale Università Padova (Italy). “I underline oral because for children, oral administration is better than the injection treatment.”
 

Key findings

Dubbed the SPROUT study, the trial set a primary endpoint of the percentage of children with a Physician’s Global Assessment (sPGA) response after 16 weeks of treatment or placebo. The sPGA is a 5-point scale ranging from 0 (clear) to 4 (severe). The study enrolled children with an sPGA greater than or equal to 3. Response was defined as a sPGA score of 0 or 1, indicating clear or almost clear skin, with at least a 2-point reduction from baseline values.

At week 16, the primary endpoint was met by 33% of 163 children treated with apremilast versus 11% of 82 children who had been given a placebo, a treatment difference of 21.7% (95% confidence interval, 11.2%-32.1%).

A greater proportion of children treated with apremilast also achieved a major secondary endpoint, a 75% or greater reduction in the Psoriasis Area and Severity Index (PASI-75) (45.4% vs. 16.1%), a treatment difference of 29.4% (95% CI, 17.8%-40.9%).
 

Results unaffected by weight and age

Regarding apremilast, “it’s important to underline that patients were dosed according to their weight,” Dr. Belloni Fortina said.

A dose of 20 mg twice daily was given to children who weighed between 20 kg and less than 50 kg, and a 30-mg twice-daily dose was given to those who weighed greater than or equal to 50 kg.

When the data were analyzed according to weight, proportionately more children on apremilast saw a sPGA response: 47.4% versus 21.8% in the lower weight and dose range and 19.2% versus 1.6% in the higher weight and dose range.

As for PASI-75, a greater proportion of children on apremilast also responded in both the lower and upper weight ranges, a respective 52.4% and 38.7% of patients, compared with 21.4% and 11% of those treated with placebo.

Data were also evaluated according to age, with a younger (aged 6-11 years) and older (age 12-17 years) group. The mean age of children was 12 years overall. Results showed a similar pattern for weight: The psoriasis of more children treated with apremilast was reduced by both measures, sPGA response, and PASI-75.
 

Safety of apremilast in children

“The overall safety profile during the placebo-controlled phase was comparable with the known safety profile of apremilast,” Dr. Belloni Fontina reported. “No new safety signals were identified.”

The rate of any adverse event was substantially higher in children given the active treatment, however, at 65% versus 41.3% for placebo.

Rates of severe and serious adverse events were low, at around 1.3%, and similar between the groups.

There was also a low rate of withdrawal because of side effects, although this was higher in the apremilast group (3.1% vs. 1.3%).

The primary reason for withdrawal of apremilast treatment were the most commonly reported adverse events: gastrointestinal disorders, including diarrhea, nausea, upper and lower abdominal pain, and vomiting. Headache, pyrexia, and nasopharyngitis were also reported.

Despite being common, most treatment-related adverse effects resolved within 3 days, Dr. Belloni Fontina said.
 

Expect further data

Further data from the trial are to be expected, because only the 16-week primary endpoint results have been released so far. The trial also included a 36-week extension phase, during which all children who had originally been randomly assigned to placebo were now eligible to be treated with apremilast, and all those who were originally given the active treatment were able to continue. This extension treatment period means that data will be available for a full year of treatment, and there will also be a further 2-week observational follow-up at the end of the trial.

The study was funded by Amgen. Dr. Belloni Fontina reported acting as an investigator and advisory board member for and receiving honoraria from Amgen, Galderma, Leo Pharma, and Pfizer. She also reported speaking on behalf of Pierre-Fabre and Galderma.

A version of this article first appeared on Medscape.com.

One gene and pathogenic missense variants in that gene account for most cases of uncombable hair syndrome (UHS), a rare hair shaft anomaly that manifests during infancy, investigators have reported.

The findings are from a cohort study published in JAMA Dermatology, which involved 107 unrelated children and adults suspected of having UHS, as well as family members, all of whom were recruited from January 2013 to December 2021. Genetic analyses were conducted in Germany from January 2014 to December 2021 with exome sequencing.
 

Study builds on prior research

Senior author Regina C. Betz, MD, professor of dermatogenetics at the Institute of Human Genetics, University Hospital Bonn, Germany, said that in 2016, she and her coinvestigators authored a study on the molecular genetics of UHS. That study, which involved 18 people with UHS, identified variants in three genes – PADI3, TCHH, and TGM3 – that encode proteins that play a role in the formation of the hair shaft. The investigators described how a deficiency in the shaping and mechanical strengthening of the hair shaft occurs in the UHS phenotype, which is characterized by dry, frizzy, and wiry hair that cannot be combed flat.

As a result of that previous work, “we base the assignment or confirmation of a clinical diagnosis of UHS on molecular genetic diagnostics,” the authors write in the new study, rather than on the clinical appearance of the hair and the physical examination of the patient, with confirmation on microscopical examination of the hair shaft.
 

Social media as instrument in finding study participants

Following the 2016 study, Dr. Betz and colleagues were contacted by many clinicians and by the public through Facebook and other social media platforms with details about possible cases of UHS, an autosomal recessive disorder. Through these contacts, blood samples, saliva, or DNA was sent to the investigators’ laboratory from 89 unrelated index patients (69 female patients, 20 male patients) suspected of having UHS. This resulted in the identification of pathogenic variants in 69 cases, the investigators write.

“In the first study, we had 18 patients, and then we tried to collect as many as possible” to determine the main mechanism behind UHS, Dr. Betz said. One question is whether there are additional genes responsible for UHS, she noted. “Even now, we are not sure, because in 25% [of cases in the new study], we didn’t find any mutation in the three known genes.”

The current study resulted in the discovery of eight novel pathogenic variants in PADI3, which are responsible for 71.0% (76) of the 107 cases. Of those, “6 were single observations and 2 were observed in 3 and 2 individuals, respectively,” the investigators write.

Children can grow out of this disorder, but it can also persist into adulthood, Dr. Betz noted. Communication that investigators had with parents of the children with UHS revealed that these children are often the targets of bullying by other children, she added.

She and her and colleagues will continue this research and are currently studying adults who have UHS.
 

Research leads to possible treatment pathways

Jeff Donovan, MD, FRCPC, FAAD, a dermatologist and medical director of the Donovan Hair Clinic in Whistler, British Columbia, described these findings as fundamental to understanding UHS and creating pathways to possible treatments.

The study “identifies more about the genetic basis of this challenging condition,” said Dr. Donovan, who is also clinical instructor in the department of dermatology at the University of British Columbia, Vancouver, and president of the Canadian Hair Loss Foundation. “We really need this type of information in order to have any sort of clue in terms of how to treat it,” he told this news organization.

“In the hair loss world, it’s pretty clear that if you can understand the genetic basis of things, or the basic science of a condition, whether it’s the basic genetics or the basic immunology, you give yourself the best chance to develop good treatments,” said Dr. Donovan.

The article provides advanced genetic information of the condition, such that geneticists can test for at least three markers if they are suspecting UHS, Dr. Donovan observed.
 

Condition can lead to bullying

Dr. Donovan also commented that UHS can have a detrimental impact on children with regard to socializing with their peers. “Having hair that sticks out and is very full like this is challenging because kids do get teased,” he said.

“It is often the parents who are the most affected” when a child aged 2-5 years has a hair condition such as UHS. But at age 5-9, “children are developing self-identity and an understanding of various aspects of self-esteem and what they look like and what others look like. And that’s where the teasing really starts. And that’s where it does become troublesome.”

Dr. Betz and Dr. Donovan have disclosed no relevant financial relationships.

A version of this article first appeared on Medscape.com.

One gene and pathogenic missense variants in that gene account for most cases of uncombable hair syndrome (UHS), a rare hair shaft anomaly that manifests during infancy, investigators have reported.

The findings are from a cohort study published in JAMA Dermatology, which involved 107 unrelated children and adults suspected of having UHS, as well as family members, all of whom were recruited from January 2013 to December 2021. Genetic analyses were conducted in Germany from January 2014 to December 2021 with exome sequencing.
 

Study builds on prior research

Senior author Regina C. Betz, MD, professor of dermatogenetics at the Institute of Human Genetics, University Hospital Bonn, Germany, said that in 2016, she and her coinvestigators authored a study on the molecular genetics of UHS. That study, which involved 18 people with UHS, identified variants in three genes – PADI3, TCHH, and TGM3 – that encode proteins that play a role in the formation of the hair shaft. The investigators described how a deficiency in the shaping and mechanical strengthening of the hair shaft occurs in the UHS phenotype, which is characterized by dry, frizzy, and wiry hair that cannot be combed flat.

As a result of that previous work, “we base the assignment or confirmation of a clinical diagnosis of UHS on molecular genetic diagnostics,” the authors write in the new study, rather than on the clinical appearance of the hair and the physical examination of the patient, with confirmation on microscopical examination of the hair shaft.
 

Social media as instrument in finding study participants

Following the 2016 study, Dr. Betz and colleagues were contacted by many clinicians and by the public through Facebook and other social media platforms with details about possible cases of UHS, an autosomal recessive disorder. Through these contacts, blood samples, saliva, or DNA was sent to the investigators’ laboratory from 89 unrelated index patients (69 female patients, 20 male patients) suspected of having UHS. This resulted in the identification of pathogenic variants in 69 cases, the investigators write.

“In the first study, we had 18 patients, and then we tried to collect as many as possible” to determine the main mechanism behind UHS, Dr. Betz said. One question is whether there are additional genes responsible for UHS, she noted. “Even now, we are not sure, because in 25% [of cases in the new study], we didn’t find any mutation in the three known genes.”

The current study resulted in the discovery of eight novel pathogenic variants in PADI3, which are responsible for 71.0% (76) of the 107 cases. Of those, “6 were single observations and 2 were observed in 3 and 2 individuals, respectively,” the investigators write.

Children can grow out of this disorder, but it can also persist into adulthood, Dr. Betz noted. Communication that investigators had with parents of the children with UHS revealed that these children are often the targets of bullying by other children, she added.

She and her and colleagues will continue this research and are currently studying adults who have UHS.
 

Research leads to possible treatment pathways

Jeff Donovan, MD, FRCPC, FAAD, a dermatologist and medical director of the Donovan Hair Clinic in Whistler, British Columbia, described these findings as fundamental to understanding UHS and creating pathways to possible treatments.

The study “identifies more about the genetic basis of this challenging condition,” said Dr. Donovan, who is also clinical instructor in the department of dermatology at the University of British Columbia, Vancouver, and president of the Canadian Hair Loss Foundation. “We really need this type of information in order to have any sort of clue in terms of how to treat it,” he told this news organization.

“In the hair loss world, it’s pretty clear that if you can understand the genetic basis of things, or the basic science of a condition, whether it’s the basic genetics or the basic immunology, you give yourself the best chance to develop good treatments,” said Dr. Donovan.

The article provides advanced genetic information of the condition, such that geneticists can test for at least three markers if they are suspecting UHS, Dr. Donovan observed.
 

Condition can lead to bullying

Dr. Donovan also commented that UHS can have a detrimental impact on children with regard to socializing with their peers. “Having hair that sticks out and is very full like this is challenging because kids do get teased,” he said.

“It is often the parents who are the most affected” when a child aged 2-5 years has a hair condition such as UHS. But at age 5-9, “children are developing self-identity and an understanding of various aspects of self-esteem and what they look like and what others look like. And that’s where the teasing really starts. And that’s where it does become troublesome.”

Dr. Betz and Dr. Donovan have disclosed no relevant financial relationships.

A version of this article first appeared on Medscape.com.

One gene and pathogenic missense variants in that gene account for most cases of uncombable hair syndrome (UHS), a rare hair shaft anomaly that manifests during infancy, investigators have reported.

The findings are from a cohort study published in JAMA Dermatology, which involved 107 unrelated children and adults suspected of having UHS, as well as family members, all of whom were recruited from January 2013 to December 2021. Genetic analyses were conducted in Germany from January 2014 to December 2021 with exome sequencing.
 

Study builds on prior research

Senior author Regina C. Betz, MD, professor of dermatogenetics at the Institute of Human Genetics, University Hospital Bonn, Germany, said that in 2016, she and her coinvestigators authored a study on the molecular genetics of UHS. That study, which involved 18 people with UHS, identified variants in three genes – PADI3, TCHH, and TGM3 – that encode proteins that play a role in the formation of the hair shaft. The investigators described how a deficiency in the shaping and mechanical strengthening of the hair shaft occurs in the UHS phenotype, which is characterized by dry, frizzy, and wiry hair that cannot be combed flat.

As a result of that previous work, “we base the assignment or confirmation of a clinical diagnosis of UHS on molecular genetic diagnostics,” the authors write in the new study, rather than on the clinical appearance of the hair and the physical examination of the patient, with confirmation on microscopical examination of the hair shaft.
 

Social media as instrument in finding study participants

Following the 2016 study, Dr. Betz and colleagues were contacted by many clinicians and by the public through Facebook and other social media platforms with details about possible cases of UHS, an autosomal recessive disorder. Through these contacts, blood samples, saliva, or DNA was sent to the investigators’ laboratory from 89 unrelated index patients (69 female patients, 20 male patients) suspected of having UHS. This resulted in the identification of pathogenic variants in 69 cases, the investigators write.

“In the first study, we had 18 patients, and then we tried to collect as many as possible” to determine the main mechanism behind UHS, Dr. Betz said. One question is whether there are additional genes responsible for UHS, she noted. “Even now, we are not sure, because in 25% [of cases in the new study], we didn’t find any mutation in the three known genes.”

The current study resulted in the discovery of eight novel pathogenic variants in PADI3, which are responsible for 71.0% (76) of the 107 cases. Of those, “6 were single observations and 2 were observed in 3 and 2 individuals, respectively,” the investigators write.

Children can grow out of this disorder, but it can also persist into adulthood, Dr. Betz noted. Communication that investigators had with parents of the children with UHS revealed that these children are often the targets of bullying by other children, she added.

She and her and colleagues will continue this research and are currently studying adults who have UHS.
 

Research leads to possible treatment pathways

Jeff Donovan, MD, FRCPC, FAAD, a dermatologist and medical director of the Donovan Hair Clinic in Whistler, British Columbia, described these findings as fundamental to understanding UHS and creating pathways to possible treatments.

The study “identifies more about the genetic basis of this challenging condition,” said Dr. Donovan, who is also clinical instructor in the department of dermatology at the University of British Columbia, Vancouver, and president of the Canadian Hair Loss Foundation. “We really need this type of information in order to have any sort of clue in terms of how to treat it,” he told this news organization.

“In the hair loss world, it’s pretty clear that if you can understand the genetic basis of things, or the basic science of a condition, whether it’s the basic genetics or the basic immunology, you give yourself the best chance to develop good treatments,” said Dr. Donovan.

The article provides advanced genetic information of the condition, such that geneticists can test for at least three markers if they are suspecting UHS, Dr. Donovan observed.
 

Condition can lead to bullying

Dr. Donovan also commented that UHS can have a detrimental impact on children with regard to socializing with their peers. “Having hair that sticks out and is very full like this is challenging because kids do get teased,” he said.

“It is often the parents who are the most affected” when a child aged 2-5 years has a hair condition such as UHS. But at age 5-9, “children are developing self-identity and an understanding of various aspects of self-esteem and what they look like and what others look like. And that’s where the teasing really starts. And that’s where it does become troublesome.”

Dr. Betz and Dr. Donovan have disclosed no relevant financial relationships.

A version of this article first appeared on Medscape.com.

Tralokinumab has received a positive opinion from the European Medicine Agency’s Committee for Medicinal Products for Human Use to extend use to adolescents aged 12 years and older with moderate-to-severe atopic dermatitis (AD) who are candidates for systemic therapy, according to a statement from the manufacturer.

The positive CHMP opinion, issued on Sept. 15, recommends extending the use of tralokinumab (Adtralza), an interleukin-13 antagonist, to adolescents aged 12-17 years in the EU. The positive opinion recommends an initial dose of 600 mg administered subcutaneously followed by 300 mg every other week, the dosing recommended for adults.

In December 2021, tralokinumab was approved for adults with moderate to severe AD in the United States, where it is marketed as Adbry. It is also approved for adults in the EU, Great Britain, Canada, the United Arab Emirates, and Switzerland. It is not currently approved for treatment of adolescents in any country, according to the LEO Pharma statement.

A regulatory filing with the U.S. Food and Drug Administration is in progress, the company said, and an additional study of tralokinumab for individuals aged 12 years and older is underway, according to the manufacturer.

The CHMP opinion was supported by data from a phase 3 study (ECZTRA 6) that assessed safety and efficacy of 150-mg or 300-mg doses of tralokinumab, compared with placebo in adolescents with moderate-to-severe AD, the company statement said. The primary outcomes were an Investigator Global Assessment score of clear or almost clear skin (IGA 0/1) and an improvement of at least a 75% on the Eczema Area and Severity Index score (EASI-75). In the study, presented as a poster at a meeting in October 2021, a total of 195 adolescents aged 12-17 with moderate to severe AD who were candidates for systemic therapy were randomly assigned to tralokinumab and 94 to placebo.

At 16 weeks, 21.4% and 17.5% of patients who received 150 mg and 300 mg, respectively, of tralokinumab had IGA scores of 0 or 1, compared with 4.3% of those on placebo (P < .001, P = .002, respectively vs. placebo). In addition, 28.6% and 27.8% of the 150-mg and 300-mg tralokinumab groups, respectively, achieved EASI-75, compared with 6.4% of placebo patients (P < .001, P = .001, respectively, compared with placebo).

Adverse events were similar between the groups, and most were mild or moderate; overall safety profiles were similar to those seen in adult patients.

The European Commission will review the positive opinion and make a final decision.

The research was supported by LEO Pharma.

A version of this article first appeared on Medscape.com.

Tralokinumab has received a positive opinion from the European Medicine Agency’s Committee for Medicinal Products for Human Use to extend use to adolescents aged 12 years and older with moderate-to-severe atopic dermatitis (AD) who are candidates for systemic therapy, according to a statement from the manufacturer.

The positive CHMP opinion, issued on Sept. 15, recommends extending the use of tralokinumab (Adtralza), an interleukin-13 antagonist, to adolescents aged 12-17 years in the EU. The positive opinion recommends an initial dose of 600 mg administered subcutaneously followed by 300 mg every other week, the dosing recommended for adults.

In December 2021, tralokinumab was approved for adults with moderate to severe AD in the United States, where it is marketed as Adbry. It is also approved for adults in the EU, Great Britain, Canada, the United Arab Emirates, and Switzerland. It is not currently approved for treatment of adolescents in any country, according to the LEO Pharma statement.

A regulatory filing with the U.S. Food and Drug Administration is in progress, the company said, and an additional study of tralokinumab for individuals aged 12 years and older is underway, according to the manufacturer.

The CHMP opinion was supported by data from a phase 3 study (ECZTRA 6) that assessed safety and efficacy of 150-mg or 300-mg doses of tralokinumab, compared with placebo in adolescents with moderate-to-severe AD, the company statement said. The primary outcomes were an Investigator Global Assessment score of clear or almost clear skin (IGA 0/1) and an improvement of at least a 75% on the Eczema Area and Severity Index score (EASI-75). In the study, presented as a poster at a meeting in October 2021, a total of 195 adolescents aged 12-17 with moderate to severe AD who were candidates for systemic therapy were randomly assigned to tralokinumab and 94 to placebo.

At 16 weeks, 21.4% and 17.5% of patients who received 150 mg and 300 mg, respectively, of tralokinumab had IGA scores of 0 or 1, compared with 4.3% of those on placebo (P < .001, P = .002, respectively vs. placebo). In addition, 28.6% and 27.8% of the 150-mg and 300-mg tralokinumab groups, respectively, achieved EASI-75, compared with 6.4% of placebo patients (P < .001, P = .001, respectively, compared with placebo).

Adverse events were similar between the groups, and most were mild or moderate; overall safety profiles were similar to those seen in adult patients.

The European Commission will review the positive opinion and make a final decision.

The research was supported by LEO Pharma.

A version of this article first appeared on Medscape.com.

Tralokinumab has received a positive opinion from the European Medicine Agency’s Committee for Medicinal Products for Human Use to extend use to adolescents aged 12 years and older with moderate-to-severe atopic dermatitis (AD) who are candidates for systemic therapy, according to a statement from the manufacturer.

The positive CHMP opinion, issued on Sept. 15, recommends extending the use of tralokinumab (Adtralza), an interleukin-13 antagonist, to adolescents aged 12-17 years in the EU. The positive opinion recommends an initial dose of 600 mg administered subcutaneously followed by 300 mg every other week, the dosing recommended for adults.

In December 2021, tralokinumab was approved for adults with moderate to severe AD in the United States, where it is marketed as Adbry. It is also approved for adults in the EU, Great Britain, Canada, the United Arab Emirates, and Switzerland. It is not currently approved for treatment of adolescents in any country, according to the LEO Pharma statement.

A regulatory filing with the U.S. Food and Drug Administration is in progress, the company said, and an additional study of tralokinumab for individuals aged 12 years and older is underway, according to the manufacturer.

The CHMP opinion was supported by data from a phase 3 study (ECZTRA 6) that assessed safety and efficacy of 150-mg or 300-mg doses of tralokinumab, compared with placebo in adolescents with moderate-to-severe AD, the company statement said. The primary outcomes were an Investigator Global Assessment score of clear or almost clear skin (IGA 0/1) and an improvement of at least a 75% on the Eczema Area and Severity Index score (EASI-75). In the study, presented as a poster at a meeting in October 2021, a total of 195 adolescents aged 12-17 with moderate to severe AD who were candidates for systemic therapy were randomly assigned to tralokinumab and 94 to placebo.

At 16 weeks, 21.4% and 17.5% of patients who received 150 mg and 300 mg, respectively, of tralokinumab had IGA scores of 0 or 1, compared with 4.3% of those on placebo (P < .001, P = .002, respectively vs. placebo). In addition, 28.6% and 27.8% of the 150-mg and 300-mg tralokinumab groups, respectively, achieved EASI-75, compared with 6.4% of placebo patients (P < .001, P = .001, respectively, compared with placebo).

Adverse events were similar between the groups, and most were mild or moderate; overall safety profiles were similar to those seen in adult patients.

The European Commission will review the positive opinion and make a final decision.

The research was supported by LEO Pharma.

A version of this article first appeared on Medscape.com.

In some cases, bruising or bleeding from bleeding disorders may look like signs of child abuse, but new guidance may help clinicians distinguish one from the other.

On Sept. 19 the American Academy of Pediatrics published two reports – a clinical report and a technical report – in the October 2022 issue of Pediatrics on evaluating for bleeding disorders when child abuse is suspected.

The reports were written by the AAP Section on Hematology/Oncology and the AAP Council on Child Abuse and Neglect.
 

One doesn’t rule out the other

The reports emphasize that laboratory testing of bleeding cannot always rule out abuse, just as a history of trauma (accidental or nonaccidental) may not rule out a bleeding disorder or other medical condition.

In the clinical report, led by James Anderst, MD, MSCI, with the division of child adversity and resilience, Children’s Mercy Hospital, University of Missouri–Kansas City, the researchers note that infants are at especially high risk of abusive bruising/bleeding, but bleeding disorders may also present in infancy.

The authors give an example of a situation when taking a thorough history won’t necessarily rule out a bleeding disorder: Male infants who have been circumcised with no significant bleeding issues may still have a bleeding disorder. Therefore, laboratory evaluations are often needed to detect disordered bleeding.

Children’s medications should be documented, the authors note, because certain drugs, such as nonsteroidal anti-inflammatory drugs, some antibiotics, antiepileptics, and herbal supplements, can affect tests that might be used to detect bleeding disorders.

Likewise, asking about restrictive or unusual diets or alternative therapies is important as some could increase the likelihood of bleeding/bruising.

Signs that bleeding disorder is not likely

The authors advise that, if a child has any of the following, an evaluation for a bleeding disorder is generally not needed:

• Caregivers’ description of trauma sufficiently explains the bruising.
• The child or an independent witness can provide a history of abuse or nonabusive trauma that explains the bruising.
• The outline of the bruising follows an object or hand pattern.
• The location of the bruising is on the ears, neck, or genitals.

“Bruising to the ears, neck, or genitals is rarely seen in either accidental injuries or in children with bleeding disorders,” the authors write.

Specification of which locations for injuries are more indicative of abuse in both mobile and immobile children was among the most important information from the paper, Seattle pediatrician Timothy Joos, MD, said in an interview.

Also very helpful, he said, was the listing of which tests should be done if bruising looks like potential abuse.

The authors write that if bruising is concerning for abuse that necessitates evaluation for bleeding disorders, the following tests should be done: PT (prothrombin time); aPTT (activated partial thromboplastin time); von Willebrand Factor (VWF) activity (Ristocetin cofactor); factor VIII activity level; factor IX activity level; and a complete blood count, including platelets.

“I think that’s what a lot of us suspected, but there’s not a lot of summary evidence regarding that until now,” Dr. Joos said.

Case-by-case decisions on when to test

The decision on whether to evaluate for a bleeding disorder may be made case by case.

If there is no obvious known trauma or intracranial hemorrhage (ICH), particularly subdural hematoma (SDH) in a nonmobile child, abuse should be suspected, the authors write.

They acknowledge that children can have ICH, such as a small SDH or an epidural hematoma, under the point of impact from a short fall.

“However,” the authors write, “short falls rarely result in significant brain injury.”

Conditions may affect screening tests

Screening tests for bleeding disorders can be falsely positive or falsely negative, the authors caution in the technical report, led by Shannon Carpenter, MD, MS, with the department of pediatrics, University of Missouri–Kansas City.

• If coagulation laboratory test specimens sit in a hot metal box all day, for instance, factor levels may be falsely low, the authors explain.
• Conversely, factors such as VWF and factor VIII are acute-phase reactants and factor levels will be deceptively high if blood specimens are taken in a stressful time.
• Patients who have a traumatic brain injury often show temporary coagulopathy that does not signal a congenital disorder.

Vitamin K deficiency

The technical report explains that if an infant, typically younger than 6 months, presents with bleeding/bruising that raises flags for abuse and has a long PT, clinicians should confirm vitamin K was provided at birth and/or testing for vitamin K deficiency should be performed.

Not all states require vitamin K to be administered at birth and some parents refuse it. Deficiency can lead to bleeding in the skin or from mucosal surfaces from circumcision, generalized ecchymoses, and large intramuscular hemorrhages or ICH.

When infants don’t get vitamin K at birth, vitamin K deficiency bleeding (VKDB) is seen most often in the first days of life, the technical report states. It can also occur 1-3 months after birth.

“Late VKDB occurs from the first month to 3 months after birth,” the authors write. “This deficiency is more prevalent in breast-fed babies, because human milk contains less vitamin K than does cow milk.”

Overall, the authors write, extensive lab tests are usually not necessary, given the rarity of most bleeding disorders and specific clinical factors that decrease the odds that a bleeding disorder caused the child’s findings.

Dr. Joos said the decisions described in this paper are the kind that can keep pediatricians up at night.

“Any kind of guidance is helpful in these difficult cases,” he said. “These are scenarios that can often happen in the middle of the night, and you’re often struggling with evidence or past experience that can help you make some of these decisions.”

Authors of the reports and Dr. Joos declared no relevant financial relationships.

In some cases, bruising or bleeding from bleeding disorders may look like signs of child abuse, but new guidance may help clinicians distinguish one from the other.

On Sept. 19 the American Academy of Pediatrics published two reports – a clinical report and a technical report – in the October 2022 issue of Pediatrics on evaluating for bleeding disorders when child abuse is suspected.

The reports were written by the AAP Section on Hematology/Oncology and the AAP Council on Child Abuse and Neglect.
 

One doesn’t rule out the other

The reports emphasize that laboratory testing of bleeding cannot always rule out abuse, just as a history of trauma (accidental or nonaccidental) may not rule out a bleeding disorder or other medical condition.

In the clinical report, led by James Anderst, MD, MSCI, with the division of child adversity and resilience, Children’s Mercy Hospital, University of Missouri–Kansas City, the researchers note that infants are at especially high risk of abusive bruising/bleeding, but bleeding disorders may also present in infancy.

The authors give an example of a situation when taking a thorough history won’t necessarily rule out a bleeding disorder: Male infants who have been circumcised with no significant bleeding issues may still have a bleeding disorder. Therefore, laboratory evaluations are often needed to detect disordered bleeding.

Children’s medications should be documented, the authors note, because certain drugs, such as nonsteroidal anti-inflammatory drugs, some antibiotics, antiepileptics, and herbal supplements, can affect tests that might be used to detect bleeding disorders.

Likewise, asking about restrictive or unusual diets or alternative therapies is important as some could increase the likelihood of bleeding/bruising.

Signs that bleeding disorder is not likely

The authors advise that, if a child has any of the following, an evaluation for a bleeding disorder is generally not needed:

• Caregivers’ description of trauma sufficiently explains the bruising.
• The child or an independent witness can provide a history of abuse or nonabusive trauma that explains the bruising.
• The outline of the bruising follows an object or hand pattern.
• The location of the bruising is on the ears, neck, or genitals.

“Bruising to the ears, neck, or genitals is rarely seen in either accidental injuries or in children with bleeding disorders,” the authors write.

Specification of which locations for injuries are more indicative of abuse in both mobile and immobile children was among the most important information from the paper, Seattle pediatrician Timothy Joos, MD, said in an interview.

Also very helpful, he said, was the listing of which tests should be done if bruising looks like potential abuse.

The authors write that if bruising is concerning for abuse that necessitates evaluation for bleeding disorders, the following tests should be done: PT (prothrombin time); aPTT (activated partial thromboplastin time); von Willebrand Factor (VWF) activity (Ristocetin cofactor); factor VIII activity level; factor IX activity level; and a complete blood count, including platelets.

“I think that’s what a lot of us suspected, but there’s not a lot of summary evidence regarding that until now,” Dr. Joos said.

Case-by-case decisions on when to test

The decision on whether to evaluate for a bleeding disorder may be made case by case.

If there is no obvious known trauma or intracranial hemorrhage (ICH), particularly subdural hematoma (SDH) in a nonmobile child, abuse should be suspected, the authors write.

They acknowledge that children can have ICH, such as a small SDH or an epidural hematoma, under the point of impact from a short fall.

“However,” the authors write, “short falls rarely result in significant brain injury.”

Conditions may affect screening tests

Screening tests for bleeding disorders can be falsely positive or falsely negative, the authors caution in the technical report, led by Shannon Carpenter, MD, MS, with the department of pediatrics, University of Missouri–Kansas City.

• If coagulation laboratory test specimens sit in a hot metal box all day, for instance, factor levels may be falsely low, the authors explain.
• Conversely, factors such as VWF and factor VIII are acute-phase reactants and factor levels will be deceptively high if blood specimens are taken in a stressful time.
• Patients who have a traumatic brain injury often show temporary coagulopathy that does not signal a congenital disorder.

Vitamin K deficiency

The technical report explains that if an infant, typically younger than 6 months, presents with bleeding/bruising that raises flags for abuse and has a long PT, clinicians should confirm vitamin K was provided at birth and/or testing for vitamin K deficiency should be performed.

Not all states require vitamin K to be administered at birth and some parents refuse it. Deficiency can lead to bleeding in the skin or from mucosal surfaces from circumcision, generalized ecchymoses, and large intramuscular hemorrhages or ICH.

When infants don’t get vitamin K at birth, vitamin K deficiency bleeding (VKDB) is seen most often in the first days of life, the technical report states. It can also occur 1-3 months after birth.

“Late VKDB occurs from the first month to 3 months after birth,” the authors write. “This deficiency is more prevalent in breast-fed babies, because human milk contains less vitamin K than does cow milk.”

Overall, the authors write, extensive lab tests are usually not necessary, given the rarity of most bleeding disorders and specific clinical factors that decrease the odds that a bleeding disorder caused the child’s findings.

Dr. Joos said the decisions described in this paper are the kind that can keep pediatricians up at night.

“Any kind of guidance is helpful in these difficult cases,” he said. “These are scenarios that can often happen in the middle of the night, and you’re often struggling with evidence or past experience that can help you make some of these decisions.”

Authors of the reports and Dr. Joos declared no relevant financial relationships.

In some cases, bruising or bleeding from bleeding disorders may look like signs of child abuse, but new guidance may help clinicians distinguish one from the other.

On Sept. 19 the American Academy of Pediatrics published two reports – a clinical report and a technical report – in the October 2022 issue of Pediatrics on evaluating for bleeding disorders when child abuse is suspected.

The reports were written by the AAP Section on Hematology/Oncology and the AAP Council on Child Abuse and Neglect.
 

One doesn’t rule out the other

The reports emphasize that laboratory testing of bleeding cannot always rule out abuse, just as a history of trauma (accidental or nonaccidental) may not rule out a bleeding disorder or other medical condition.

In the clinical report, led by James Anderst, MD, MSCI, with the division of child adversity and resilience, Children’s Mercy Hospital, University of Missouri–Kansas City, the researchers note that infants are at especially high risk of abusive bruising/bleeding, but bleeding disorders may also present in infancy.

The authors give an example of a situation when taking a thorough history won’t necessarily rule out a bleeding disorder: Male infants who have been circumcised with no significant bleeding issues may still have a bleeding disorder. Therefore, laboratory evaluations are often needed to detect disordered bleeding.

Children’s medications should be documented, the authors note, because certain drugs, such as nonsteroidal anti-inflammatory drugs, some antibiotics, antiepileptics, and herbal supplements, can affect tests that might be used to detect bleeding disorders.

Likewise, asking about restrictive or unusual diets or alternative therapies is important as some could increase the likelihood of bleeding/bruising.

Signs that bleeding disorder is not likely

The authors advise that, if a child has any of the following, an evaluation for a bleeding disorder is generally not needed:

• Caregivers’ description of trauma sufficiently explains the bruising.
• The child or an independent witness can provide a history of abuse or nonabusive trauma that explains the bruising.
• The outline of the bruising follows an object or hand pattern.
• The location of the bruising is on the ears, neck, or genitals.

“Bruising to the ears, neck, or genitals is rarely seen in either accidental injuries or in children with bleeding disorders,” the authors write.

Specification of which locations for injuries are more indicative of abuse in both mobile and immobile children was among the most important information from the paper, Seattle pediatrician Timothy Joos, MD, said in an interview.

Also very helpful, he said, was the listing of which tests should be done if bruising looks like potential abuse.

The authors write that if bruising is concerning for abuse that necessitates evaluation for bleeding disorders, the following tests should be done: PT (prothrombin time); aPTT (activated partial thromboplastin time); von Willebrand Factor (VWF) activity (Ristocetin cofactor); factor VIII activity level; factor IX activity level; and a complete blood count, including platelets.

“I think that’s what a lot of us suspected, but there’s not a lot of summary evidence regarding that until now,” Dr. Joos said.

Case-by-case decisions on when to test

The decision on whether to evaluate for a bleeding disorder may be made case by case.

If there is no obvious known trauma or intracranial hemorrhage (ICH), particularly subdural hematoma (SDH) in a nonmobile child, abuse should be suspected, the authors write.

They acknowledge that children can have ICH, such as a small SDH or an epidural hematoma, under the point of impact from a short fall.

“However,” the authors write, “short falls rarely result in significant brain injury.”

Conditions may affect screening tests

Screening tests for bleeding disorders can be falsely positive or falsely negative, the authors caution in the technical report, led by Shannon Carpenter, MD, MS, with the department of pediatrics, University of Missouri–Kansas City.

• If coagulation laboratory test specimens sit in a hot metal box all day, for instance, factor levels may be falsely low, the authors explain.
• Conversely, factors such as VWF and factor VIII are acute-phase reactants and factor levels will be deceptively high if blood specimens are taken in a stressful time.
• Patients who have a traumatic brain injury often show temporary coagulopathy that does not signal a congenital disorder.

Vitamin K deficiency

The technical report explains that if an infant, typically younger than 6 months, presents with bleeding/bruising that raises flags for abuse and has a long PT, clinicians should confirm vitamin K was provided at birth and/or testing for vitamin K deficiency should be performed.

Not all states require vitamin K to be administered at birth and some parents refuse it. Deficiency can lead to bleeding in the skin or from mucosal surfaces from circumcision, generalized ecchymoses, and large intramuscular hemorrhages or ICH.

When infants don’t get vitamin K at birth, vitamin K deficiency bleeding (VKDB) is seen most often in the first days of life, the technical report states. It can also occur 1-3 months after birth.

“Late VKDB occurs from the first month to 3 months after birth,” the authors write. “This deficiency is more prevalent in breast-fed babies, because human milk contains less vitamin K than does cow milk.”

Overall, the authors write, extensive lab tests are usually not necessary, given the rarity of most bleeding disorders and specific clinical factors that decrease the odds that a bleeding disorder caused the child’s findings.

Dr. Joos said the decisions described in this paper are the kind that can keep pediatricians up at night.

“Any kind of guidance is helpful in these difficult cases,” he said. “These are scenarios that can often happen in the middle of the night, and you’re often struggling with evidence or past experience that can help you make some of these decisions.”

Authors of the reports and Dr. Joos declared no relevant financial relationships.

Long-awaited global transgender care guidelines have dropped, with no recommendations regarding age limits for treatment and surgery in teenagers but acknowledging the complexity of dealing with such adolescents amid lack of longitudinal research on the impact of transitioning gender.

The World Professional Association of Transgender Health published its latest standards of care (SOC8) as it opens its annual meeting on Sept. 16 in Montreal.

Origovisualis/Getty Images

These are “the most comprehensive set of guidelines ever produced to assist health care professionals around the world in support of transgender and gender diverse adults, adolescents, and children who are taking steps to live their lives authentically,” wrote WPATH President Walter Bouman, MD, PhD, and WPATH President-Elect Marci Bowers, MD, in a news release.

The SOC8 is the first update to guidance on the treatment of transgender individuals in 10 years and appears online in the International Journal of Transgender Health.

For the first time, the association wrote a chapter dedicated to transgender and gender-diverse adolescents – distinct from the child chapter.
 

The complexity of treating adolescents

WPATH officials said that this was owed to exponential growth in adolescent referral rates, more research on adolescent gender diversity–related care, and the unique developmental and care issues of this age group.

Until recently, there was limited information regarding the prevalence of gender diversity among adolescents. Studies from high-school samples indicate much higher rates than was earlier thought, with reports of up to 1.2% of participants identifying as transgender and up to 2.7% or more (for example, 7%-9%) experiencing some level of self-reported gender diversity, WPATH said.

The new chapter “applies to adolescents from the start of puberty until the legal age of majority (in most cases 18 years),” it stated.

However, WPATH did not go as far as to recommend lowering the age at which youth can receive cross-sex hormone therapy or gender-affirming surgeries, as earlier decreed in a draft of the guidelines. That draft suggested that young people could receive hormone therapy at age 14 years and surgeries for double mastectomies at age 15 years and for genital reassignment at age 17 years.

The exception was phalloplasty – surgery to construct a penis in female-to-male individuals – which WPATH stressed should not be performed under the age of 18 years owing to its complexity.

Now, the final SOC8 emphasizes that each transgender adolescent is unique, and decisions must be made on an individual basis, with no recommendations on specific ages for any treatment. This could be interpreted in many ways.

The SOC8 also acknowledges the “very rare” regret of individuals who have transitioned to the opposite gender and then changed their minds.

“[Health care] providers may consider the possibility an adolescent may regret gender-affirming decisions made during adolescence, and a young person will want to stop treatment and return to living in the birth-assigned gender role in the future. Providers may discuss this topic in a collaborative and trusting manner with the adolescent and their parents/caregivers before gender-affirming medical treatments are started,” it states.

WPATH, in addition, stressed the importance of counseling and supporting regretting patients, many who “expressed difficulties finding help during their detransition process and reported their detransition was an isolating experience during which they did not receive either sufficient or appropriate support.”

Although it doesn’t put a firm figure on the rate of regret overall, in its chapter on surgery, WPATH estimates that 0.3%-3.8% of transgender individuals regret gender-affirming surgery.

SOC8 also acknowledges “A pattern of uneven ratios by assigned sex has been reported in gender clinics, with assigned female-at-birth patients initiating care 2.5-7.1 times more frequently” than patients who were assigned male at birth.

And WPATH states in SOC8 that another phenomenon is the growing number of adolescents seeking care who had not previously experienced or expressed gender diversity during their childhood years.

It goes on to cite the 2018 paper of Lisa Littman, MD, MPH, now president of the Institute for Comprehensive Gender Dysphoria Research. Dr. Littman coined the term, “rapid-onset gender dysphoria” to describe this phenomenon; SOC8 refrains from using this phrase, but does acknowledge: “For a select subgroup of young people, susceptibility to social influence impacting gender may be an important differential to consider.”

SOC8 recommends that before any medical or surgical treatment is considered, health care professionals “undertake a comprehensive biopsychosocial assessment of adolescents who present with gender identity-related concerns and seek medical/surgical transition-related care.”

And it specifically mentions that transgender adolescents “show high rates of autism spectrum disorder/characteristics,” and notes that “other neurodevelopmental presentations and/or mental health challenges may also be present, (e.g., ADHD, intellectual disability, and psychotic disorders).”

Who uses WPATH to guide care? This is ‘a big unknown’

WPATH is an umbrella organization with offshoots in most Western nations, such as USPATH in the United States, EPATH in Europe, and AUSPATH and NZPATH in Australia and New Zealand.

However, it is not the only organization to issue guidance on the care of transgender individuals; several specialties take care of this patient population, including, but not limited to: pediatricians, endocrinologists, psychiatrists, psychologists and plastic surgeons.

The extent to which any health care professional, or professional body, follows WPATH guidance is extremely varied.

“There is nothing binding clinicians to the SOC, and the SOC is so broad and vague that anyone can say they’re following it but according to their own biases and interpretation,” Aaron Kimberly, a trans man and mental health clinician from the Gender Dysphoria Alliance, said in an interview.

In North America, some clinics practice full “informed consent” with no assessment and prescriptions at the first visit, Mr. Kimberly said, whereas others do comprehensive assessments.

“I think SOC should be observed. It shouldn’t just be people going rogue,” Erica Anderson, a clinical psychologist in Berkeley, Calif., former president of USPATH, and former member of WPATH, who is herself transgender, said in an interview. “The reason there are standards of care is because hundreds of scientists have weighed in – is it perfect? No. We have a long way to go. But you can’t just ignore whatever it is that we know and let people make their own decisions.”

A version of this article first appeared on Medscape.com.

Long-awaited global transgender care guidelines have dropped, with no recommendations regarding age limits for treatment and surgery in teenagers but acknowledging the complexity of dealing with such adolescents amid lack of longitudinal research on the impact of transitioning gender.

The World Professional Association of Transgender Health published its latest standards of care (SOC8) as it opens its annual meeting on Sept. 16 in Montreal.

Origovisualis/Getty Images

These are “the most comprehensive set of guidelines ever produced to assist health care professionals around the world in support of transgender and gender diverse adults, adolescents, and children who are taking steps to live their lives authentically,” wrote WPATH President Walter Bouman, MD, PhD, and WPATH President-Elect Marci Bowers, MD, in a news release.

The SOC8 is the first update to guidance on the treatment of transgender individuals in 10 years and appears online in the International Journal of Transgender Health.

For the first time, the association wrote a chapter dedicated to transgender and gender-diverse adolescents – distinct from the child chapter.
 

The complexity of treating adolescents

WPATH officials said that this was owed to exponential growth in adolescent referral rates, more research on adolescent gender diversity–related care, and the unique developmental and care issues of this age group.

Until recently, there was limited information regarding the prevalence of gender diversity among adolescents. Studies from high-school samples indicate much higher rates than was earlier thought, with reports of up to 1.2% of participants identifying as transgender and up to 2.7% or more (for example, 7%-9%) experiencing some level of self-reported gender diversity, WPATH said.

The new chapter “applies to adolescents from the start of puberty until the legal age of majority (in most cases 18 years),” it stated.

However, WPATH did not go as far as to recommend lowering the age at which youth can receive cross-sex hormone therapy or gender-affirming surgeries, as earlier decreed in a draft of the guidelines. That draft suggested that young people could receive hormone therapy at age 14 years and surgeries for double mastectomies at age 15 years and for genital reassignment at age 17 years.

The exception was phalloplasty – surgery to construct a penis in female-to-male individuals – which WPATH stressed should not be performed under the age of 18 years owing to its complexity.

Now, the final SOC8 emphasizes that each transgender adolescent is unique, and decisions must be made on an individual basis, with no recommendations on specific ages for any treatment. This could be interpreted in many ways.

The SOC8 also acknowledges the “very rare” regret of individuals who have transitioned to the opposite gender and then changed their minds.

“[Health care] providers may consider the possibility an adolescent may regret gender-affirming decisions made during adolescence, and a young person will want to stop treatment and return to living in the birth-assigned gender role in the future. Providers may discuss this topic in a collaborative and trusting manner with the adolescent and their parents/caregivers before gender-affirming medical treatments are started,” it states.

WPATH, in addition, stressed the importance of counseling and supporting regretting patients, many who “expressed difficulties finding help during their detransition process and reported their detransition was an isolating experience during which they did not receive either sufficient or appropriate support.”

Although it doesn’t put a firm figure on the rate of regret overall, in its chapter on surgery, WPATH estimates that 0.3%-3.8% of transgender individuals regret gender-affirming surgery.

SOC8 also acknowledges “A pattern of uneven ratios by assigned sex has been reported in gender clinics, with assigned female-at-birth patients initiating care 2.5-7.1 times more frequently” than patients who were assigned male at birth.

And WPATH states in SOC8 that another phenomenon is the growing number of adolescents seeking care who had not previously experienced or expressed gender diversity during their childhood years.

It goes on to cite the 2018 paper of Lisa Littman, MD, MPH, now president of the Institute for Comprehensive Gender Dysphoria Research. Dr. Littman coined the term, “rapid-onset gender dysphoria” to describe this phenomenon; SOC8 refrains from using this phrase, but does acknowledge: “For a select subgroup of young people, susceptibility to social influence impacting gender may be an important differential to consider.”

SOC8 recommends that before any medical or surgical treatment is considered, health care professionals “undertake a comprehensive biopsychosocial assessment of adolescents who present with gender identity-related concerns and seek medical/surgical transition-related care.”

And it specifically mentions that transgender adolescents “show high rates of autism spectrum disorder/characteristics,” and notes that “other neurodevelopmental presentations and/or mental health challenges may also be present, (e.g., ADHD, intellectual disability, and psychotic disorders).”

Who uses WPATH to guide care? This is ‘a big unknown’

WPATH is an umbrella organization with offshoots in most Western nations, such as USPATH in the United States, EPATH in Europe, and AUSPATH and NZPATH in Australia and New Zealand.

However, it is not the only organization to issue guidance on the care of transgender individuals; several specialties take care of this patient population, including, but not limited to: pediatricians, endocrinologists, psychiatrists, psychologists and plastic surgeons.

The extent to which any health care professional, or professional body, follows WPATH guidance is extremely varied.

“There is nothing binding clinicians to the SOC, and the SOC is so broad and vague that anyone can say they’re following it but according to their own biases and interpretation,” Aaron Kimberly, a trans man and mental health clinician from the Gender Dysphoria Alliance, said in an interview.

In North America, some clinics practice full “informed consent” with no assessment and prescriptions at the first visit, Mr. Kimberly said, whereas others do comprehensive assessments.

“I think SOC should be observed. It shouldn’t just be people going rogue,” Erica Anderson, a clinical psychologist in Berkeley, Calif., former president of USPATH, and former member of WPATH, who is herself transgender, said in an interview. “The reason there are standards of care is because hundreds of scientists have weighed in – is it perfect? No. We have a long way to go. But you can’t just ignore whatever it is that we know and let people make their own decisions.”

A version of this article first appeared on Medscape.com.

Long-awaited global transgender care guidelines have dropped, with no recommendations regarding age limits for treatment and surgery in teenagers but acknowledging the complexity of dealing with such adolescents amid lack of longitudinal research on the impact of transitioning gender.

The World Professional Association of Transgender Health published its latest standards of care (SOC8) as it opens its annual meeting on Sept. 16 in Montreal.

Origovisualis/Getty Images

These are “the most comprehensive set of guidelines ever produced to assist health care professionals around the world in support of transgender and gender diverse adults, adolescents, and children who are taking steps to live their lives authentically,” wrote WPATH President Walter Bouman, MD, PhD, and WPATH President-Elect Marci Bowers, MD, in a news release.

The SOC8 is the first update to guidance on the treatment of transgender individuals in 10 years and appears online in the International Journal of Transgender Health.

For the first time, the association wrote a chapter dedicated to transgender and gender-diverse adolescents – distinct from the child chapter.
 

The complexity of treating adolescents

WPATH officials said that this was owed to exponential growth in adolescent referral rates, more research on adolescent gender diversity–related care, and the unique developmental and care issues of this age group.

Until recently, there was limited information regarding the prevalence of gender diversity among adolescents. Studies from high-school samples indicate much higher rates than was earlier thought, with reports of up to 1.2% of participants identifying as transgender and up to 2.7% or more (for example, 7%-9%) experiencing some level of self-reported gender diversity, WPATH said.

The new chapter “applies to adolescents from the start of puberty until the legal age of majority (in most cases 18 years),” it stated.

However, WPATH did not go as far as to recommend lowering the age at which youth can receive cross-sex hormone therapy or gender-affirming surgeries, as earlier decreed in a draft of the guidelines. That draft suggested that young people could receive hormone therapy at age 14 years and surgeries for double mastectomies at age 15 years and for genital reassignment at age 17 years.

The exception was phalloplasty – surgery to construct a penis in female-to-male individuals – which WPATH stressed should not be performed under the age of 18 years owing to its complexity.

Now, the final SOC8 emphasizes that each transgender adolescent is unique, and decisions must be made on an individual basis, with no recommendations on specific ages for any treatment. This could be interpreted in many ways.

The SOC8 also acknowledges the “very rare” regret of individuals who have transitioned to the opposite gender and then changed their minds.

“[Health care] providers may consider the possibility an adolescent may regret gender-affirming decisions made during adolescence, and a young person will want to stop treatment and return to living in the birth-assigned gender role in the future. Providers may discuss this topic in a collaborative and trusting manner with the adolescent and their parents/caregivers before gender-affirming medical treatments are started,” it states.

WPATH, in addition, stressed the importance of counseling and supporting regretting patients, many who “expressed difficulties finding help during their detransition process and reported their detransition was an isolating experience during which they did not receive either sufficient or appropriate support.”

Although it doesn’t put a firm figure on the rate of regret overall, in its chapter on surgery, WPATH estimates that 0.3%-3.8% of transgender individuals regret gender-affirming surgery.

SOC8 also acknowledges “A pattern of uneven ratios by assigned sex has been reported in gender clinics, with assigned female-at-birth patients initiating care 2.5-7.1 times more frequently” than patients who were assigned male at birth.

And WPATH states in SOC8 that another phenomenon is the growing number of adolescents seeking care who had not previously experienced or expressed gender diversity during their childhood years.

It goes on to cite the 2018 paper of Lisa Littman, MD, MPH, now president of the Institute for Comprehensive Gender Dysphoria Research. Dr. Littman coined the term, “rapid-onset gender dysphoria” to describe this phenomenon; SOC8 refrains from using this phrase, but does acknowledge: “For a select subgroup of young people, susceptibility to social influence impacting gender may be an important differential to consider.”

SOC8 recommends that before any medical or surgical treatment is considered, health care professionals “undertake a comprehensive biopsychosocial assessment of adolescents who present with gender identity-related concerns and seek medical/surgical transition-related care.”

And it specifically mentions that transgender adolescents “show high rates of autism spectrum disorder/characteristics,” and notes that “other neurodevelopmental presentations and/or mental health challenges may also be present, (e.g., ADHD, intellectual disability, and psychotic disorders).”

Who uses WPATH to guide care? This is ‘a big unknown’

WPATH is an umbrella organization with offshoots in most Western nations, such as USPATH in the United States, EPATH in Europe, and AUSPATH and NZPATH in Australia and New Zealand.

However, it is not the only organization to issue guidance on the care of transgender individuals; several specialties take care of this patient population, including, but not limited to: pediatricians, endocrinologists, psychiatrists, psychologists and plastic surgeons.

The extent to which any health care professional, or professional body, follows WPATH guidance is extremely varied.

“There is nothing binding clinicians to the SOC, and the SOC is so broad and vague that anyone can say they’re following it but according to their own biases and interpretation,” Aaron Kimberly, a trans man and mental health clinician from the Gender Dysphoria Alliance, said in an interview.

In North America, some clinics practice full “informed consent” with no assessment and prescriptions at the first visit, Mr. Kimberly said, whereas others do comprehensive assessments.

“I think SOC should be observed. It shouldn’t just be people going rogue,” Erica Anderson, a clinical psychologist in Berkeley, Calif., former president of USPATH, and former member of WPATH, who is herself transgender, said in an interview. “The reason there are standards of care is because hundreds of scientists have weighed in – is it perfect? No. We have a long way to go. But you can’t just ignore whatever it is that we know and let people make their own decisions.”

A version of this article first appeared on Medscape.com.

Who would have thought we would need to refresh our knowledge on polio virus in 2022? Fate seems cruel to add this concern on the heels of SARS-CoV-2, monkeypox, abnormal seasons for RSV, acute flaccid myelitis (AFM) linked to enteroviruses, and a summer of parechovirus causing infant meningitis. But confirmation that indeed an adult had polio with paralytic disease raises concerns among public health groups and ordinary citizens alike, particularly those who remember polio in its heyday.

History: In the summer of 1952, polio was among the most feared diseases on the planet. Families were advised to not allow children to congregate in groups or use public swimming pools; little league baseball games were being canceled and there was talk of not opening schools for the fall. Every parent’s nightmare seemed to be the nonspecific febrile summer illness that led to paralytic sequelae. TV news included videos of the iron lung wards in hospitals across the country. Medical providers felt powerless, only able to give nonspecific preventive advice. There was no specific antiviral (there still isn’t) and vaccines seemed a long way off.

Then came the news that Dr. Jonas Salk’s group had gotten an inactivated polio vaccine (IPV) approved for general use in 1955. Families were excited to have their children vaccinated. Paralytic polio cases dropped like a rock from approximately 22,000/year in 1952 to approximately 2,200 in 1956. A surge to near 6,000 cases in 1959 led to Dr. Albert Sabin’s oral polio vaccine (OPV), which supplanted IPV in 1961. OPV had the advantages of: 1) Inducing mucosal as well as serum antibodies, 2) more durable responses, and 3) immunity in unvaccinated persons exposed to vaccine virus that had been shed in stools into wastewater and rivers.

By 1964, polio had nearly disappeared. The last wild-type indigenous U.S. case was in 1979. By 1994, all the Americas were declared polio free. Because the only U.S. paralytic polio cases thereafter were foreign imports or were associated with oral vaccine strains (so-called vaccine-associated paralytic polio [VAPP]), OPV was replaced by an enhanced IPV in 2000 to prevent further VAPP.

Polio facts: Polio is asymptomatic in about 70% of infections. Among the 30% with symptoms, paralysis occurs infrequently, with the overall rate of paralytic infections being 0.5% (rate varies by virus type with type 3 having the highest rate).¹ Why then was the world so afraid of polio? If every person in a U.S. birth cohort (about 3.7 million) was unvaccinated and became infected with poliovirus, more than 18,000 would get paralytic polio and almost 1,300 would die. Of note, adults have a higher chance of paralytic polio after infection than children.

Concerns in 2022: Persons vaccinated with at least three doses of either IPV or OPV have historically been protected from paralytic polio (99% protection). But are we sure that the United States remains protected against polio after 2 decades of IPV being the only vaccine? Polio could be reintroduced at any time to the United States from countries with reported cases that likely arose because of low vaccination rates related to war, famine, or political upheavals (Malawi, Mozambique, Nigeria, Pakistan, and Afghanistan).² The proof? The recent confirmed New York case.

International efforts resulted in global eradication of two polio wild-types viruses (type 2 in 2015 and type 3 in 2019). Nevertheless, vaccine-derived, virulent polio virus (VDPV) type 2 and VDPV-3 still circulate in some areas, particularly Africa (VDPV-2) and Israel (VDPV-3). The above-mentioned U.S. case is an unvaccinated adult traveler who went to an area where VDPV-2 circulates and developed disease after returning home.³ So, it was not an indigenous reappearance in the United States and it was not a breakthrough case in a vaccinated person. But it is sobering to realize that all who are unvaccinated remain at risk for paralytic polio in 2022, particularly because vaccination rates declined nearly everywhere during the initial COVID-19 pandemic. We are still catching up, with vaccination rates under 50% in some ZIP codes.⁴

Are VDPVs circulating in some parts of the United States? Interestingly, wastewater surveillance programs may be the most economical and practical way to perform polio surveillance. Such a program detected polio virus in London wastewater in June 2022.⁵ New York has recently detected polio in wastewater during testing begun because of the recent case.⁶

Good news: For paralytic polio, seropositivity at any titer indicates protection, so U.S. serosurveillance data would also be informative. How durable is polio protection in the IPV era? Available data suggest that even though we have used only IPV these past 20 years, seropositivity rates among vaccinees with at least three doses of either IPV or OPV should persist for decades and likely for life. Even before polio became a concern this year, the Centers for Disease Control and Prevention, being proactive, wanted to ensure that the enhanced IPV was producing durable immunity and that persons of all ages remained seropositive to the three polio virus types over 10 years after discontinuing OPV use in 2012.

The CDC collaborated with investigators in Kansas City, Mo., to evaluate titers and seropositivity to all three types in a 2- to 85-year-old otherwise healthy cohort with demographics that mirrored the 2010 census for the Kansas City region, which in turn mirrored the national 2021 census data.⁷ There were approximately 100 persons in each age cohort, with 200 below age 11 years (the cohort that had received only IPV). Serology was performed at the CDC.

Overall seropositivity rates were high, but lower for type 3 (83.3%) and type 2 (90.7%) than type 1 (94.4%). Of note, most of those seronegative for one or more types were among 2- to 3-year-olds who had not completed their full IPV series, with most seronegative results being against polio types 1 and 3. Further, five, who were confirmed as having received no polio vaccine, were seronegative for all three types. Two with no available vaccine records (over 18 years old) were also seronegative for all three types.

So, regardless of the era in which one got polio vaccine, vaccine protection appears to persist indefinitely after three doses. Even 80-year-olds were still seropositive if they had three doses. We can confidently reassure our patients that the vaccine still works; the persons who need to fear polio in 2022 are those who are not vaccinated or have had fewer than three doses, particularly if they travel to areas of persistent polio. Wild type 1 virus persists in a few countries as does VDPV type 2 and VDPV type 3. Importantly, wild type 2 and wild type 3 (with the lowest seropositivity in 2012 study) have been eliminated globally so the only circulating type 2 and type 3 polio virus is VDPV in a few countries. Travel to these countries warrants review of polio vaccine records and CDC or WHO current recommendations for travelers to those countries.
 

Dr. Harrison is a professor, University of Missouri Kansas City School of Medicine, department of medicine, infectious diseases section, Kansas City. Email him at pdnews@mdedge.com.

References

1. Poliomyelitis. World Health Organization fact sheet, 2022 Jul 4..

2. Franco-Paredes C et al. Lancet Infect Dis. 2022 Aug 16. doi: 10.1016/S1473-3099(22)00548-5.

3. Link-Gelles R et al. MMWR Morb Mortal Wkly Rep. 2022 Aug 19;71(33):1065-8.

4. “Polio vaccination rate for 2-year-olds is as low as 37% in parts of N.Y. county where paralysis case was found,” NBC News, Erika Edwards, 2022 Aug 16. 5. Vaccine-derived poliovirus type 2 (VDPV2) detected in environmental samples in London. Polioeradication.org. 2022 Jun 22.

6. “NYSDOH and NYCDOHMH wastewater monitoring identifies polio in New York City and urges unvaccinated New Yorkers to get vaccinated now,” nyc.gov. 2022 Aug 12.

7. Wallace GS et al. Hum Vaccin Immunother. 2017;13(4):776-83.

Who would have thought we would need to refresh our knowledge on polio virus in 2022? Fate seems cruel to add this concern on the heels of SARS-CoV-2, monkeypox, abnormal seasons for RSV, acute flaccid myelitis (AFM) linked to enteroviruses, and a summer of parechovirus causing infant meningitis. But confirmation that indeed an adult had polio with paralytic disease raises concerns among public health groups and ordinary citizens alike, particularly those who remember polio in its heyday.

History: In the summer of 1952, polio was among the most feared diseases on the planet. Families were advised to not allow children to congregate in groups or use public swimming pools; little league baseball games were being canceled and there was talk of not opening schools for the fall. Every parent’s nightmare seemed to be the nonspecific febrile summer illness that led to paralytic sequelae. TV news included videos of the iron lung wards in hospitals across the country. Medical providers felt powerless, only able to give nonspecific preventive advice. There was no specific antiviral (there still isn’t) and vaccines seemed a long way off.

Then came the news that Dr. Jonas Salk’s group had gotten an inactivated polio vaccine (IPV) approved for general use in 1955. Families were excited to have their children vaccinated. Paralytic polio cases dropped like a rock from approximately 22,000/year in 1952 to approximately 2,200 in 1956. A surge to near 6,000 cases in 1959 led to Dr. Albert Sabin’s oral polio vaccine (OPV), which supplanted IPV in 1961. OPV had the advantages of: 1) Inducing mucosal as well as serum antibodies, 2) more durable responses, and 3) immunity in unvaccinated persons exposed to vaccine virus that had been shed in stools into wastewater and rivers.

By 1964, polio had nearly disappeared. The last wild-type indigenous U.S. case was in 1979. By 1994, all the Americas were declared polio free. Because the only U.S. paralytic polio cases thereafter were foreign imports or were associated with oral vaccine strains (so-called vaccine-associated paralytic polio [VAPP]), OPV was replaced by an enhanced IPV in 2000 to prevent further VAPP.

Polio facts: Polio is asymptomatic in about 70% of infections. Among the 30% with symptoms, paralysis occurs infrequently, with the overall rate of paralytic infections being 0.5% (rate varies by virus type with type 3 having the highest rate).¹ Why then was the world so afraid of polio? If every person in a U.S. birth cohort (about 3.7 million) was unvaccinated and became infected with poliovirus, more than 18,000 would get paralytic polio and almost 1,300 would die. Of note, adults have a higher chance of paralytic polio after infection than children.

Concerns in 2022: Persons vaccinated with at least three doses of either IPV or OPV have historically been protected from paralytic polio (99% protection). But are we sure that the United States remains protected against polio after 2 decades of IPV being the only vaccine? Polio could be reintroduced at any time to the United States from countries with reported cases that likely arose because of low vaccination rates related to war, famine, or political upheavals (Malawi, Mozambique, Nigeria, Pakistan, and Afghanistan).² The proof? The recent confirmed New York case.

International efforts resulted in global eradication of two polio wild-types viruses (type 2 in 2015 and type 3 in 2019). Nevertheless, vaccine-derived, virulent polio virus (VDPV) type 2 and VDPV-3 still circulate in some areas, particularly Africa (VDPV-2) and Israel (VDPV-3). The above-mentioned U.S. case is an unvaccinated adult traveler who went to an area where VDPV-2 circulates and developed disease after returning home.³ So, it was not an indigenous reappearance in the United States and it was not a breakthrough case in a vaccinated person. But it is sobering to realize that all who are unvaccinated remain at risk for paralytic polio in 2022, particularly because vaccination rates declined nearly everywhere during the initial COVID-19 pandemic. We are still catching up, with vaccination rates under 50% in some ZIP codes.⁴

Are VDPVs circulating in some parts of the United States? Interestingly, wastewater surveillance programs may be the most economical and practical way to perform polio surveillance. Such a program detected polio virus in London wastewater in June 2022.⁵ New York has recently detected polio in wastewater during testing begun because of the recent case.⁶

Good news: For paralytic polio, seropositivity at any titer indicates protection, so U.S. serosurveillance data would also be informative. How durable is polio protection in the IPV era? Available data suggest that even though we have used only IPV these past 20 years, seropositivity rates among vaccinees with at least three doses of either IPV or OPV should persist for decades and likely for life. Even before polio became a concern this year, the Centers for Disease Control and Prevention, being proactive, wanted to ensure that the enhanced IPV was producing durable immunity and that persons of all ages remained seropositive to the three polio virus types over 10 years after discontinuing OPV use in 2012.

The CDC collaborated with investigators in Kansas City, Mo., to evaluate titers and seropositivity to all three types in a 2- to 85-year-old otherwise healthy cohort with demographics that mirrored the 2010 census for the Kansas City region, which in turn mirrored the national 2021 census data.⁷ There were approximately 100 persons in each age cohort, with 200 below age 11 years (the cohort that had received only IPV). Serology was performed at the CDC.

Overall seropositivity rates were high, but lower for type 3 (83.3%) and type 2 (90.7%) than type 1 (94.4%). Of note, most of those seronegative for one or more types were among 2- to 3-year-olds who had not completed their full IPV series, with most seronegative results being against polio types 1 and 3. Further, five, who were confirmed as having received no polio vaccine, were seronegative for all three types. Two with no available vaccine records (over 18 years old) were also seronegative for all three types.

So, regardless of the era in which one got polio vaccine, vaccine protection appears to persist indefinitely after three doses. Even 80-year-olds were still seropositive if they had three doses. We can confidently reassure our patients that the vaccine still works; the persons who need to fear polio in 2022 are those who are not vaccinated or have had fewer than three doses, particularly if they travel to areas of persistent polio. Wild type 1 virus persists in a few countries as does VDPV type 2 and VDPV type 3. Importantly, wild type 2 and wild type 3 (with the lowest seropositivity in 2012 study) have been eliminated globally so the only circulating type 2 and type 3 polio virus is VDPV in a few countries. Travel to these countries warrants review of polio vaccine records and CDC or WHO current recommendations for travelers to those countries.
 

Dr. Harrison is a professor, University of Missouri Kansas City School of Medicine, department of medicine, infectious diseases section, Kansas City. Email him at pdnews@mdedge.com.

References

1. Poliomyelitis. World Health Organization fact sheet, 2022 Jul 4..

2. Franco-Paredes C et al. Lancet Infect Dis. 2022 Aug 16. doi: 10.1016/S1473-3099(22)00548-5.

3. Link-Gelles R et al. MMWR Morb Mortal Wkly Rep. 2022 Aug 19;71(33):1065-8.

4. “Polio vaccination rate for 2-year-olds is as low as 37% in parts of N.Y. county where paralysis case was found,” NBC News, Erika Edwards, 2022 Aug 16. 5. Vaccine-derived poliovirus type 2 (VDPV2) detected in environmental samples in London. Polioeradication.org. 2022 Jun 22.

6. “NYSDOH and NYCDOHMH wastewater monitoring identifies polio in New York City and urges unvaccinated New Yorkers to get vaccinated now,” nyc.gov. 2022 Aug 12.

7. Wallace GS et al. Hum Vaccin Immunother. 2017;13(4):776-83.

Who would have thought we would need to refresh our knowledge on polio virus in 2022? Fate seems cruel to add this concern on the heels of SARS-CoV-2, monkeypox, abnormal seasons for RSV, acute flaccid myelitis (AFM) linked to enteroviruses, and a summer of parechovirus causing infant meningitis. But confirmation that indeed an adult had polio with paralytic disease raises concerns among public health groups and ordinary citizens alike, particularly those who remember polio in its heyday.

History: In the summer of 1952, polio was among the most feared diseases on the planet. Families were advised to not allow children to congregate in groups or use public swimming pools; little league baseball games were being canceled and there was talk of not opening schools for the fall. Every parent’s nightmare seemed to be the nonspecific febrile summer illness that led to paralytic sequelae. TV news included videos of the iron lung wards in hospitals across the country. Medical providers felt powerless, only able to give nonspecific preventive advice. There was no specific antiviral (there still isn’t) and vaccines seemed a long way off.

Then came the news that Dr. Jonas Salk’s group had gotten an inactivated polio vaccine (IPV) approved for general use in 1955. Families were excited to have their children vaccinated. Paralytic polio cases dropped like a rock from approximately 22,000/year in 1952 to approximately 2,200 in 1956. A surge to near 6,000 cases in 1959 led to Dr. Albert Sabin’s oral polio vaccine (OPV), which supplanted IPV in 1961. OPV had the advantages of: 1) Inducing mucosal as well as serum antibodies, 2) more durable responses, and 3) immunity in unvaccinated persons exposed to vaccine virus that had been shed in stools into wastewater and rivers.

By 1964, polio had nearly disappeared. The last wild-type indigenous U.S. case was in 1979. By 1994, all the Americas were declared polio free. Because the only U.S. paralytic polio cases thereafter were foreign imports or were associated with oral vaccine strains (so-called vaccine-associated paralytic polio [VAPP]), OPV was replaced by an enhanced IPV in 2000 to prevent further VAPP.

Polio facts: Polio is asymptomatic in about 70% of infections. Among the 30% with symptoms, paralysis occurs infrequently, with the overall rate of paralytic infections being 0.5% (rate varies by virus type with type 3 having the highest rate).¹ Why then was the world so afraid of polio? If every person in a U.S. birth cohort (about 3.7 million) was unvaccinated and became infected with poliovirus, more than 18,000 would get paralytic polio and almost 1,300 would die. Of note, adults have a higher chance of paralytic polio after infection than children.

Concerns in 2022: Persons vaccinated with at least three doses of either IPV or OPV have historically been protected from paralytic polio (99% protection). But are we sure that the United States remains protected against polio after 2 decades of IPV being the only vaccine? Polio could be reintroduced at any time to the United States from countries with reported cases that likely arose because of low vaccination rates related to war, famine, or political upheavals (Malawi, Mozambique, Nigeria, Pakistan, and Afghanistan).² The proof? The recent confirmed New York case.

International efforts resulted in global eradication of two polio wild-types viruses (type 2 in 2015 and type 3 in 2019). Nevertheless, vaccine-derived, virulent polio virus (VDPV) type 2 and VDPV-3 still circulate in some areas, particularly Africa (VDPV-2) and Israel (VDPV-3). The above-mentioned U.S. case is an unvaccinated adult traveler who went to an area where VDPV-2 circulates and developed disease after returning home.³ So, it was not an indigenous reappearance in the United States and it was not a breakthrough case in a vaccinated person. But it is sobering to realize that all who are unvaccinated remain at risk for paralytic polio in 2022, particularly because vaccination rates declined nearly everywhere during the initial COVID-19 pandemic. We are still catching up, with vaccination rates under 50% in some ZIP codes.⁴

Are VDPVs circulating in some parts of the United States? Interestingly, wastewater surveillance programs may be the most economical and practical way to perform polio surveillance. Such a program detected polio virus in London wastewater in June 2022.⁵ New York has recently detected polio in wastewater during testing begun because of the recent case.⁶

Good news: For paralytic polio, seropositivity at any titer indicates protection, so U.S. serosurveillance data would also be informative. How durable is polio protection in the IPV era? Available data suggest that even though we have used only IPV these past 20 years, seropositivity rates among vaccinees with at least three doses of either IPV or OPV should persist for decades and likely for life. Even before polio became a concern this year, the Centers for Disease Control and Prevention, being proactive, wanted to ensure that the enhanced IPV was producing durable immunity and that persons of all ages remained seropositive to the three polio virus types over 10 years after discontinuing OPV use in 2012.

The CDC collaborated with investigators in Kansas City, Mo., to evaluate titers and seropositivity to all three types in a 2- to 85-year-old otherwise healthy cohort with demographics that mirrored the 2010 census for the Kansas City region, which in turn mirrored the national 2021 census data.⁷ There were approximately 100 persons in each age cohort, with 200 below age 11 years (the cohort that had received only IPV). Serology was performed at the CDC.

Overall seropositivity rates were high, but lower for type 3 (83.3%) and type 2 (90.7%) than type 1 (94.4%). Of note, most of those seronegative for one or more types were among 2- to 3-year-olds who had not completed their full IPV series, with most seronegative results being against polio types 1 and 3. Further, five, who were confirmed as having received no polio vaccine, were seronegative for all three types. Two with no available vaccine records (over 18 years old) were also seronegative for all three types.

So, regardless of the era in which one got polio vaccine, vaccine protection appears to persist indefinitely after three doses. Even 80-year-olds were still seropositive if they had three doses. We can confidently reassure our patients that the vaccine still works; the persons who need to fear polio in 2022 are those who are not vaccinated or have had fewer than three doses, particularly if they travel to areas of persistent polio. Wild type 1 virus persists in a few countries as does VDPV type 2 and VDPV type 3. Importantly, wild type 2 and wild type 3 (with the lowest seropositivity in 2012 study) have been eliminated globally so the only circulating type 2 and type 3 polio virus is VDPV in a few countries. Travel to these countries warrants review of polio vaccine records and CDC or WHO current recommendations for travelers to those countries.
 

Dr. Harrison is a professor, University of Missouri Kansas City School of Medicine, department of medicine, infectious diseases section, Kansas City. Email him at pdnews@mdedge.com.

References

1. Poliomyelitis. World Health Organization fact sheet, 2022 Jul 4..

2. Franco-Paredes C et al. Lancet Infect Dis. 2022 Aug 16. doi: 10.1016/S1473-3099(22)00548-5.

3. Link-Gelles R et al. MMWR Morb Mortal Wkly Rep. 2022 Aug 19;71(33):1065-8.

4. “Polio vaccination rate for 2-year-olds is as low as 37% in parts of N.Y. county where paralysis case was found,” NBC News, Erika Edwards, 2022 Aug 16. 5. Vaccine-derived poliovirus type 2 (VDPV2) detected in environmental samples in London. Polioeradication.org. 2022 Jun 22.

6. “NYSDOH and NYCDOHMH wastewater monitoring identifies polio in New York City and urges unvaccinated New Yorkers to get vaccinated now,” nyc.gov. 2022 Aug 12.

7. Wallace GS et al. Hum Vaccin Immunother. 2017;13(4):776-83.

From the T1D Exchange, Boston, MA (Ann Mungmode, Nicole Rioles, Jesse Cases, Dr. Ebekozien); The Leona M. and Harry B. Hemsley Charitable Trust, New York, NY (Laurel Koester); and the University of Mississippi School of Population Health, Jackson, MS (Dr. Ebekozien).

Abstract

There have been remarkable innovations in diabetes management since the start of the COVID-19 pandemic, but these groundbreaking innovations are drawing limited focus as the field focuses on the adverse impact of the pandemic on patients with diabetes. This article reviews select population health innovations in diabetes management that have become available over the past 2 years of the COVID-19 pandemic from the perspective of the T1D Exchange Quality Improvement Collaborative, a learning health network that focuses on improving care and outcomes for individuals with type 1 diabetes (T1D). Such innovations include expanded telemedicine access, collection of real-world data, machine learning and artificial intelligence, and new diabetes medications and devices. In addition, multiple innovative studies have been undertaken to explore contributors to health inequities in diabetes, and advocacy efforts for specific populations have been successful. Looking to the future, work is required to explore additional health equity successes that do not further exacerbate inequities and to look for additional innovative ways to engage people with T1D in their health care through conversations on social determinants of health and societal structures.

Keywords: type 1 diabetes, learning health network, continuous glucose monitoring, health equity

One in 10 people in the United States has diabetes.¹ Diabetes is the nation’s second leading cause of death, costing the US health system more than $300 billion annually.² The COVID-19 pandemic presented additional health burdens for people living with diabetes. For example, preexisting diabetes was identified as a risk factor for COVID-19–associated morbidity and mortality.^3,4 Over the past 2 years, there have been remarkable innovations in diabetes management, including stem cell therapy and new medication options. Additionally, improved technology solutions have aided in diabetes management through continuous glucose monitors (CGM), smart insulin pens, advanced hybrid closed-loop systems, and continuous subcutaneous insulin injections.^5,6 Unfortunately, these groundbreaking innovations are drawing limited focus, as the field is rightfully focused on the adverse impact of the pandemic on patients with diabetes.

Learning health networks like the T1D Exchange Quality Improvement Collaborative (T1DX-QI) have implemented some of these innovative solutions to improve care for people with diabetes.⁷ T1DX-QI has more than 50 data-sharing endocrinology centers that care for over 75,000 people with diabetes across the United States (Figure 1). Centers participating in the T1DX-QI use quality improvement (QI) and implementation science methods to quickly translate research into evidence-based clinical practice. T1DX-QI leads diabetes population health and health system research and supports widespread transferability across health care organizations through regular collaborative calls, conferences, and case study documentation.⁸

In this review, we summarize impactful population health innovations in diabetes management that have become available over the past 2 years of the COVID-19 pandemic from the perspective of T1DX-QI (see Figure 2 for relevant definitions). This review is limited in scope and is not meant to be an exhaustive list of innovations. The review also reflects significant changes from the perspective of academic diabetes centers, which may not apply to rural or primary care diabetes practices.

The first (A.M.), second (H.H.), and senior (O.E.) authors conducted a scoping review of published literature using terms related to diabetes, population health, and innovation on PubMed Central and Google Scholar for the period March 2020 to June 2022. To complement the review, A.M. and O.E. also reviewed abstracts from presentations at major international diabetes conferences, including the American Diabetes Association (ADA), the International Society for Pediatric and Adolescent Diabetes (ISPAD), the T1DX-QI Learning Session Conference, and the Advanced Technologies & Treatments for Diabetes (ATTD) 2020 to 2022 conferences.^9-14 The authors also searched FDA.gov and ClinicalTrials.gov for relevant insights. A.M. and O.E. sorted the reviewed literature into major themes (Figure 3) from the population health improvement perspective of the T1DX-QI.

Population Health Innovations in Diabetes Management

Expansion of Telemedicine Access

Telemedicine is cost-effective for patients with diabetes,¹⁵ including those with complex cases.¹⁶ Before the COVID-19 pandemic, telemedicine and virtual care were rare in diabetes management. However, the pandemic offered a new opportunity to expand the practice of telemedicine in diabetes management. A study from the T1DX-QI showed that telemedicine visits grew from comprising <1% of visits pre-pandemic (December 2019) to 95.2% during the pandemic (August 2020).¹⁷ Additional studies, like those conducted by Phillip et al,¹⁸ confirmed the noninferiority of telemedicine practice for patients with diabetes.Telemedicine was also found to be an effective strategy to educate patients on the use of diabetes technologies.¹⁹

Real-World Data and Disease Surveillance

As the COVID-19 pandemic exacerbated outcomes for people with type 1 diabetes (T1D), a need arose to understand the immediate effects of the pandemic on people with T1D through real-world data and disease surveillance. In April 2020, the T1DX-QI initiated a multicenter surveillance study to collect data and analyze the impact of COVID-19 on people with T1D. The existing health collaborative served as a springboard for robust surveillance study, documenting numerous works on the effects of COVID-19.^3,4,20-28 Other investigators also embraced the power of real-world surveillance and real-world data.^29,30

Big Data, Machine Learning, and Artificial Intelligence

The past 2 years have seen a shift toward embracing the incredible opportunity to tap the large volume of data generated from routine care for practical insights.³¹ In particular, researchers have demonstrated the widespread application of machine learning and artificial intelligence to improve diabetes management.³² The T1DX-QI also harnessed the growing power of big data by expanding the functionality of innovative benchmarking software. The T1DX QI Portal uses electronic medical record data of diabetes patients for clinic-to-clinic benchmarking and data analysis, using business intelligence solutions.³³

Health Equity

While inequities across various health outcomes have been well documented for years,³⁴ the COVID-19 pandemic further exaggerated racial/ethnic health inequities in T1D.^23,35 In response, several organizations have outlined specific strategies to address these health inequities. Emboldened by the pandemic, the T1DX-QI announced a multipronged approach to address health inequities among patients with T1D through the Health Equity Advancement Lab (HEAL).³⁶ One of HEAL’s main components is using real-world data to champion population-level insights and demonstrate progress in QI efforts.

Multiple innovative studies have been undertaken to explore contributors to health inequities in diabetes, and these studies are expanding our understanding of the chasm.³⁷ There have also been innovative solutions to addressing these inequities, with multiple studies published over the past 2 years.³⁸ A source of inequity among patients with T1D is the lack of representation of racial/ethnic minorities with T1D in clinical trials.³⁹ The T1DX-QI suggests that the equity-adapted framework for QI can be applied by research leaders to support trial diversity and representation, ensuring future device innovations are meaningful for all people with T1D.⁴⁰

Diabetes Devices

Glucose monitoring and insulin therapy are vital tools to support individuals living with T1D, and devices such as CGM and insulin pumps have become the standard of care for diabetes management (Table).⁴¹ Innovations in diabetes technology and device access are imperative for a chronic disease with no cure.

The COVID-19 pandemic created an opportunity to increase access to diabetes devices in inpatient settings. In 2020, the US Food and Drug Administration expanded the use of CGM to support remote monitoring of patients in inpatient hospital settings, simultaneously supporting the glucose monitoring needs of patients with T1D and reducing COVID-19 transmission through reduced patient-clinician contact.⁴² This effort has been expanded and will continue in 2022 and beyond,⁴³ and aligns with the growing consensus that supports patients wearing both CGMs and insulin pumps in ambulatory settings to improve patient health outcomes.⁴⁴

Since 2020, innovations in diabetes technology have improved and increased the variety of options available to people with T1D and made them easier to use (Table). New, advanced hybrid closed-loop systems have progressed to offer Bluetooth features, including automatic software upgrades, tubeless systems, and the ability to allow parents to use their smartphones to bolus for children.^45-47 The next big step in insulin delivery innovation is the release of functioning, fully closed loop systems, of which several are currently in clinical trials.⁴⁸ These systems support reduced hypoglycemia and improved time in range.⁴⁹

Additional innovations in insulin delivery have improved the user experience and expanded therapeutic options, including a variety of smart insulin pens complete with dosing logs^50,51 and even a patch to deliver insulin without the burden of injections.⁵² As barriers to diabetes technology persist,⁵³ innovations in alternate insulin delivery provide people with T1D more options to align with their personal access and technology preferences.

Innovations in CGM address cited barriers to their use, including size or overall wear.^53-55 CGMs released in the past few years are smaller in physical size, have longer durations of time between changings, are more accurate, and do not require calibrations for accuracy.

New Diabetes Medications

Many new medications and therapeutic advances have become available in the past 2 years.⁵⁶ Additionally, more medications are being tested as adjunct therapies to support glycemic management in patients with T1D, including metformin, sodium-glucose cotransporter 1 and 2 inhibitors, pramlintide, glucagon-like polypeptide-1 analogs, and glucagon receptor agonists.⁵⁷ Other recent advances include stem cell replacement therapy for patients with T1D.⁵⁸ The ultra-long-acting biosimilar insulins are one medical innovation that has been stalled, rather than propelled, during the COVID-19 pandemic.⁵⁹

Diabetes Policy Advocacy

People with T1D require insulin to survive. The cost of insulin has increased in recent years, with some studies citing a 64% to 100% increase in the past decade.^60,61 In fact, 1 in 4 insulin users report that cost has impacted their insulin use, including rationing their insulin.⁶² Lockdowns during the COVID-19 pandemic stressed US families financially, increasing the urgency for insulin cost caps.

Although the COVID-19 pandemic halted national conversations on drug financing,⁶³ advocacy efforts have succeeded for specific populations. The new Medicare Part D Senior Savings Model will cap the cost of insulin at $35 for a 30-day supply,⁶⁴ and 20 states passed legislation capping insulin pricing.⁶² Efforts to codify national cost caps are under debate, including the passage of the Affordable Insulin Now Act, which passed the House in March 2022 and is currently under review in the Senate.⁶⁵

Funders and industry partners play a crucial role in leading and supporting innovations that improve the lives of people with T1D and reduce society’s costs of living with the disease. Data infrastructure is critical to supporting population health. While building the data infrastructure to support population health is both time- and resource-intensive, private foundations such as Helmsley are uniquely positioned—and have a responsibility—to take large, informed risks to help reach all communities with T1D.

The T1DX-QI is the largest source of population health data on T1D in the United States and is becoming the premiere data authority on its incidence, prevalence, and outcomes. The T1DX-QI enables a robust understanding of T1D-related health trends at the population level, as well as trends among clinics and providers. Pilot centers in the T1DX-QI have reported reductions in patients’ A1c and acute diabetes-related events, as well as improvements in device usage and depression screening. The ability to capture changes speaks to the promise and power of these data to demonstrate the clinical impact of QI interventions and to support the spread of best practices and learnings across health systems.

Additional philanthropic efforts have supported innovation in the last 2 years. For example, the JDRF, a nonprofit philanthropic equity firm, has supported efforts in developing artificial pancreas systems and cell therapies currently in clinical trials like teplizumab, a drug that has demonstrated delayed onset of T1D through JDRF’s T1D Fund.⁶⁶ Industry partners also have an opportunity for significant influence in this area, as they continue to fund meaningful projects to advance care for people with T1D.⁶⁷

Conclusion

We are optimistic that the innovations summarized here describe a shift in the tide of equitable T1D outcomes; however, future work is required to explore additional health equity successes that do not further exacerbate inequities. We also see further opportunities for innovative ways to engage people with T1D in their health care through conversations on social determinants of health and societal structures.

Corresponding author: Ann Mungmode, MPH, T1D Exchange, 11 Avenue de Lafayette, Boston, MA 02111; Email: amungmode@t1dexchange.org

Disclosures: Dr. Ebekozien serve(d) as a director, officer, partner, employee, advisor, consultant, or trustee for the Medtronic Advisory Board and received research grants from Medtronic Diabetes, Eli Lilly, and Dexcom.

Funding: The T1DX-QI is funded by The Leona M. and Harry B. Hemsley Charitable Trust.

From the T1D Exchange, Boston, MA (Ann Mungmode, Nicole Rioles, Jesse Cases, Dr. Ebekozien); The Leona M. and Harry B. Hemsley Charitable Trust, New York, NY (Laurel Koester); and the University of Mississippi School of Population Health, Jackson, MS (Dr. Ebekozien).

Abstract

There have been remarkable innovations in diabetes management since the start of the COVID-19 pandemic, but these groundbreaking innovations are drawing limited focus as the field focuses on the adverse impact of the pandemic on patients with diabetes. This article reviews select population health innovations in diabetes management that have become available over the past 2 years of the COVID-19 pandemic from the perspective of the T1D Exchange Quality Improvement Collaborative, a learning health network that focuses on improving care and outcomes for individuals with type 1 diabetes (T1D). Such innovations include expanded telemedicine access, collection of real-world data, machine learning and artificial intelligence, and new diabetes medications and devices. In addition, multiple innovative studies have been undertaken to explore contributors to health inequities in diabetes, and advocacy efforts for specific populations have been successful. Looking to the future, work is required to explore additional health equity successes that do not further exacerbate inequities and to look for additional innovative ways to engage people with T1D in their health care through conversations on social determinants of health and societal structures.

Keywords: type 1 diabetes, learning health network, continuous glucose monitoring, health equity

One in 10 people in the United States has diabetes.¹ Diabetes is the nation’s second leading cause of death, costing the US health system more than $300 billion annually.² The COVID-19 pandemic presented additional health burdens for people living with diabetes. For example, preexisting diabetes was identified as a risk factor for COVID-19–associated morbidity and mortality.^3,4 Over the past 2 years, there have been remarkable innovations in diabetes management, including stem cell therapy and new medication options. Additionally, improved technology solutions have aided in diabetes management through continuous glucose monitors (CGM), smart insulin pens, advanced hybrid closed-loop systems, and continuous subcutaneous insulin injections.^5,6 Unfortunately, these groundbreaking innovations are drawing limited focus, as the field is rightfully focused on the adverse impact of the pandemic on patients with diabetes.

Learning health networks like the T1D Exchange Quality Improvement Collaborative (T1DX-QI) have implemented some of these innovative solutions to improve care for people with diabetes.⁷ T1DX-QI has more than 50 data-sharing endocrinology centers that care for over 75,000 people with diabetes across the United States (Figure 1). Centers participating in the T1DX-QI use quality improvement (QI) and implementation science methods to quickly translate research into evidence-based clinical practice. T1DX-QI leads diabetes population health and health system research and supports widespread transferability across health care organizations through regular collaborative calls, conferences, and case study documentation.⁸

In this review, we summarize impactful population health innovations in diabetes management that have become available over the past 2 years of the COVID-19 pandemic from the perspective of T1DX-QI (see Figure 2 for relevant definitions). This review is limited in scope and is not meant to be an exhaustive list of innovations. The review also reflects significant changes from the perspective of academic diabetes centers, which may not apply to rural or primary care diabetes practices.

The first (A.M.), second (H.H.), and senior (O.E.) authors conducted a scoping review of published literature using terms related to diabetes, population health, and innovation on PubMed Central and Google Scholar for the period March 2020 to June 2022. To complement the review, A.M. and O.E. also reviewed abstracts from presentations at major international diabetes conferences, including the American Diabetes Association (ADA), the International Society for Pediatric and Adolescent Diabetes (ISPAD), the T1DX-QI Learning Session Conference, and the Advanced Technologies & Treatments for Diabetes (ATTD) 2020 to 2022 conferences.^9-14 The authors also searched FDA.gov and ClinicalTrials.gov for relevant insights. A.M. and O.E. sorted the reviewed literature into major themes (Figure 3) from the population health improvement perspective of the T1DX-QI.

Population Health Innovations in Diabetes Management

Expansion of Telemedicine Access

Telemedicine is cost-effective for patients with diabetes,¹⁵ including those with complex cases.¹⁶ Before the COVID-19 pandemic, telemedicine and virtual care were rare in diabetes management. However, the pandemic offered a new opportunity to expand the practice of telemedicine in diabetes management. A study from the T1DX-QI showed that telemedicine visits grew from comprising <1% of visits pre-pandemic (December 2019) to 95.2% during the pandemic (August 2020).¹⁷ Additional studies, like those conducted by Phillip et al,¹⁸ confirmed the noninferiority of telemedicine practice for patients with diabetes.Telemedicine was also found to be an effective strategy to educate patients on the use of diabetes technologies.¹⁹

Real-World Data and Disease Surveillance

As the COVID-19 pandemic exacerbated outcomes for people with type 1 diabetes (T1D), a need arose to understand the immediate effects of the pandemic on people with T1D through real-world data and disease surveillance. In April 2020, the T1DX-QI initiated a multicenter surveillance study to collect data and analyze the impact of COVID-19 on people with T1D. The existing health collaborative served as a springboard for robust surveillance study, documenting numerous works on the effects of COVID-19.^3,4,20-28 Other investigators also embraced the power of real-world surveillance and real-world data.^29,30

Big Data, Machine Learning, and Artificial Intelligence

The past 2 years have seen a shift toward embracing the incredible opportunity to tap the large volume of data generated from routine care for practical insights.³¹ In particular, researchers have demonstrated the widespread application of machine learning and artificial intelligence to improve diabetes management.³² The T1DX-QI also harnessed the growing power of big data by expanding the functionality of innovative benchmarking software. The T1DX QI Portal uses electronic medical record data of diabetes patients for clinic-to-clinic benchmarking and data analysis, using business intelligence solutions.³³

Health Equity

While inequities across various health outcomes have been well documented for years,³⁴ the COVID-19 pandemic further exaggerated racial/ethnic health inequities in T1D.^23,35 In response, several organizations have outlined specific strategies to address these health inequities. Emboldened by the pandemic, the T1DX-QI announced a multipronged approach to address health inequities among patients with T1D through the Health Equity Advancement Lab (HEAL).³⁶ One of HEAL’s main components is using real-world data to champion population-level insights and demonstrate progress in QI efforts.

Multiple innovative studies have been undertaken to explore contributors to health inequities in diabetes, and these studies are expanding our understanding of the chasm.³⁷ There have also been innovative solutions to addressing these inequities, with multiple studies published over the past 2 years.³⁸ A source of inequity among patients with T1D is the lack of representation of racial/ethnic minorities with T1D in clinical trials.³⁹ The T1DX-QI suggests that the equity-adapted framework for QI can be applied by research leaders to support trial diversity and representation, ensuring future device innovations are meaningful for all people with T1D.⁴⁰

Diabetes Devices

Glucose monitoring and insulin therapy are vital tools to support individuals living with T1D, and devices such as CGM and insulin pumps have become the standard of care for diabetes management (Table).⁴¹ Innovations in diabetes technology and device access are imperative for a chronic disease with no cure.

The COVID-19 pandemic created an opportunity to increase access to diabetes devices in inpatient settings. In 2020, the US Food and Drug Administration expanded the use of CGM to support remote monitoring of patients in inpatient hospital settings, simultaneously supporting the glucose monitoring needs of patients with T1D and reducing COVID-19 transmission through reduced patient-clinician contact.⁴² This effort has been expanded and will continue in 2022 and beyond,⁴³ and aligns with the growing consensus that supports patients wearing both CGMs and insulin pumps in ambulatory settings to improve patient health outcomes.⁴⁴

Since 2020, innovations in diabetes technology have improved and increased the variety of options available to people with T1D and made them easier to use (Table). New, advanced hybrid closed-loop systems have progressed to offer Bluetooth features, including automatic software upgrades, tubeless systems, and the ability to allow parents to use their smartphones to bolus for children.^45-47 The next big step in insulin delivery innovation is the release of functioning, fully closed loop systems, of which several are currently in clinical trials.⁴⁸ These systems support reduced hypoglycemia and improved time in range.⁴⁹

Additional innovations in insulin delivery have improved the user experience and expanded therapeutic options, including a variety of smart insulin pens complete with dosing logs^50,51 and even a patch to deliver insulin without the burden of injections.⁵² As barriers to diabetes technology persist,⁵³ innovations in alternate insulin delivery provide people with T1D more options to align with their personal access and technology preferences.

Innovations in CGM address cited barriers to their use, including size or overall wear.^53-55 CGMs released in the past few years are smaller in physical size, have longer durations of time between changings, are more accurate, and do not require calibrations for accuracy.

New Diabetes Medications

Many new medications and therapeutic advances have become available in the past 2 years.⁵⁶ Additionally, more medications are being tested as adjunct therapies to support glycemic management in patients with T1D, including metformin, sodium-glucose cotransporter 1 and 2 inhibitors, pramlintide, glucagon-like polypeptide-1 analogs, and glucagon receptor agonists.⁵⁷ Other recent advances include stem cell replacement therapy for patients with T1D.⁵⁸ The ultra-long-acting biosimilar insulins are one medical innovation that has been stalled, rather than propelled, during the COVID-19 pandemic.⁵⁹

Diabetes Policy Advocacy

People with T1D require insulin to survive. The cost of insulin has increased in recent years, with some studies citing a 64% to 100% increase in the past decade.^60,61 In fact, 1 in 4 insulin users report that cost has impacted their insulin use, including rationing their insulin.⁶² Lockdowns during the COVID-19 pandemic stressed US families financially, increasing the urgency for insulin cost caps.

Although the COVID-19 pandemic halted national conversations on drug financing,⁶³ advocacy efforts have succeeded for specific populations. The new Medicare Part D Senior Savings Model will cap the cost of insulin at $35 for a 30-day supply,⁶⁴ and 20 states passed legislation capping insulin pricing.⁶² Efforts to codify national cost caps are under debate, including the passage of the Affordable Insulin Now Act, which passed the House in March 2022 and is currently under review in the Senate.⁶⁵

Funders and industry partners play a crucial role in leading and supporting innovations that improve the lives of people with T1D and reduce society’s costs of living with the disease. Data infrastructure is critical to supporting population health. While building the data infrastructure to support population health is both time- and resource-intensive, private foundations such as Helmsley are uniquely positioned—and have a responsibility—to take large, informed risks to help reach all communities with T1D.

The T1DX-QI is the largest source of population health data on T1D in the United States and is becoming the premiere data authority on its incidence, prevalence, and outcomes. The T1DX-QI enables a robust understanding of T1D-related health trends at the population level, as well as trends among clinics and providers. Pilot centers in the T1DX-QI have reported reductions in patients’ A1c and acute diabetes-related events, as well as improvements in device usage and depression screening. The ability to capture changes speaks to the promise and power of these data to demonstrate the clinical impact of QI interventions and to support the spread of best practices and learnings across health systems.

Additional philanthropic efforts have supported innovation in the last 2 years. For example, the JDRF, a nonprofit philanthropic equity firm, has supported efforts in developing artificial pancreas systems and cell therapies currently in clinical trials like teplizumab, a drug that has demonstrated delayed onset of T1D through JDRF’s T1D Fund.⁶⁶ Industry partners also have an opportunity for significant influence in this area, as they continue to fund meaningful projects to advance care for people with T1D.⁶⁷

Conclusion

We are optimistic that the innovations summarized here describe a shift in the tide of equitable T1D outcomes; however, future work is required to explore additional health equity successes that do not further exacerbate inequities. We also see further opportunities for innovative ways to engage people with T1D in their health care through conversations on social determinants of health and societal structures.

Corresponding author: Ann Mungmode, MPH, T1D Exchange, 11 Avenue de Lafayette, Boston, MA 02111; Email: amungmode@t1dexchange.org

Disclosures: Dr. Ebekozien serve(d) as a director, officer, partner, employee, advisor, consultant, or trustee for the Medtronic Advisory Board and received research grants from Medtronic Diabetes, Eli Lilly, and Dexcom.

Funding: The T1DX-QI is funded by The Leona M. and Harry B. Hemsley Charitable Trust.

From the T1D Exchange, Boston, MA (Ann Mungmode, Nicole Rioles, Jesse Cases, Dr. Ebekozien); The Leona M. and Harry B. Hemsley Charitable Trust, New York, NY (Laurel Koester); and the University of Mississippi School of Population Health, Jackson, MS (Dr. Ebekozien).

Abstract

There have been remarkable innovations in diabetes management since the start of the COVID-19 pandemic, but these groundbreaking innovations are drawing limited focus as the field focuses on the adverse impact of the pandemic on patients with diabetes. This article reviews select population health innovations in diabetes management that have become available over the past 2 years of the COVID-19 pandemic from the perspective of the T1D Exchange Quality Improvement Collaborative, a learning health network that focuses on improving care and outcomes for individuals with type 1 diabetes (T1D). Such innovations include expanded telemedicine access, collection of real-world data, machine learning and artificial intelligence, and new diabetes medications and devices. In addition, multiple innovative studies have been undertaken to explore contributors to health inequities in diabetes, and advocacy efforts for specific populations have been successful. Looking to the future, work is required to explore additional health equity successes that do not further exacerbate inequities and to look for additional innovative ways to engage people with T1D in their health care through conversations on social determinants of health and societal structures.

Keywords: type 1 diabetes, learning health network, continuous glucose monitoring, health equity

One in 10 people in the United States has diabetes.¹ Diabetes is the nation’s second leading cause of death, costing the US health system more than $300 billion annually.² The COVID-19 pandemic presented additional health burdens for people living with diabetes. For example, preexisting diabetes was identified as a risk factor for COVID-19–associated morbidity and mortality.^3,4 Over the past 2 years, there have been remarkable innovations in diabetes management, including stem cell therapy and new medication options. Additionally, improved technology solutions have aided in diabetes management through continuous glucose monitors (CGM), smart insulin pens, advanced hybrid closed-loop systems, and continuous subcutaneous insulin injections.^5,6 Unfortunately, these groundbreaking innovations are drawing limited focus, as the field is rightfully focused on the adverse impact of the pandemic on patients with diabetes.

Learning health networks like the T1D Exchange Quality Improvement Collaborative (T1DX-QI) have implemented some of these innovative solutions to improve care for people with diabetes.⁷ T1DX-QI has more than 50 data-sharing endocrinology centers that care for over 75,000 people with diabetes across the United States (Figure 1). Centers participating in the T1DX-QI use quality improvement (QI) and implementation science methods to quickly translate research into evidence-based clinical practice. T1DX-QI leads diabetes population health and health system research and supports widespread transferability across health care organizations through regular collaborative calls, conferences, and case study documentation.⁸

In this review, we summarize impactful population health innovations in diabetes management that have become available over the past 2 years of the COVID-19 pandemic from the perspective of T1DX-QI (see Figure 2 for relevant definitions). This review is limited in scope and is not meant to be an exhaustive list of innovations. The review also reflects significant changes from the perspective of academic diabetes centers, which may not apply to rural or primary care diabetes practices.

The first (A.M.), second (H.H.), and senior (O.E.) authors conducted a scoping review of published literature using terms related to diabetes, population health, and innovation on PubMed Central and Google Scholar for the period March 2020 to June 2022. To complement the review, A.M. and O.E. also reviewed abstracts from presentations at major international diabetes conferences, including the American Diabetes Association (ADA), the International Society for Pediatric and Adolescent Diabetes (ISPAD), the T1DX-QI Learning Session Conference, and the Advanced Technologies & Treatments for Diabetes (ATTD) 2020 to 2022 conferences.^9-14 The authors also searched FDA.gov and ClinicalTrials.gov for relevant insights. A.M. and O.E. sorted the reviewed literature into major themes (Figure 3) from the population health improvement perspective of the T1DX-QI.

Population Health Innovations in Diabetes Management

Expansion of Telemedicine Access

Telemedicine is cost-effective for patients with diabetes,¹⁵ including those with complex cases.¹⁶ Before the COVID-19 pandemic, telemedicine and virtual care were rare in diabetes management. However, the pandemic offered a new opportunity to expand the practice of telemedicine in diabetes management. A study from the T1DX-QI showed that telemedicine visits grew from comprising <1% of visits pre-pandemic (December 2019) to 95.2% during the pandemic (August 2020).¹⁷ Additional studies, like those conducted by Phillip et al,¹⁸ confirmed the noninferiority of telemedicine practice for patients with diabetes.Telemedicine was also found to be an effective strategy to educate patients on the use of diabetes technologies.¹⁹

Real-World Data and Disease Surveillance

As the COVID-19 pandemic exacerbated outcomes for people with type 1 diabetes (T1D), a need arose to understand the immediate effects of the pandemic on people with T1D through real-world data and disease surveillance. In April 2020, the T1DX-QI initiated a multicenter surveillance study to collect data and analyze the impact of COVID-19 on people with T1D. The existing health collaborative served as a springboard for robust surveillance study, documenting numerous works on the effects of COVID-19.^3,4,20-28 Other investigators also embraced the power of real-world surveillance and real-world data.^29,30

Big Data, Machine Learning, and Artificial Intelligence

The past 2 years have seen a shift toward embracing the incredible opportunity to tap the large volume of data generated from routine care for practical insights.³¹ In particular, researchers have demonstrated the widespread application of machine learning and artificial intelligence to improve diabetes management.³² The T1DX-QI also harnessed the growing power of big data by expanding the functionality of innovative benchmarking software. The T1DX QI Portal uses electronic medical record data of diabetes patients for clinic-to-clinic benchmarking and data analysis, using business intelligence solutions.³³

Health Equity

While inequities across various health outcomes have been well documented for years,³⁴ the COVID-19 pandemic further exaggerated racial/ethnic health inequities in T1D.^23,35 In response, several organizations have outlined specific strategies to address these health inequities. Emboldened by the pandemic, the T1DX-QI announced a multipronged approach to address health inequities among patients with T1D through the Health Equity Advancement Lab (HEAL).³⁶ One of HEAL’s main components is using real-world data to champion population-level insights and demonstrate progress in QI efforts.

Multiple innovative studies have been undertaken to explore contributors to health inequities in diabetes, and these studies are expanding our understanding of the chasm.³⁷ There have also been innovative solutions to addressing these inequities, with multiple studies published over the past 2 years.³⁸ A source of inequity among patients with T1D is the lack of representation of racial/ethnic minorities with T1D in clinical trials.³⁹ The T1DX-QI suggests that the equity-adapted framework for QI can be applied by research leaders to support trial diversity and representation, ensuring future device innovations are meaningful for all people with T1D.⁴⁰

Diabetes Devices

Glucose monitoring and insulin therapy are vital tools to support individuals living with T1D, and devices such as CGM and insulin pumps have become the standard of care for diabetes management (Table).⁴¹ Innovations in diabetes technology and device access are imperative for a chronic disease with no cure.

The COVID-19 pandemic created an opportunity to increase access to diabetes devices in inpatient settings. In 2020, the US Food and Drug Administration expanded the use of CGM to support remote monitoring of patients in inpatient hospital settings, simultaneously supporting the glucose monitoring needs of patients with T1D and reducing COVID-19 transmission through reduced patient-clinician contact.⁴² This effort has been expanded and will continue in 2022 and beyond,⁴³ and aligns with the growing consensus that supports patients wearing both CGMs and insulin pumps in ambulatory settings to improve patient health outcomes.⁴⁴

Since 2020, innovations in diabetes technology have improved and increased the variety of options available to people with T1D and made them easier to use (Table). New, advanced hybrid closed-loop systems have progressed to offer Bluetooth features, including automatic software upgrades, tubeless systems, and the ability to allow parents to use their smartphones to bolus for children.^45-47 The next big step in insulin delivery innovation is the release of functioning, fully closed loop systems, of which several are currently in clinical trials.⁴⁸ These systems support reduced hypoglycemia and improved time in range.⁴⁹

Additional innovations in insulin delivery have improved the user experience and expanded therapeutic options, including a variety of smart insulin pens complete with dosing logs^50,51 and even a patch to deliver insulin without the burden of injections.⁵² As barriers to diabetes technology persist,⁵³ innovations in alternate insulin delivery provide people with T1D more options to align with their personal access and technology preferences.

Innovations in CGM address cited barriers to their use, including size or overall wear.^53-55 CGMs released in the past few years are smaller in physical size, have longer durations of time between changings, are more accurate, and do not require calibrations for accuracy.

New Diabetes Medications

Many new medications and therapeutic advances have become available in the past 2 years.⁵⁶ Additionally, more medications are being tested as adjunct therapies to support glycemic management in patients with T1D, including metformin, sodium-glucose cotransporter 1 and 2 inhibitors, pramlintide, glucagon-like polypeptide-1 analogs, and glucagon receptor agonists.⁵⁷ Other recent advances include stem cell replacement therapy for patients with T1D.⁵⁸ The ultra-long-acting biosimilar insulins are one medical innovation that has been stalled, rather than propelled, during the COVID-19 pandemic.⁵⁹

Diabetes Policy Advocacy

People with T1D require insulin to survive. The cost of insulin has increased in recent years, with some studies citing a 64% to 100% increase in the past decade.^60,61 In fact, 1 in 4 insulin users report that cost has impacted their insulin use, including rationing their insulin.⁶² Lockdowns during the COVID-19 pandemic stressed US families financially, increasing the urgency for insulin cost caps.

Although the COVID-19 pandemic halted national conversations on drug financing,⁶³ advocacy efforts have succeeded for specific populations. The new Medicare Part D Senior Savings Model will cap the cost of insulin at $35 for a 30-day supply,⁶⁴ and 20 states passed legislation capping insulin pricing.⁶² Efforts to codify national cost caps are under debate, including the passage of the Affordable Insulin Now Act, which passed the House in March 2022 and is currently under review in the Senate.⁶⁵

Funders and industry partners play a crucial role in leading and supporting innovations that improve the lives of people with T1D and reduce society’s costs of living with the disease. Data infrastructure is critical to supporting population health. While building the data infrastructure to support population health is both time- and resource-intensive, private foundations such as Helmsley are uniquely positioned—and have a responsibility—to take large, informed risks to help reach all communities with T1D.

The T1DX-QI is the largest source of population health data on T1D in the United States and is becoming the premiere data authority on its incidence, prevalence, and outcomes. The T1DX-QI enables a robust understanding of T1D-related health trends at the population level, as well as trends among clinics and providers. Pilot centers in the T1DX-QI have reported reductions in patients’ A1c and acute diabetes-related events, as well as improvements in device usage and depression screening. The ability to capture changes speaks to the promise and power of these data to demonstrate the clinical impact of QI interventions and to support the spread of best practices and learnings across health systems.

Additional philanthropic efforts have supported innovation in the last 2 years. For example, the JDRF, a nonprofit philanthropic equity firm, has supported efforts in developing artificial pancreas systems and cell therapies currently in clinical trials like teplizumab, a drug that has demonstrated delayed onset of T1D through JDRF’s T1D Fund.⁶⁶ Industry partners also have an opportunity for significant influence in this area, as they continue to fund meaningful projects to advance care for people with T1D.⁶⁷

Conclusion

We are optimistic that the innovations summarized here describe a shift in the tide of equitable T1D outcomes; however, future work is required to explore additional health equity successes that do not further exacerbate inequities. We also see further opportunities for innovative ways to engage people with T1D in their health care through conversations on social determinants of health and societal structures.

Corresponding author: Ann Mungmode, MPH, T1D Exchange, 11 Avenue de Lafayette, Boston, MA 02111; Email: amungmode@t1dexchange.org

Disclosures: Dr. Ebekozien serve(d) as a director, officer, partner, employee, advisor, consultant, or trustee for the Medtronic Advisory Board and received research grants from Medtronic Diabetes, Eli Lilly, and Dexcom.

Funding: The T1DX-QI is funded by The Leona M. and Harry B. Hemsley Charitable Trust.

After 15 years as a high school teacher at urban schools, I realized adults widely misunderstand that teenagers do not want to talk to them. In fact, most crave finding an adult they can trust and have serious conversations about issues like sex, drugs, and death. G was a sophomore who was going blind from a rare degenerative disease and one day sought my guidance about a sexual orgy he accidentally got in involved in. Was it wrong? Would God send him to hell? Why was he now so anxious after?

Because I was an openly gay teacher, students every semester would come out to me, asking what the “gay scene” was like, or how to deal with a homophobic family. Sometimes, students would seek counsel about an unplanned pregnancy, about abortion. In one instance, a student sought counsel about her violent thoughts, and eventually checked herself into a psychiatric ward. Five separate times, students in my class were murdered and I accompanied my classes through mourning.

Unlike many pediatricians, a teacher has a lot of time with these young adults: daily, sometimes over years. Students often admit they spend more time with their teachers than their parents. I can’t give you that time, but here are some general tips.
 

Attitude promoting trust

My guiding attitude toward teens was that they were my equals. I would “do unto them as I would have them do unto me.” Not less, but also not more – because sometimes “more” can cloak condescension. When I was a student, I trusted teachers who shared their fears and mistakes, not performing under a confessional spotlight, but to establish commonality, to flatten hierarchy. I also trusted those who could set boundaries and wield authority compassionately. Because sometimes I needed a firm hand. And so, as an adult I tried to give this to my students as well.

Although my students and I were equals, our situations are different. That is true with gender, race, and class, and it is also true with adults versus teens. The first step toward treating someone authentically as an equal when in a position of authority is to understand the unique stressors of their life. That means asking questions and listening to what they need.
 

Stressors in a teen’s life

A typical high school junior or senior goes to work 8-10 hours a day. Unpaid. They sit for hours at a small desk in a small room with sometimes 34 others. Most of the time they cannot eat or use their phone. If they need to pee, they need to ask permission. They have to ask permission to speak. And then when they go home, they sit at a small desk again for homework. They often do not even have their own room. They also have to ask for permission to buy something for themselves, for money, for a ride anywhere. Their values are often compromised so they won’t get kicked out of a house or a class. The life of a teen is not at all “carefree” but largely prescribed and with little control.

When I think about my youth and how little freedom, privacy, and control I had compared with now, it softens my attitude to even the rudest student. (Isn’t rudeness often a sign of resistance against an oppressive system?) But, some may say, these teens do not have to worry about bills. But if I think back honestly to my teen years, would I trade the responsibilities I have now for those supposed carefree years? Carefree is not how most teens describe their lives but a nostalgic rosy retrospection adults assign. Almost all teens I taught would rather work to gain some control over their lives. Which is why so many work 4-5 hours after school on top of homework, giving up their weekends, and binding themselves to a “carefree” 60- to 80-hour work week.
 

Talking about drugs, sex, and mental health

Drugs

It’s a good idea to first disarm teens of their fear of judgment or punishment, saying things like: “It’s normal to experiment with drugs, even hard ones.” The most successful, respected adults you see now have, so it’s not a reflection of who you are. Tell me what you’re worried about and it’ll be just between us.

After rapport is established, follow-up questions that elicit and affirm their feelings and thoughts can encourage more revelations: Do you think you have a problem? Why? How do you get your drugs and I’m curious only because finding that out can help us understand risks and solutions. What made you start? And keep on using?
 

Sex

Again, first disarm their fears: You can talk to me freely and confidently about sex: What you do, who you do it with, how you do it, and how often – I know that people are very different in their sexual interests and activities.

It is also good to set up clear boundaries. I had instances where students had romantic interest in me and would use these conversations as overtures. If you feel like your patient may be interested in you, then be explicit about boundaries: I’m a doctor who can point you to resources or offer treatments related to any sexual practice and its consequences, but that is all I am. Anything else is illegal and would end our patient-doctor relationship. (I would also immediately document the interaction and tell it to a witness.)

I never escalated incidences like this because I understood that most teens are naturally curious and often not taught about sexual boundaries, so I tried to make these encounters “teachable moments,” not punitive ones. Many teens are more aware of health consequences, like STDs or pregnancy, than psychological ones. So, it’s useful to ask: When you have sex outside your relationship, how does that make you feel? Does sex with multiple partners make you anxious or guilty afterwards? I like to use straightforward language and normalize taboo sexual practices with an even tone to allow teens to speak truthfully.
 

Suicide/depression

First, disarm and normalize: It is very common for people to have depression or thoughts of suicide. Most of the adults around you probably have and so have I (if that is true). Have you experienced this? Older teens often crave an intelligent open discussion about depression and suicide. If they look particularly distressed, I also tell them that I, and countless others, found strategies to deal with these thoughts. For most older teens, talking about causes of mental health issues and treatments is a breath of fresh air. This is especially true for teens from urban communities who have dealt precociously with death and violence, minority communities where mental health is often stigmatized, and young males whose machismo code can prevent them from acknowledging their feelings.

Some follow-up questions: Where do you think these thoughts come from? And if they don’t know: It’s perfectly normal for there to be no reason. The important thing is that they don’t last too long and that you know that. And if they do, then I can provide you resources and potential treatments.
 

Summary

Treating teens as equals by understanding their situation allows understanding and compassion for their stressors. This motivates an inquisitive and collaborative patient-centric approach that allows a sharing of sensitive topics like drugs, sex, and mental health.

Dr. Nguyen is a resident in psychiatry at the University of California, San Francisco.

*This story was updated on Nov. 3, 2022.

After 15 years as a high school teacher at urban schools, I realized adults widely misunderstand that teenagers do not want to talk to them. In fact, most crave finding an adult they can trust and have serious conversations about issues like sex, drugs, and death. G was a sophomore who was going blind from a rare degenerative disease and one day sought my guidance about a sexual orgy he accidentally got in involved in. Was it wrong? Would God send him to hell? Why was he now so anxious after?

Because I was an openly gay teacher, students every semester would come out to me, asking what the “gay scene” was like, or how to deal with a homophobic family. Sometimes, students would seek counsel about an unplanned pregnancy, about abortion. In one instance, a student sought counsel about her violent thoughts, and eventually checked herself into a psychiatric ward. Five separate times, students in my class were murdered and I accompanied my classes through mourning.

Unlike many pediatricians, a teacher has a lot of time with these young adults: daily, sometimes over years. Students often admit they spend more time with their teachers than their parents. I can’t give you that time, but here are some general tips.
 

Attitude promoting trust

My guiding attitude toward teens was that they were my equals. I would “do unto them as I would have them do unto me.” Not less, but also not more – because sometimes “more” can cloak condescension. When I was a student, I trusted teachers who shared their fears and mistakes, not performing under a confessional spotlight, but to establish commonality, to flatten hierarchy. I also trusted those who could set boundaries and wield authority compassionately. Because sometimes I needed a firm hand. And so, as an adult I tried to give this to my students as well.

Although my students and I were equals, our situations are different. That is true with gender, race, and class, and it is also true with adults versus teens. The first step toward treating someone authentically as an equal when in a position of authority is to understand the unique stressors of their life. That means asking questions and listening to what they need.
 

Stressors in a teen’s life

A typical high school junior or senior goes to work 8-10 hours a day. Unpaid. They sit for hours at a small desk in a small room with sometimes 34 others. Most of the time they cannot eat or use their phone. If they need to pee, they need to ask permission. They have to ask permission to speak. And then when they go home, they sit at a small desk again for homework. They often do not even have their own room. They also have to ask for permission to buy something for themselves, for money, for a ride anywhere. Their values are often compromised so they won’t get kicked out of a house or a class. The life of a teen is not at all “carefree” but largely prescribed and with little control.

When I think about my youth and how little freedom, privacy, and control I had compared with now, it softens my attitude to even the rudest student. (Isn’t rudeness often a sign of resistance against an oppressive system?) But, some may say, these teens do not have to worry about bills. But if I think back honestly to my teen years, would I trade the responsibilities I have now for those supposed carefree years? Carefree is not how most teens describe their lives but a nostalgic rosy retrospection adults assign. Almost all teens I taught would rather work to gain some control over their lives. Which is why so many work 4-5 hours after school on top of homework, giving up their weekends, and binding themselves to a “carefree” 60- to 80-hour work week.
 

Talking about drugs, sex, and mental health

Drugs

It’s a good idea to first disarm teens of their fear of judgment or punishment, saying things like: “It’s normal to experiment with drugs, even hard ones.” The most successful, respected adults you see now have, so it’s not a reflection of who you are. Tell me what you’re worried about and it’ll be just between us.

After rapport is established, follow-up questions that elicit and affirm their feelings and thoughts can encourage more revelations: Do you think you have a problem? Why? How do you get your drugs and I’m curious only because finding that out can help us understand risks and solutions. What made you start? And keep on using?
 

Sex

Again, first disarm their fears: You can talk to me freely and confidently about sex: What you do, who you do it with, how you do it, and how often – I know that people are very different in their sexual interests and activities.

It is also good to set up clear boundaries. I had instances where students had romantic interest in me and would use these conversations as overtures. If you feel like your patient may be interested in you, then be explicit about boundaries: I’m a doctor who can point you to resources or offer treatments related to any sexual practice and its consequences, but that is all I am. Anything else is illegal and would end our patient-doctor relationship. (I would also immediately document the interaction and tell it to a witness.)

I never escalated incidences like this because I understood that most teens are naturally curious and often not taught about sexual boundaries, so I tried to make these encounters “teachable moments,” not punitive ones. Many teens are more aware of health consequences, like STDs or pregnancy, than psychological ones. So, it’s useful to ask: When you have sex outside your relationship, how does that make you feel? Does sex with multiple partners make you anxious or guilty afterwards? I like to use straightforward language and normalize taboo sexual practices with an even tone to allow teens to speak truthfully.
 

Suicide/depression

First, disarm and normalize: It is very common for people to have depression or thoughts of suicide. Most of the adults around you probably have and so have I (if that is true). Have you experienced this? Older teens often crave an intelligent open discussion about depression and suicide. If they look particularly distressed, I also tell them that I, and countless others, found strategies to deal with these thoughts. For most older teens, talking about causes of mental health issues and treatments is a breath of fresh air. This is especially true for teens from urban communities who have dealt precociously with death and violence, minority communities where mental health is often stigmatized, and young males whose machismo code can prevent them from acknowledging their feelings.

Some follow-up questions: Where do you think these thoughts come from? And if they don’t know: It’s perfectly normal for there to be no reason. The important thing is that they don’t last too long and that you know that. And if they do, then I can provide you resources and potential treatments.
 

Summary

Treating teens as equals by understanding their situation allows understanding and compassion for their stressors. This motivates an inquisitive and collaborative patient-centric approach that allows a sharing of sensitive topics like drugs, sex, and mental health.

Dr. Nguyen is a resident in psychiatry at the University of California, San Francisco.

*This story was updated on Nov. 3, 2022.

After 15 years as a high school teacher at urban schools, I realized adults widely misunderstand that teenagers do not want to talk to them. In fact, most crave finding an adult they can trust and have serious conversations about issues like sex, drugs, and death. G was a sophomore who was going blind from a rare degenerative disease and one day sought my guidance about a sexual orgy he accidentally got in involved in. Was it wrong? Would God send him to hell? Why was he now so anxious after?

Because I was an openly gay teacher, students every semester would come out to me, asking what the “gay scene” was like, or how to deal with a homophobic family. Sometimes, students would seek counsel about an unplanned pregnancy, about abortion. In one instance, a student sought counsel about her violent thoughts, and eventually checked herself into a psychiatric ward. Five separate times, students in my class were murdered and I accompanied my classes through mourning.

Unlike many pediatricians, a teacher has a lot of time with these young adults: daily, sometimes over years. Students often admit they spend more time with their teachers than their parents. I can’t give you that time, but here are some general tips.
 

Attitude promoting trust

My guiding attitude toward teens was that they were my equals. I would “do unto them as I would have them do unto me.” Not less, but also not more – because sometimes “more” can cloak condescension. When I was a student, I trusted teachers who shared their fears and mistakes, not performing under a confessional spotlight, but to establish commonality, to flatten hierarchy. I also trusted those who could set boundaries and wield authority compassionately. Because sometimes I needed a firm hand. And so, as an adult I tried to give this to my students as well.

Although my students and I were equals, our situations are different. That is true with gender, race, and class, and it is also true with adults versus teens. The first step toward treating someone authentically as an equal when in a position of authority is to understand the unique stressors of their life. That means asking questions and listening to what they need.
 

Stressors in a teen’s life

A typical high school junior or senior goes to work 8-10 hours a day. Unpaid. They sit for hours at a small desk in a small room with sometimes 34 others. Most of the time they cannot eat or use their phone. If they need to pee, they need to ask permission. They have to ask permission to speak. And then when they go home, they sit at a small desk again for homework. They often do not even have their own room. They also have to ask for permission to buy something for themselves, for money, for a ride anywhere. Their values are often compromised so they won’t get kicked out of a house or a class. The life of a teen is not at all “carefree” but largely prescribed and with little control.

When I think about my youth and how little freedom, privacy, and control I had compared with now, it softens my attitude to even the rudest student. (Isn’t rudeness often a sign of resistance against an oppressive system?) But, some may say, these teens do not have to worry about bills. But if I think back honestly to my teen years, would I trade the responsibilities I have now for those supposed carefree years? Carefree is not how most teens describe their lives but a nostalgic rosy retrospection adults assign. Almost all teens I taught would rather work to gain some control over their lives. Which is why so many work 4-5 hours after school on top of homework, giving up their weekends, and binding themselves to a “carefree” 60- to 80-hour work week.
 

Talking about drugs, sex, and mental health

Drugs

It’s a good idea to first disarm teens of their fear of judgment or punishment, saying things like: “It’s normal to experiment with drugs, even hard ones.” The most successful, respected adults you see now have, so it’s not a reflection of who you are. Tell me what you’re worried about and it’ll be just between us.

After rapport is established, follow-up questions that elicit and affirm their feelings and thoughts can encourage more revelations: Do you think you have a problem? Why? How do you get your drugs and I’m curious only because finding that out can help us understand risks and solutions. What made you start? And keep on using?
 

Sex

Again, first disarm their fears: You can talk to me freely and confidently about sex: What you do, who you do it with, how you do it, and how often – I know that people are very different in their sexual interests and activities.

It is also good to set up clear boundaries. I had instances where students had romantic interest in me and would use these conversations as overtures. If you feel like your patient may be interested in you, then be explicit about boundaries: I’m a doctor who can point you to resources or offer treatments related to any sexual practice and its consequences, but that is all I am. Anything else is illegal and would end our patient-doctor relationship. (I would also immediately document the interaction and tell it to a witness.)

I never escalated incidences like this because I understood that most teens are naturally curious and often not taught about sexual boundaries, so I tried to make these encounters “teachable moments,” not punitive ones. Many teens are more aware of health consequences, like STDs or pregnancy, than psychological ones. So, it’s useful to ask: When you have sex outside your relationship, how does that make you feel? Does sex with multiple partners make you anxious or guilty afterwards? I like to use straightforward language and normalize taboo sexual practices with an even tone to allow teens to speak truthfully.
 

Suicide/depression

First, disarm and normalize: It is very common for people to have depression or thoughts of suicide. Most of the adults around you probably have and so have I (if that is true). Have you experienced this? Older teens often crave an intelligent open discussion about depression and suicide. If they look particularly distressed, I also tell them that I, and countless others, found strategies to deal with these thoughts. For most older teens, talking about causes of mental health issues and treatments is a breath of fresh air. This is especially true for teens from urban communities who have dealt precociously with death and violence, minority communities where mental health is often stigmatized, and young males whose machismo code can prevent them from acknowledging their feelings.

Some follow-up questions: Where do you think these thoughts come from? And if they don’t know: It’s perfectly normal for there to be no reason. The important thing is that they don’t last too long and that you know that. And if they do, then I can provide you resources and potential treatments.
 

Summary

Treating teens as equals by understanding their situation allows understanding and compassion for their stressors. This motivates an inquisitive and collaborative patient-centric approach that allows a sharing of sensitive topics like drugs, sex, and mental health.

Dr. Nguyen is a resident in psychiatry at the University of California, San Francisco.

*This story was updated on Nov. 3, 2022.

Close to 4 million babies are born in the United States every year, and within their first 48 hours nearly all are pricked in the heel so their blood can be tested for dozens of life-threatening genetic and metabolic problems. The heel-stick test is considered such a crucial public health measure that states typically require it and parents aren’t asked for their permission before it’s done.

But the lab tests for newborn screenings generally don’t use all of the half-dozen or so drops of blood collected on filter paper cards. So states hold on to the leftover “dried blood spots,” as they’re called, often without parents’ knowledge or consent. In recent years, privacy-related concerns have grown about the sometimes decades-long storage and use of the material.

Some states allow the blood spots to be used in research studies, sometimes by third parties for a fee, or provided to law enforcement personnel investigating a crime. Permitting these or other uses without parents’ informed consent that they understand and agree to the use has prompted lawsuits from parents who want to make those decisions themselves and who seek to protect their children’s medical and genetic information.

In May, Michigan officials reportedly agreed to destroy more than 3 million blood spots as a partial settlement in a lawsuit brought by parents who said they didn’t receive enough clear information to provide informed consent for the blood to be used in research the state might conduct. The fate of millions of additional blood spots stored by the state will be determined at trial.

Philip L. Ellison, an attorney in Hemlock, Mich., who is spearheading the suit, said he became aware of the issue when his son was born 5 years ago. Mr. Ellison’s son, Patton, spent his first days in the neonatal intensive care unit after his blood sugar levels dropped precipitously after birth. The next morning, Mr. Ellison said, he was approached by a hospital staffer who asked whether he wanted to sign a consent form allowing the blood from Patton’s heel-stick test to be donated for research.

The unexpected request set off alarm bells for Mr. Ellison.

“We don’t know what the future will bring in terms of information that can be extracted from our blood,” he said. How the rules for using that blood might evolve over time is difficult to know. “A program that first starts out for one purpose, to test for disease, has now crept into medical research and then to law enforcement.”

Michigan is the rare state that asks parents for permission to use leftover newborn blood spots in research. Most do not, experts said. The state screens newborns for more than 50 diseases, such as cystic fibrosis and congenital hypothyroidism, because identifying and treating such illnesses early in a child’s life are crucial.

Afterward, whatever is left over is stored for up to 100 years and, if parents agree to it, may be used in research approved by the Michigan Department of Health and Human Services. Some recent studies have used deidentified blood spots to study the relationship between viral infection at birth and the development of autism later in life, as well as the impact of maternal exposure to manufactured chemicals known as PFAS on health outcomes.

Parents have also asked that their children’s blood spots be sent to researchers to help diagnose a disorder or to try to find a reason for a child’s death, said Chelsea Wuth, a spokesperson for the Michigan Department of Health and Human Services.

Michigan parents can request that the state destroy the leftover blood spots if they don’t want the state to hold on to them.

Since the 1960s, states have screened newborn blood for conditions that can lead to devastating physical or mental disabilities or death if they are not diagnosed and treated. The federal government recommends that roughly three dozen screening tests be performed, but some states conduct many more. Every year, an estimated 13,000 infants with serious medical conditions are identified through newborn screening programs, according to data published by the federal Centers for Disease Control and Prevention.

Many public health experts strongly support mandatory newborn screening as a critical component of infants’ clinical care. But some are receptive to giving parents a say in what happens to the blood after the screening.

“I have always believed that parents should be able to have the opportunity to say ‘yes’ or ‘no’ ” to having their newborns’ leftover blood used in research, said Beth Tarini, MD, a pediatrician and the associate director of the Center for Translational Research at Children’s National Research Institute in Washington, D.C. “Since it is not part of the clinical care, it is a different standard of engagement with the parents.”

In Michigan, 64% of parents consented to participate, according to court documents in Mr. Ellison’s case.

Encouraging people to participate is important, some public health experts say, because the blood spot repositories provide a rare opportunity for population-level research. People of European descent are often overrepresented in genetic databases, which can skew the results of studies. But the newborn screening program includes virtually everyone born in the United States.

“There’s strong evidence that research conducted on samples of white people creates disparities in the benefits of biomedical research for people who are not white,” said Kyle Brothers, MD, PhD, a pediatrician and bioethicist at Norton Children’s Research Institute in Louisville, Ky.

After privacy-related lawsuits were brought in 2009 and 2011 by parents in Texas and Minnesota, respectively, millions of blood spots were destroyed.

Brothers said an unwillingness to participate in research programs reflects larger trends, including more emphasis on the individual and less on contributing to the general good.

To those who might argue that parents’ privacy concerns are overblown, a recent lawsuit in New Jersey raises troubling questions.

In a public records lawsuit, the New Jersey Office of the Public Defender and the New Jersey Monitor, a nonprofit news site, charge that the state police used a subpoena to obtain an infant blood spot of a child who is now 9 years old from the state’s newborn screening laboratory. The lawsuit says a DNA analysis was conducted on the blood spot so evidence could be gathered against the child’s father, who was being represented by the public defender’s office, in connection with a sexual assault committed in 1996. The effort allowed police to get the DNA information without having to show a court probable cause, the suit alleges.

The lawsuit seeks to find out how often in the past 5 years New Jersey law enforcement agencies have used the newborn screening lab as a tool in investigations and subjected defendants to “warrantless searches and seizures.”

New Jersey keeps the records on file for 23 years, said CJ Griffin, a lawyer representing the public defender’s office and the New Jersey Monitor in the lawsuit.

Ms. Griffin said her clients aren’t challenging the program to test newborn blood for diseases. “It’s more the lack of transparency, and safeguards, and information about storage, and we don’t have any information about appropriate use.”

The New Jersey Department of Health doesn’t comment on pending litigation, spokesperson Nancy Kearney said. Ms. Kearney didn’t respond to a request for information about the state’s practices and policies related to the newborn screening program.

A recent Texas Law Review article found that more than a quarter of states lack policies on law enforcement access to newborn blood spot samples and related information and that nearly a third may allow access in certain circumstances.

In Michigan, the state gives law enforcement agencies dried blood spots only to identify the victim of a crime, Ms. Wuth said. “Typically, this means someone has been killed or gone missing,” she added.

Many clinicians and bioethicists say that standards for the use of blood spots need to be set.

“It’s nearly impossible for us to monitor the potential uses of our data,” said Andrew Crawford, senior policy counsel for the privacy and data project at the Center for Democracy and Technology. “That’s why need to put limitations on the use.”

KHN (Kaiser Health News) is a national newsroom that produces in-depth journalism about health issues. Together with Policy Analysis and Polling, KHN is one of the three major operating programs at KFF (Kaiser Family Foundation). KFF is an endowed nonprofit organization providing information on health issues to the nation.

Close to 4 million babies are born in the United States every year, and within their first 48 hours nearly all are pricked in the heel so their blood can be tested for dozens of life-threatening genetic and metabolic problems. The heel-stick test is considered such a crucial public health measure that states typically require it and parents aren’t asked for their permission before it’s done.

But the lab tests for newborn screenings generally don’t use all of the half-dozen or so drops of blood collected on filter paper cards. So states hold on to the leftover “dried blood spots,” as they’re called, often without parents’ knowledge or consent. In recent years, privacy-related concerns have grown about the sometimes decades-long storage and use of the material.

Some states allow the blood spots to be used in research studies, sometimes by third parties for a fee, or provided to law enforcement personnel investigating a crime. Permitting these or other uses without parents’ informed consent that they understand and agree to the use has prompted lawsuits from parents who want to make those decisions themselves and who seek to protect their children’s medical and genetic information.

In May, Michigan officials reportedly agreed to destroy more than 3 million blood spots as a partial settlement in a lawsuit brought by parents who said they didn’t receive enough clear information to provide informed consent for the blood to be used in research the state might conduct. The fate of millions of additional blood spots stored by the state will be determined at trial.

Philip L. Ellison, an attorney in Hemlock, Mich., who is spearheading the suit, said he became aware of the issue when his son was born 5 years ago. Mr. Ellison’s son, Patton, spent his first days in the neonatal intensive care unit after his blood sugar levels dropped precipitously after birth. The next morning, Mr. Ellison said, he was approached by a hospital staffer who asked whether he wanted to sign a consent form allowing the blood from Patton’s heel-stick test to be donated for research.

The unexpected request set off alarm bells for Mr. Ellison.

“We don’t know what the future will bring in terms of information that can be extracted from our blood,” he said. How the rules for using that blood might evolve over time is difficult to know. “A program that first starts out for one purpose, to test for disease, has now crept into medical research and then to law enforcement.”

Michigan is the rare state that asks parents for permission to use leftover newborn blood spots in research. Most do not, experts said. The state screens newborns for more than 50 diseases, such as cystic fibrosis and congenital hypothyroidism, because identifying and treating such illnesses early in a child’s life are crucial.

Afterward, whatever is left over is stored for up to 100 years and, if parents agree to it, may be used in research approved by the Michigan Department of Health and Human Services. Some recent studies have used deidentified blood spots to study the relationship between viral infection at birth and the development of autism later in life, as well as the impact of maternal exposure to manufactured chemicals known as PFAS on health outcomes.

Parents have also asked that their children’s blood spots be sent to researchers to help diagnose a disorder or to try to find a reason for a child’s death, said Chelsea Wuth, a spokesperson for the Michigan Department of Health and Human Services.

Michigan parents can request that the state destroy the leftover blood spots if they don’t want the state to hold on to them.

Since the 1960s, states have screened newborn blood for conditions that can lead to devastating physical or mental disabilities or death if they are not diagnosed and treated. The federal government recommends that roughly three dozen screening tests be performed, but some states conduct many more. Every year, an estimated 13,000 infants with serious medical conditions are identified through newborn screening programs, according to data published by the federal Centers for Disease Control and Prevention.

Many public health experts strongly support mandatory newborn screening as a critical component of infants’ clinical care. But some are receptive to giving parents a say in what happens to the blood after the screening.

“I have always believed that parents should be able to have the opportunity to say ‘yes’ or ‘no’ ” to having their newborns’ leftover blood used in research, said Beth Tarini, MD, a pediatrician and the associate director of the Center for Translational Research at Children’s National Research Institute in Washington, D.C. “Since it is not part of the clinical care, it is a different standard of engagement with the parents.”

In Michigan, 64% of parents consented to participate, according to court documents in Mr. Ellison’s case.

Encouraging people to participate is important, some public health experts say, because the blood spot repositories provide a rare opportunity for population-level research. People of European descent are often overrepresented in genetic databases, which can skew the results of studies. But the newborn screening program includes virtually everyone born in the United States.

“There’s strong evidence that research conducted on samples of white people creates disparities in the benefits of biomedical research for people who are not white,” said Kyle Brothers, MD, PhD, a pediatrician and bioethicist at Norton Children’s Research Institute in Louisville, Ky.

After privacy-related lawsuits were brought in 2009 and 2011 by parents in Texas and Minnesota, respectively, millions of blood spots were destroyed.

Brothers said an unwillingness to participate in research programs reflects larger trends, including more emphasis on the individual and less on contributing to the general good.

To those who might argue that parents’ privacy concerns are overblown, a recent lawsuit in New Jersey raises troubling questions.

In a public records lawsuit, the New Jersey Office of the Public Defender and the New Jersey Monitor, a nonprofit news site, charge that the state police used a subpoena to obtain an infant blood spot of a child who is now 9 years old from the state’s newborn screening laboratory. The lawsuit says a DNA analysis was conducted on the blood spot so evidence could be gathered against the child’s father, who was being represented by the public defender’s office, in connection with a sexual assault committed in 1996. The effort allowed police to get the DNA information without having to show a court probable cause, the suit alleges.

The lawsuit seeks to find out how often in the past 5 years New Jersey law enforcement agencies have used the newborn screening lab as a tool in investigations and subjected defendants to “warrantless searches and seizures.”

New Jersey keeps the records on file for 23 years, said CJ Griffin, a lawyer representing the public defender’s office and the New Jersey Monitor in the lawsuit.

Ms. Griffin said her clients aren’t challenging the program to test newborn blood for diseases. “It’s more the lack of transparency, and safeguards, and information about storage, and we don’t have any information about appropriate use.”

The New Jersey Department of Health doesn’t comment on pending litigation, spokesperson Nancy Kearney said. Ms. Kearney didn’t respond to a request for information about the state’s practices and policies related to the newborn screening program.

A recent Texas Law Review article found that more than a quarter of states lack policies on law enforcement access to newborn blood spot samples and related information and that nearly a third may allow access in certain circumstances.

In Michigan, the state gives law enforcement agencies dried blood spots only to identify the victim of a crime, Ms. Wuth said. “Typically, this means someone has been killed or gone missing,” she added.

Many clinicians and bioethicists say that standards for the use of blood spots need to be set.

“It’s nearly impossible for us to monitor the potential uses of our data,” said Andrew Crawford, senior policy counsel for the privacy and data project at the Center for Democracy and Technology. “That’s why need to put limitations on the use.”

KHN (Kaiser Health News) is a national newsroom that produces in-depth journalism about health issues. Together with Policy Analysis and Polling, KHN is one of the three major operating programs at KFF (Kaiser Family Foundation). KFF is an endowed nonprofit organization providing information on health issues to the nation.

Close to 4 million babies are born in the United States every year, and within their first 48 hours nearly all are pricked in the heel so their blood can be tested for dozens of life-threatening genetic and metabolic problems. The heel-stick test is considered such a crucial public health measure that states typically require it and parents aren’t asked for their permission before it’s done.

But the lab tests for newborn screenings generally don’t use all of the half-dozen or so drops of blood collected on filter paper cards. So states hold on to the leftover “dried blood spots,” as they’re called, often without parents’ knowledge or consent. In recent years, privacy-related concerns have grown about the sometimes decades-long storage and use of the material.

Some states allow the blood spots to be used in research studies, sometimes by third parties for a fee, or provided to law enforcement personnel investigating a crime. Permitting these or other uses without parents’ informed consent that they understand and agree to the use has prompted lawsuits from parents who want to make those decisions themselves and who seek to protect their children’s medical and genetic information.

In May, Michigan officials reportedly agreed to destroy more than 3 million blood spots as a partial settlement in a lawsuit brought by parents who said they didn’t receive enough clear information to provide informed consent for the blood to be used in research the state might conduct. The fate of millions of additional blood spots stored by the state will be determined at trial.

Philip L. Ellison, an attorney in Hemlock, Mich., who is spearheading the suit, said he became aware of the issue when his son was born 5 years ago. Mr. Ellison’s son, Patton, spent his first days in the neonatal intensive care unit after his blood sugar levels dropped precipitously after birth. The next morning, Mr. Ellison said, he was approached by a hospital staffer who asked whether he wanted to sign a consent form allowing the blood from Patton’s heel-stick test to be donated for research.

The unexpected request set off alarm bells for Mr. Ellison.

“We don’t know what the future will bring in terms of information that can be extracted from our blood,” he said. How the rules for using that blood might evolve over time is difficult to know. “A program that first starts out for one purpose, to test for disease, has now crept into medical research and then to law enforcement.”

Michigan is the rare state that asks parents for permission to use leftover newborn blood spots in research. Most do not, experts said. The state screens newborns for more than 50 diseases, such as cystic fibrosis and congenital hypothyroidism, because identifying and treating such illnesses early in a child’s life are crucial.

Afterward, whatever is left over is stored for up to 100 years and, if parents agree to it, may be used in research approved by the Michigan Department of Health and Human Services. Some recent studies have used deidentified blood spots to study the relationship between viral infection at birth and the development of autism later in life, as well as the impact of maternal exposure to manufactured chemicals known as PFAS on health outcomes.

Parents have also asked that their children’s blood spots be sent to researchers to help diagnose a disorder or to try to find a reason for a child’s death, said Chelsea Wuth, a spokesperson for the Michigan Department of Health and Human Services.

Michigan parents can request that the state destroy the leftover blood spots if they don’t want the state to hold on to them.

Since the 1960s, states have screened newborn blood for conditions that can lead to devastating physical or mental disabilities or death if they are not diagnosed and treated. The federal government recommends that roughly three dozen screening tests be performed, but some states conduct many more. Every year, an estimated 13,000 infants with serious medical conditions are identified through newborn screening programs, according to data published by the federal Centers for Disease Control and Prevention.

Many public health experts strongly support mandatory newborn screening as a critical component of infants’ clinical care. But some are receptive to giving parents a say in what happens to the blood after the screening.

“I have always believed that parents should be able to have the opportunity to say ‘yes’ or ‘no’ ” to having their newborns’ leftover blood used in research, said Beth Tarini, MD, a pediatrician and the associate director of the Center for Translational Research at Children’s National Research Institute in Washington, D.C. “Since it is not part of the clinical care, it is a different standard of engagement with the parents.”

In Michigan, 64% of parents consented to participate, according to court documents in Mr. Ellison’s case.

Encouraging people to participate is important, some public health experts say, because the blood spot repositories provide a rare opportunity for population-level research. People of European descent are often overrepresented in genetic databases, which can skew the results of studies. But the newborn screening program includes virtually everyone born in the United States.

“There’s strong evidence that research conducted on samples of white people creates disparities in the benefits of biomedical research for people who are not white,” said Kyle Brothers, MD, PhD, a pediatrician and bioethicist at Norton Children’s Research Institute in Louisville, Ky.

After privacy-related lawsuits were brought in 2009 and 2011 by parents in Texas and Minnesota, respectively, millions of blood spots were destroyed.

Brothers said an unwillingness to participate in research programs reflects larger trends, including more emphasis on the individual and less on contributing to the general good.

To those who might argue that parents’ privacy concerns are overblown, a recent lawsuit in New Jersey raises troubling questions.

In a public records lawsuit, the New Jersey Office of the Public Defender and the New Jersey Monitor, a nonprofit news site, charge that the state police used a subpoena to obtain an infant blood spot of a child who is now 9 years old from the state’s newborn screening laboratory. The lawsuit says a DNA analysis was conducted on the blood spot so evidence could be gathered against the child’s father, who was being represented by the public defender’s office, in connection with a sexual assault committed in 1996. The effort allowed police to get the DNA information without having to show a court probable cause, the suit alleges.

The lawsuit seeks to find out how often in the past 5 years New Jersey law enforcement agencies have used the newborn screening lab as a tool in investigations and subjected defendants to “warrantless searches and seizures.”

New Jersey keeps the records on file for 23 years, said CJ Griffin, a lawyer representing the public defender’s office and the New Jersey Monitor in the lawsuit.

Ms. Griffin said her clients aren’t challenging the program to test newborn blood for diseases. “It’s more the lack of transparency, and safeguards, and information about storage, and we don’t have any information about appropriate use.”

The New Jersey Department of Health doesn’t comment on pending litigation, spokesperson Nancy Kearney said. Ms. Kearney didn’t respond to a request for information about the state’s practices and policies related to the newborn screening program.

A recent Texas Law Review article found that more than a quarter of states lack policies on law enforcement access to newborn blood spot samples and related information and that nearly a third may allow access in certain circumstances.

In Michigan, the state gives law enforcement agencies dried blood spots only to identify the victim of a crime, Ms. Wuth said. “Typically, this means someone has been killed or gone missing,” she added.

Many clinicians and bioethicists say that standards for the use of blood spots need to be set.

“It’s nearly impossible for us to monitor the potential uses of our data,” said Andrew Crawford, senior policy counsel for the privacy and data project at the Center for Democracy and Technology. “That’s why need to put limitations on the use.”

KHN (Kaiser Health News) is a national newsroom that produces in-depth journalism about health issues. Together with Policy Analysis and Polling, KHN is one of the three major operating programs at KFF (Kaiser Family Foundation). KFF is an endowed nonprofit organization providing information on health issues to the nation.

California and New York are on the cusp of going further than the Food and Drug Administration in restricting the sale of nonprescription diet pills to minors as pediatricians and public health advocates try to protect kids from extreme weight-loss gimmicks online.

A bill before Gov. Gavin Newsom would bar anyone under 18 in California from buying over-the-counter weight-loss supplements – whether online or in shops – without a prescription. A similar bill passed by New York lawmakers is on Gov. Kathy Hochul’s desk. Neither Democrat has indicated how he or she will act.

kaarsten/Thinkstock

If both bills are signed into law, proponents hope the momentum will build to restrict diet pill sales to children in more states. Massachusetts, New Jersey, and Missouri have introduced similar bills and backers plan to continue their push next year.

Nearly 30 million people in the United States will have an eating disorder in their lifetime; 95% of them are aged between 12 and 25, according to Johns Hopkins All Children’s Hospital. The hospital added that eating disorders pose the highest risk of mortality of any mental health disorder. And it has become easier than ever for minors to get pills that are sold online or on drugstore shelves. All dietary supplements, which include those for weight loss, accounted for nearly 35% of the $63 billion over-the-counter health products industry in 2021, according to Vision Research Reports, a market research firm.

Dietary supplements, which encompass a broad range of vitamins, herbs, and minerals, are classified by the FDA as food and don’t undergo scientific and safety testing as prescription drugs and over-the-counter medicines do.

Public health advocates want to keep weight-loss products – with ads that may promise to “Drop 5 pounds a week!” and pill names like Slim Sense – away from young people, particularly girls, since some research has linked some products to eating disorders. A study in the American Journal of Public Health, which followed more than 10,000 women aged 14-36 over 15 years, found that “those who used diet pills had more than 5 times higher adjusted odds of receiving an eating disorder diagnosis from a health care provider within 1-3 years than those who did not.”

Many pills have been found tainted with banned and dangerous ingredients that may cause cancer, heart attacks, strokes, and other ailments. For example, the FDA advised the public to avoid Slim Sense by Dr. Reade because it contains lorcaserin, which has been found to cause psychiatric disturbances and impairments in attention or memory. The FDA ordered it discontinued and the company couldn’t be reached for comment.

“Unscrupulous manufacturers are willing to take risks with consumers’ health – and they are lacing their products with illegal pharmaceuticals, banned pharmaceuticals, steroids, excessive stimulants, even experimental stimulants,” said S. Bryn Austin, ScD, founding director of the Strategic Training Initiative for the Prevention of Eating Disorders, or STRIPED, which supports the restrictions. “Consumers have no idea that this is what’s in these types of products.”

STRIPED is a public health initiative based at the Harvard School of Public Health, Boston, and Boston Children’s Hospital.

An industry trade group, the Natural Products Association, disputes that diet pills cause eating disorders, citing the lack of consumer complaints to the FDA of adverse events from their members’ products. “According to FDA data, there is no association between the two,” said Kyle Turk, the association’s director of government affairs.

The association contends that its members adhere to safe manufacturing processes, random product testing, and appropriate marketing guidelines. Representatives also worry that if minors can’t buy supplements over the counter, they may buy them from “crooks” on the black market and undermine the integrity of the industry. Under the bills, minors purchasing weight-loss products must show identification along with a prescription.

Not all business groups oppose the ban. The American Herbal Products Association, a trade group representing dietary supplement manufacturers and retailers, dropped its opposition to California’s bill once it was amended to remove ingredient categories that are found in non-diet supplements and vitamins, according to Robert Marriott, director of regulatory affairs.

Children’s advocates have found worrisome trends among young people who envision their ideal body type based on what they see on social media. According to a study commissioned by Fairplay, a nonprofit that seeks to stop harmful marketing practices targeting children, kids as young as 9 were found to be following three or more eating disorder accounts on Instagram, while the median age was 19. The authors called it a “pro–eating disorder bubble.”

Meta, which owns Instagram and Facebook, said the report lacks nuance, such as recognizing the human need to share life’s difficult moments. The company argues that blanket censorship isn’t the answer. “Experts and safety organizations have told us it’s important to strike a balance and allow people to share their personal stories while removing any content that encourages or promotes eating disorders,” Liza Crenshaw, a Meta spokesperson, said in an email.

Jason Nagata, MD, a pediatrician who cares for children and young adults with life-threatening eating disorders, believes that easy access to diet pills contributes to his patients’ conditions at UCSF Benioff Children’s Hospital in San Francisco. That was the case for one of his patients, an emaciated 11-year-old girl.

“She had basically entered a starvation state because she was not getting enough nutrition,” said Dr. Nagata, who provided supporting testimony for the California bill. “She was taking these pills and using other kinds of extreme behaviors to lose weight.”

Dr. Nagata said the number of patients he sees with eating disorders has tripled since the pandemic began. They are desperate to get diet pills, some with modest results. “We’ve had patients who have been so dependent on these products that they will be hospitalized and they’re still ordering these products on Amazon,” he said.

Public health advocates turned to state legislatures in response to the federal government’s limited authority to regulate diet pills. Under a 1994 federal law known as the Dietary Supplement Health and Education Act, the FDA “cannot step in until after there is a clear issue of harm to consumers,” said Dr. Austin.

No match for the supplement industry’s heavy lobbying on Capitol Hill, public health advocates shifted to a state-by-state approach.

There is, however, a push for the FDA to improve oversight of what goes into diet pills. Sen. Dick Durbin (D-Ill.) in April introduced a bill that would require dietary supplement manufacturers to register their products – along with the ingredients – with the regulator.

Proponents say the change is needed because manufacturers have been known to include dangerous ingredients. C. Michael White, PharmD, of the University of Connecticut, Storrs, found 35% of tainted health products came from weight-loss supplements in a review of a health fraud database.

A few ingredients have been banned, including sibutramine, a stimulant. “It was a very commonly used weight-loss supplement that ended up being removed from the U.S. market because of its elevated risk of causing things like heart attacks, strokes, and arrhythmias,” Dr. White said.

Another ingredient was phenolphthalein, which was used in laxatives until it was identified as a suspected carcinogen and banned in 1999. “To think,” he said, “that that product would still be on the U.S. market is just unconscionable.”

This story was produced by KHN, which publishes California Healthline, an editorially independent service of the California Health Care Foundation. KHN (Kaiser Health News) is a national newsroom that produces in-depth journalism about health issues. Together with Policy Analysis and Polling, KHN is one of the three major operating programs at KFF (Kaiser Family Foundation). KFF is an endowed nonprofit organization providing information on health issues to the nation.

California and New York are on the cusp of going further than the Food and Drug Administration in restricting the sale of nonprescription diet pills to minors as pediatricians and public health advocates try to protect kids from extreme weight-loss gimmicks online.

A bill before Gov. Gavin Newsom would bar anyone under 18 in California from buying over-the-counter weight-loss supplements – whether online or in shops – without a prescription. A similar bill passed by New York lawmakers is on Gov. Kathy Hochul’s desk. Neither Democrat has indicated how he or she will act.

kaarsten/Thinkstock

If both bills are signed into law, proponents hope the momentum will build to restrict diet pill sales to children in more states. Massachusetts, New Jersey, and Missouri have introduced similar bills and backers plan to continue their push next year.

Nearly 30 million people in the United States will have an eating disorder in their lifetime; 95% of them are aged between 12 and 25, according to Johns Hopkins All Children’s Hospital. The hospital added that eating disorders pose the highest risk of mortality of any mental health disorder. And it has become easier than ever for minors to get pills that are sold online or on drugstore shelves. All dietary supplements, which include those for weight loss, accounted for nearly 35% of the $63 billion over-the-counter health products industry in 2021, according to Vision Research Reports, a market research firm.

Dietary supplements, which encompass a broad range of vitamins, herbs, and minerals, are classified by the FDA as food and don’t undergo scientific and safety testing as prescription drugs and over-the-counter medicines do.

Public health advocates want to keep weight-loss products – with ads that may promise to “Drop 5 pounds a week!” and pill names like Slim Sense – away from young people, particularly girls, since some research has linked some products to eating disorders. A study in the American Journal of Public Health, which followed more than 10,000 women aged 14-36 over 15 years, found that “those who used diet pills had more than 5 times higher adjusted odds of receiving an eating disorder diagnosis from a health care provider within 1-3 years than those who did not.”

Many pills have been found tainted with banned and dangerous ingredients that may cause cancer, heart attacks, strokes, and other ailments. For example, the FDA advised the public to avoid Slim Sense by Dr. Reade because it contains lorcaserin, which has been found to cause psychiatric disturbances and impairments in attention or memory. The FDA ordered it discontinued and the company couldn’t be reached for comment.

“Unscrupulous manufacturers are willing to take risks with consumers’ health – and they are lacing their products with illegal pharmaceuticals, banned pharmaceuticals, steroids, excessive stimulants, even experimental stimulants,” said S. Bryn Austin, ScD, founding director of the Strategic Training Initiative for the Prevention of Eating Disorders, or STRIPED, which supports the restrictions. “Consumers have no idea that this is what’s in these types of products.”

STRIPED is a public health initiative based at the Harvard School of Public Health, Boston, and Boston Children’s Hospital.

An industry trade group, the Natural Products Association, disputes that diet pills cause eating disorders, citing the lack of consumer complaints to the FDA of adverse events from their members’ products. “According to FDA data, there is no association between the two,” said Kyle Turk, the association’s director of government affairs.

The association contends that its members adhere to safe manufacturing processes, random product testing, and appropriate marketing guidelines. Representatives also worry that if minors can’t buy supplements over the counter, they may buy them from “crooks” on the black market and undermine the integrity of the industry. Under the bills, minors purchasing weight-loss products must show identification along with a prescription.

Not all business groups oppose the ban. The American Herbal Products Association, a trade group representing dietary supplement manufacturers and retailers, dropped its opposition to California’s bill once it was amended to remove ingredient categories that are found in non-diet supplements and vitamins, according to Robert Marriott, director of regulatory affairs.

Children’s advocates have found worrisome trends among young people who envision their ideal body type based on what they see on social media. According to a study commissioned by Fairplay, a nonprofit that seeks to stop harmful marketing practices targeting children, kids as young as 9 were found to be following three or more eating disorder accounts on Instagram, while the median age was 19. The authors called it a “pro–eating disorder bubble.”

Meta, which owns Instagram and Facebook, said the report lacks nuance, such as recognizing the human need to share life’s difficult moments. The company argues that blanket censorship isn’t the answer. “Experts and safety organizations have told us it’s important to strike a balance and allow people to share their personal stories while removing any content that encourages or promotes eating disorders,” Liza Crenshaw, a Meta spokesperson, said in an email.

Jason Nagata, MD, a pediatrician who cares for children and young adults with life-threatening eating disorders, believes that easy access to diet pills contributes to his patients’ conditions at UCSF Benioff Children’s Hospital in San Francisco. That was the case for one of his patients, an emaciated 11-year-old girl.

“She had basically entered a starvation state because she was not getting enough nutrition,” said Dr. Nagata, who provided supporting testimony for the California bill. “She was taking these pills and using other kinds of extreme behaviors to lose weight.”

Dr. Nagata said the number of patients he sees with eating disorders has tripled since the pandemic began. They are desperate to get diet pills, some with modest results. “We’ve had patients who have been so dependent on these products that they will be hospitalized and they’re still ordering these products on Amazon,” he said.

Public health advocates turned to state legislatures in response to the federal government’s limited authority to regulate diet pills. Under a 1994 federal law known as the Dietary Supplement Health and Education Act, the FDA “cannot step in until after there is a clear issue of harm to consumers,” said Dr. Austin.

No match for the supplement industry’s heavy lobbying on Capitol Hill, public health advocates shifted to a state-by-state approach.

There is, however, a push for the FDA to improve oversight of what goes into diet pills. Sen. Dick Durbin (D-Ill.) in April introduced a bill that would require dietary supplement manufacturers to register their products – along with the ingredients – with the regulator.

Proponents say the change is needed because manufacturers have been known to include dangerous ingredients. C. Michael White, PharmD, of the University of Connecticut, Storrs, found 35% of tainted health products came from weight-loss supplements in a review of a health fraud database.

A few ingredients have been banned, including sibutramine, a stimulant. “It was a very commonly used weight-loss supplement that ended up being removed from the U.S. market because of its elevated risk of causing things like heart attacks, strokes, and arrhythmias,” Dr. White said.

Another ingredient was phenolphthalein, which was used in laxatives until it was identified as a suspected carcinogen and banned in 1999. “To think,” he said, “that that product would still be on the U.S. market is just unconscionable.”

This story was produced by KHN, which publishes California Healthline, an editorially independent service of the California Health Care Foundation. KHN (Kaiser Health News) is a national newsroom that produces in-depth journalism about health issues. Together with Policy Analysis and Polling, KHN is one of the three major operating programs at KFF (Kaiser Family Foundation). KFF is an endowed nonprofit organization providing information on health issues to the nation.

California and New York are on the cusp of going further than the Food and Drug Administration in restricting the sale of nonprescription diet pills to minors as pediatricians and public health advocates try to protect kids from extreme weight-loss gimmicks online.

A bill before Gov. Gavin Newsom would bar anyone under 18 in California from buying over-the-counter weight-loss supplements – whether online or in shops – without a prescription. A similar bill passed by New York lawmakers is on Gov. Kathy Hochul’s desk. Neither Democrat has indicated how he or she will act.

kaarsten/Thinkstock

If both bills are signed into law, proponents hope the momentum will build to restrict diet pill sales to children in more states. Massachusetts, New Jersey, and Missouri have introduced similar bills and backers plan to continue their push next year.

Nearly 30 million people in the United States will have an eating disorder in their lifetime; 95% of them are aged between 12 and 25, according to Johns Hopkins All Children’s Hospital. The hospital added that eating disorders pose the highest risk of mortality of any mental health disorder. And it has become easier than ever for minors to get pills that are sold online or on drugstore shelves. All dietary supplements, which include those for weight loss, accounted for nearly 35% of the $63 billion over-the-counter health products industry in 2021, according to Vision Research Reports, a market research firm.

Dietary supplements, which encompass a broad range of vitamins, herbs, and minerals, are classified by the FDA as food and don’t undergo scientific and safety testing as prescription drugs and over-the-counter medicines do.

Public health advocates want to keep weight-loss products – with ads that may promise to “Drop 5 pounds a week!” and pill names like Slim Sense – away from young people, particularly girls, since some research has linked some products to eating disorders. A study in the American Journal of Public Health, which followed more than 10,000 women aged 14-36 over 15 years, found that “those who used diet pills had more than 5 times higher adjusted odds of receiving an eating disorder diagnosis from a health care provider within 1-3 years than those who did not.”

Many pills have been found tainted with banned and dangerous ingredients that may cause cancer, heart attacks, strokes, and other ailments. For example, the FDA advised the public to avoid Slim Sense by Dr. Reade because it contains lorcaserin, which has been found to cause psychiatric disturbances and impairments in attention or memory. The FDA ordered it discontinued and the company couldn’t be reached for comment.

“Unscrupulous manufacturers are willing to take risks with consumers’ health – and they are lacing their products with illegal pharmaceuticals, banned pharmaceuticals, steroids, excessive stimulants, even experimental stimulants,” said S. Bryn Austin, ScD, founding director of the Strategic Training Initiative for the Prevention of Eating Disorders, or STRIPED, which supports the restrictions. “Consumers have no idea that this is what’s in these types of products.”

STRIPED is a public health initiative based at the Harvard School of Public Health, Boston, and Boston Children’s Hospital.

An industry trade group, the Natural Products Association, disputes that diet pills cause eating disorders, citing the lack of consumer complaints to the FDA of adverse events from their members’ products. “According to FDA data, there is no association between the two,” said Kyle Turk, the association’s director of government affairs.

The association contends that its members adhere to safe manufacturing processes, random product testing, and appropriate marketing guidelines. Representatives also worry that if minors can’t buy supplements over the counter, they may buy them from “crooks” on the black market and undermine the integrity of the industry. Under the bills, minors purchasing weight-loss products must show identification along with a prescription.

Not all business groups oppose the ban. The American Herbal Products Association, a trade group representing dietary supplement manufacturers and retailers, dropped its opposition to California’s bill once it was amended to remove ingredient categories that are found in non-diet supplements and vitamins, according to Robert Marriott, director of regulatory affairs.

Children’s advocates have found worrisome trends among young people who envision their ideal body type based on what they see on social media. According to a study commissioned by Fairplay, a nonprofit that seeks to stop harmful marketing practices targeting children, kids as young as 9 were found to be following three or more eating disorder accounts on Instagram, while the median age was 19. The authors called it a “pro–eating disorder bubble.”

Meta, which owns Instagram and Facebook, said the report lacks nuance, such as recognizing the human need to share life’s difficult moments. The company argues that blanket censorship isn’t the answer. “Experts and safety organizations have told us it’s important to strike a balance and allow people to share their personal stories while removing any content that encourages or promotes eating disorders,” Liza Crenshaw, a Meta spokesperson, said in an email.

Jason Nagata, MD, a pediatrician who cares for children and young adults with life-threatening eating disorders, believes that easy access to diet pills contributes to his patients’ conditions at UCSF Benioff Children’s Hospital in San Francisco. That was the case for one of his patients, an emaciated 11-year-old girl.

“She had basically entered a starvation state because she was not getting enough nutrition,” said Dr. Nagata, who provided supporting testimony for the California bill. “She was taking these pills and using other kinds of extreme behaviors to lose weight.”

Dr. Nagata said the number of patients he sees with eating disorders has tripled since the pandemic began. They are desperate to get diet pills, some with modest results. “We’ve had patients who have been so dependent on these products that they will be hospitalized and they’re still ordering these products on Amazon,” he said.

Public health advocates turned to state legislatures in response to the federal government’s limited authority to regulate diet pills. Under a 1994 federal law known as the Dietary Supplement Health and Education Act, the FDA “cannot step in until after there is a clear issue of harm to consumers,” said Dr. Austin.

No match for the supplement industry’s heavy lobbying on Capitol Hill, public health advocates shifted to a state-by-state approach.

There is, however, a push for the FDA to improve oversight of what goes into diet pills. Sen. Dick Durbin (D-Ill.) in April introduced a bill that would require dietary supplement manufacturers to register their products – along with the ingredients – with the regulator.

Proponents say the change is needed because manufacturers have been known to include dangerous ingredients. C. Michael White, PharmD, of the University of Connecticut, Storrs, found 35% of tainted health products came from weight-loss supplements in a review of a health fraud database.

A few ingredients have been banned, including sibutramine, a stimulant. “It was a very commonly used weight-loss supplement that ended up being removed from the U.S. market because of its elevated risk of causing things like heart attacks, strokes, and arrhythmias,” Dr. White said.

Another ingredient was phenolphthalein, which was used in laxatives until it was identified as a suspected carcinogen and banned in 1999. “To think,” he said, “that that product would still be on the U.S. market is just unconscionable.”

This story was produced by KHN, which publishes California Healthline, an editorially independent service of the California Health Care Foundation. KHN (Kaiser Health News) is a national newsroom that produces in-depth journalism about health issues. Together with Policy Analysis and Polling, KHN is one of the three major operating programs at KFF (Kaiser Family Foundation). KFF is an endowed nonprofit organization providing information on health issues to the nation.