Pediatrics

Respiratory syncytial virus (RSV) is the leading cause of U.S. infant hospitalizations overall and across population subgroups, new data published in the Journal of Infectious Diseases confirm.

Acute bronchiolitis caused by RSV accounted for 9.6% (95% confidence interval, 9.4%-9.9%) and 9.3% (95% CI, 9.0%-9.6%) of total infant hospitalizations from January 2009 to September 2015 and October 2015 to December 2019, respectively.
 

Journal issue includes 14 RSV studies

The latest issue of the journal includes a special section with results from 14 studies related to the widespread, easy-to-catch virus, highlighting the urgency of finding a solution for all infants.

In one study, authors led by Mina Suh, MPH, with EpidStrategies, a division of ToxStrategies in Rockville, Md., reported that, in children under the age of 5 years in the United States, RSV caused 58,000 annual hospitalizations and from 100 to 500 annual deaths from 2009 to 2019 (the latest year data were available).

Globally, in 2015, among infants younger than 6 months, an estimated 1.4 million hospital admissions and 27,300 in-hospital deaths were attributed to RSV lower respiratory tract infection (LRTI).

The researchers used the largest publicly available, all-payer database in the United States – the National (Nationwide) Inpatient Sample – to describe the leading causes of infant hospitalizations.

The authors noted that, because clinicians don’t routinely perform lab tests for RSV, the true health care burden is likely higher and its public health impact greater than these numbers show.

Immunization candidates advance

There are no preventative options currently available to substantially cut RSV infections in all infants, though immunization candidates are advancing, showing safety and efficacy in clinical trials.

Palivizumab is currently the only available option in the United States to prevent RSV and is recommended only for a small group of infants with particular forms of heart or lung disease and those born prematurely at 29 weeks’ gestational age. Further, palivizumab has to be given monthly throughout the RSV season.

Another of the studies in the journal supplement concluded that a universal immunization strategy with one of the candidates, nirsevimab (Sanofi, AstraZeneca), an investigational long-acting monoclonal antibody, could substantially reduce the health burden and economic burden for U.S. infants in their first RSV season.

The researchers, led by Alexia Kieffer, MSc, MPH, with Sanofi, used static decision-analytic modeling for the estimates. Modeled RSV-related outcomes included primary care and ED visits, hospitalizations, including ICU admission and mechanical ventilations, and RSV-related deaths.

“The results of this model suggested that the use of nirsevimab in all infants could reduce health events by 55% and the overall costs to the payer by 49%,” the authors of the study wrote.

According to the study, universal immunization of all infants with nirsevimab is expected to reduce 290,174 RSV-related medically attended LRTI (MALRTI), 24,986 hospitalizations, and cut $612 million in costs to the health care system.

The authors wrote: “While this reduction would be driven by term infants, who account for most of the RSV-MALRTI burden; all infants, including palivizumab-eligible and preterm infants who suffer from significantly higher rates of disease, would benefit from this immunization strategy.”
 

Excitement for another option

Jörn-Hendrik Weitkamp, MD, professor of pediatrics and director for patient-oriented research at Monroe Carell Jr. Children’s Hospital at Vanderbilt University, Nashville, Tenn., said in an interview there is much excitement in the field for nirsevimab as it has significant advantages over palivizumab.

RSV “is a huge burden to the children, the families, the hospitals, and the medical system,” he said.

Ideally there would be a vaccine to offer the best protection, he noted.

“People have spent their lives, their careers trying to develop a vaccine for RSV,” he said, but that has been elusive for more than 60 years. Therefore, passive immunization is the best of the current options, he says, and nirsevimab “seems to be very effective.”

What’s not clear, Dr. Weitkamp said, is how much nirsevimab will cost as it is not yet approved by the Food and Drug Administration. However, it has the great advantage of being given only once before the season starts instead of monthly (as required for palivizumab) through the season, “which is painful, inconvenient, and traumatizing. We limit that one to the children at highest risk.”

Rolling out an infant nirsevimab program would likely vary by geographic region, Ms. Kieffer and colleagues said, to help ensure infants are protected during the peak of their region’s RSV season.

The journal’s RSV supplement was supported by Sanofi and AstraZeneca. The studies by Ms. Suh and colleagues and Ms. Kieffer and colleagues were supported by AstraZeneca and Sanofi. Ms. Suh and several coauthors are employees of EpidStrategies. One coauthor is an employee of Sanofi and may hold shares and/or stock options in the company. Ms. Kieffer and several coauthors are employees of Sanofi and may hold shares and/or stock options in the company. Dr. Weitkamp reported no relevant financial relationships.

Respiratory syncytial virus (RSV) is the leading cause of U.S. infant hospitalizations overall and across population subgroups, new data published in the Journal of Infectious Diseases confirm.

Acute bronchiolitis caused by RSV accounted for 9.6% (95% confidence interval, 9.4%-9.9%) and 9.3% (95% CI, 9.0%-9.6%) of total infant hospitalizations from January 2009 to September 2015 and October 2015 to December 2019, respectively.
 

Journal issue includes 14 RSV studies

The latest issue of the journal includes a special section with results from 14 studies related to the widespread, easy-to-catch virus, highlighting the urgency of finding a solution for all infants.

In one study, authors led by Mina Suh, MPH, with EpidStrategies, a division of ToxStrategies in Rockville, Md., reported that, in children under the age of 5 years in the United States, RSV caused 58,000 annual hospitalizations and from 100 to 500 annual deaths from 2009 to 2019 (the latest year data were available).

Globally, in 2015, among infants younger than 6 months, an estimated 1.4 million hospital admissions and 27,300 in-hospital deaths were attributed to RSV lower respiratory tract infection (LRTI).

The researchers used the largest publicly available, all-payer database in the United States – the National (Nationwide) Inpatient Sample – to describe the leading causes of infant hospitalizations.

The authors noted that, because clinicians don’t routinely perform lab tests for RSV, the true health care burden is likely higher and its public health impact greater than these numbers show.

Immunization candidates advance

There are no preventative options currently available to substantially cut RSV infections in all infants, though immunization candidates are advancing, showing safety and efficacy in clinical trials.

Palivizumab is currently the only available option in the United States to prevent RSV and is recommended only for a small group of infants with particular forms of heart or lung disease and those born prematurely at 29 weeks’ gestational age. Further, palivizumab has to be given monthly throughout the RSV season.

Another of the studies in the journal supplement concluded that a universal immunization strategy with one of the candidates, nirsevimab (Sanofi, AstraZeneca), an investigational long-acting monoclonal antibody, could substantially reduce the health burden and economic burden for U.S. infants in their first RSV season.

The researchers, led by Alexia Kieffer, MSc, MPH, with Sanofi, used static decision-analytic modeling for the estimates. Modeled RSV-related outcomes included primary care and ED visits, hospitalizations, including ICU admission and mechanical ventilations, and RSV-related deaths.

“The results of this model suggested that the use of nirsevimab in all infants could reduce health events by 55% and the overall costs to the payer by 49%,” the authors of the study wrote.

According to the study, universal immunization of all infants with nirsevimab is expected to reduce 290,174 RSV-related medically attended LRTI (MALRTI), 24,986 hospitalizations, and cut $612 million in costs to the health care system.

The authors wrote: “While this reduction would be driven by term infants, who account for most of the RSV-MALRTI burden; all infants, including palivizumab-eligible and preterm infants who suffer from significantly higher rates of disease, would benefit from this immunization strategy.”
 

Excitement for another option

Jörn-Hendrik Weitkamp, MD, professor of pediatrics and director for patient-oriented research at Monroe Carell Jr. Children’s Hospital at Vanderbilt University, Nashville, Tenn., said in an interview there is much excitement in the field for nirsevimab as it has significant advantages over palivizumab.

RSV “is a huge burden to the children, the families, the hospitals, and the medical system,” he said.

Ideally there would be a vaccine to offer the best protection, he noted.

“People have spent their lives, their careers trying to develop a vaccine for RSV,” he said, but that has been elusive for more than 60 years. Therefore, passive immunization is the best of the current options, he says, and nirsevimab “seems to be very effective.”

What’s not clear, Dr. Weitkamp said, is how much nirsevimab will cost as it is not yet approved by the Food and Drug Administration. However, it has the great advantage of being given only once before the season starts instead of monthly (as required for palivizumab) through the season, “which is painful, inconvenient, and traumatizing. We limit that one to the children at highest risk.”

Rolling out an infant nirsevimab program would likely vary by geographic region, Ms. Kieffer and colleagues said, to help ensure infants are protected during the peak of their region’s RSV season.

The journal’s RSV supplement was supported by Sanofi and AstraZeneca. The studies by Ms. Suh and colleagues and Ms. Kieffer and colleagues were supported by AstraZeneca and Sanofi. Ms. Suh and several coauthors are employees of EpidStrategies. One coauthor is an employee of Sanofi and may hold shares and/or stock options in the company. Ms. Kieffer and several coauthors are employees of Sanofi and may hold shares and/or stock options in the company. Dr. Weitkamp reported no relevant financial relationships.

Respiratory syncytial virus (RSV) is the leading cause of U.S. infant hospitalizations overall and across population subgroups, new data published in the Journal of Infectious Diseases confirm.

Acute bronchiolitis caused by RSV accounted for 9.6% (95% confidence interval, 9.4%-9.9%) and 9.3% (95% CI, 9.0%-9.6%) of total infant hospitalizations from January 2009 to September 2015 and October 2015 to December 2019, respectively.
 

Journal issue includes 14 RSV studies

The latest issue of the journal includes a special section with results from 14 studies related to the widespread, easy-to-catch virus, highlighting the urgency of finding a solution for all infants.

In one study, authors led by Mina Suh, MPH, with EpidStrategies, a division of ToxStrategies in Rockville, Md., reported that, in children under the age of 5 years in the United States, RSV caused 58,000 annual hospitalizations and from 100 to 500 annual deaths from 2009 to 2019 (the latest year data were available).

Globally, in 2015, among infants younger than 6 months, an estimated 1.4 million hospital admissions and 27,300 in-hospital deaths were attributed to RSV lower respiratory tract infection (LRTI).

The researchers used the largest publicly available, all-payer database in the United States – the National (Nationwide) Inpatient Sample – to describe the leading causes of infant hospitalizations.

The authors noted that, because clinicians don’t routinely perform lab tests for RSV, the true health care burden is likely higher and its public health impact greater than these numbers show.

Immunization candidates advance

There are no preventative options currently available to substantially cut RSV infections in all infants, though immunization candidates are advancing, showing safety and efficacy in clinical trials.

Palivizumab is currently the only available option in the United States to prevent RSV and is recommended only for a small group of infants with particular forms of heart or lung disease and those born prematurely at 29 weeks’ gestational age. Further, palivizumab has to be given monthly throughout the RSV season.

Another of the studies in the journal supplement concluded that a universal immunization strategy with one of the candidates, nirsevimab (Sanofi, AstraZeneca), an investigational long-acting monoclonal antibody, could substantially reduce the health burden and economic burden for U.S. infants in their first RSV season.

The researchers, led by Alexia Kieffer, MSc, MPH, with Sanofi, used static decision-analytic modeling for the estimates. Modeled RSV-related outcomes included primary care and ED visits, hospitalizations, including ICU admission and mechanical ventilations, and RSV-related deaths.

“The results of this model suggested that the use of nirsevimab in all infants could reduce health events by 55% and the overall costs to the payer by 49%,” the authors of the study wrote.

According to the study, universal immunization of all infants with nirsevimab is expected to reduce 290,174 RSV-related medically attended LRTI (MALRTI), 24,986 hospitalizations, and cut $612 million in costs to the health care system.

The authors wrote: “While this reduction would be driven by term infants, who account for most of the RSV-MALRTI burden; all infants, including palivizumab-eligible and preterm infants who suffer from significantly higher rates of disease, would benefit from this immunization strategy.”
 

Excitement for another option

Jörn-Hendrik Weitkamp, MD, professor of pediatrics and director for patient-oriented research at Monroe Carell Jr. Children’s Hospital at Vanderbilt University, Nashville, Tenn., said in an interview there is much excitement in the field for nirsevimab as it has significant advantages over palivizumab.

RSV “is a huge burden to the children, the families, the hospitals, and the medical system,” he said.

Ideally there would be a vaccine to offer the best protection, he noted.

“People have spent their lives, their careers trying to develop a vaccine for RSV,” he said, but that has been elusive for more than 60 years. Therefore, passive immunization is the best of the current options, he says, and nirsevimab “seems to be very effective.”

What’s not clear, Dr. Weitkamp said, is how much nirsevimab will cost as it is not yet approved by the Food and Drug Administration. However, it has the great advantage of being given only once before the season starts instead of monthly (as required for palivizumab) through the season, “which is painful, inconvenient, and traumatizing. We limit that one to the children at highest risk.”

Rolling out an infant nirsevimab program would likely vary by geographic region, Ms. Kieffer and colleagues said, to help ensure infants are protected during the peak of their region’s RSV season.

The journal’s RSV supplement was supported by Sanofi and AstraZeneca. The studies by Ms. Suh and colleagues and Ms. Kieffer and colleagues were supported by AstraZeneca and Sanofi. Ms. Suh and several coauthors are employees of EpidStrategies. One coauthor is an employee of Sanofi and may hold shares and/or stock options in the company. Ms. Kieffer and several coauthors are employees of Sanofi and may hold shares and/or stock options in the company. Dr. Weitkamp reported no relevant financial relationships.

For younger generations, TikTok is a go-to site for those who like short and catchy video clips. As a social media platform that allows concise video sharing, TikTok has over 1 billion monthly global users. Because of its platform size, a plethora of resources, and influence on media discourse, TikTok is the place for content creators to share visual media. Its cursory, condensed content delivery with videos capped at 1-minute focuses on high-yield information and rapid identification of fundamental points that are both engaging and entertaining.

Currently, on TikTok, 40 billion views are associated with the hashtag #mentalhealth. Content creators and regular users are employing this platform to share their own experiences, opinions, and strategies to overcome their struggles. While it is understandable for creators to share their personal stories that may be abusive, traumatic, or violent, they may not be prepared for their video to “go viral.”

Like any other social media platform, hateful speech such as racism, sexism, or xenophobia can accumulate on TikTok, which may cause more self-harm than self-help. Oversharing about personal strategies may lead to misconceived advice for TikTok viewers, while watching these TikTok videos can have negative mental health effects, even though there are no malicious intentions behind the creators who post these videos.

Hence, public health should pay more attention to the potential health-related implications this platform can create, as the quality of the information and the qualifications of the creators are mostly unrevealed. The concerns include undisclosed conflicts of interest, unchecked spread of misinformation, difficulty identifying source credibility, and excessive false information that viewers must filter through.^1,2

Individual TikTok users may follow accounts and interpret these content creators as therapists and the content they see as therapy. They may also believe that a close relationship with the content creator exists when it does not. Specifically, these relationships may be defined as parasocial relationships, which are one-sided relationships where one person (the TikTok viewer) extends emotional energy, interest, and time, and the other party (the content creator) is completely unaware of the other’s existence.³ Additionally, Americans who are uninsured/underinsured may turn to this diluted version of therapy to compensate for the one-on-one or group therapy they need.

While TikTok may seem like a dangerous platform to browse through or post on, its growing influence cannot be underestimated. With 41% of TikTok users between the ages of 16 and 24, this is an ideal platform to disseminate public health information pertaining to this age group (for example, safe sex practices, substance abuse, and mental health issues).⁴ Because younger generations have incorporated social media into their daily lives, the medical community can harness TikTok’s potential to disseminate accurate information to potential patients for targeted medical education.

For example, Jake Goodman, MD, MBA, and Melissa Shepard, MD, each have more than a million TikTok followers and are notable psychiatrists who post a variety of content ranging from recognizing signs of depression to reducing stigma around mental health. Similarly, Justin Puder, PhD, is a licensed psychologist who advocates for ways to overcome mental health issues. By creating diverse content with appealing strategies, spreading accurate medical knowledge, and answering common medical questions for the public, these ‘mental health influencers’ educate potential patients to create patient-centered interactions.

Given the ever-changing digital media landscape, an emphasis must be placed on understanding how adolescents respond to social media in maladaptive or adaptive ways by pointing out the common strengths and weaknesses adolescents share. While there are many pros and cons to social media platforms, it is undeniable that these platforms – such as TikTok – are here to stay. It is crucial for members of the medical community to recognize the outlets that younger generations use to express themselves and to exploit these media channels therapeutically.
 

Ms. Wong is a fourth-year medical student at the New York Institute of Technology College of Osteopathic Medicine in Old Westbury, N.Y. Dr. Chua is a psychiatrist with the department of child and adolescent psychiatry and behavioral sciences at Children’s Hospital of Philadelphia, and assistant professor of clinical psychiatry at the University of Pennsylvania, also in Philadelphia.

References

1. Gottlieb M and Dyer S. Information and Disinformation: Social Media in the COVID-19 Crisis. Acad Emerg Med. 2020 Jul;27(7):640-1. doi: 10.1111/acem.14036.

2. De Veirman M et al. Front Psychol. 2019;10:2685. doi: 10.3389/fpsyg.2019.02685.

3. Bennett N-K et al. “Parasocial Relationships: The Nature of Celebrity Fascinations.” National Register of Health Service Psychologists. https://www.findapsychologist.org/parasocial-relationships-the-nature-of-celebrity-fascinations/.

4. Eghtesadi M and Florea A. Can J Public Health. 2020 Jun;111(3):389-91. doi: 10.17269/s41997-020-00343-0.

For younger generations, TikTok is a go-to site for those who like short and catchy video clips. As a social media platform that allows concise video sharing, TikTok has over 1 billion monthly global users. Because of its platform size, a plethora of resources, and influence on media discourse, TikTok is the place for content creators to share visual media. Its cursory, condensed content delivery with videos capped at 1-minute focuses on high-yield information and rapid identification of fundamental points that are both engaging and entertaining.

Currently, on TikTok, 40 billion views are associated with the hashtag #mentalhealth. Content creators and regular users are employing this platform to share their own experiences, opinions, and strategies to overcome their struggles. While it is understandable for creators to share their personal stories that may be abusive, traumatic, or violent, they may not be prepared for their video to “go viral.”

Like any other social media platform, hateful speech such as racism, sexism, or xenophobia can accumulate on TikTok, which may cause more self-harm than self-help. Oversharing about personal strategies may lead to misconceived advice for TikTok viewers, while watching these TikTok videos can have negative mental health effects, even though there are no malicious intentions behind the creators who post these videos.

Hence, public health should pay more attention to the potential health-related implications this platform can create, as the quality of the information and the qualifications of the creators are mostly unrevealed. The concerns include undisclosed conflicts of interest, unchecked spread of misinformation, difficulty identifying source credibility, and excessive false information that viewers must filter through.^1,2

Individual TikTok users may follow accounts and interpret these content creators as therapists and the content they see as therapy. They may also believe that a close relationship with the content creator exists when it does not. Specifically, these relationships may be defined as parasocial relationships, which are one-sided relationships where one person (the TikTok viewer) extends emotional energy, interest, and time, and the other party (the content creator) is completely unaware of the other’s existence.³ Additionally, Americans who are uninsured/underinsured may turn to this diluted version of therapy to compensate for the one-on-one or group therapy they need.

While TikTok may seem like a dangerous platform to browse through or post on, its growing influence cannot be underestimated. With 41% of TikTok users between the ages of 16 and 24, this is an ideal platform to disseminate public health information pertaining to this age group (for example, safe sex practices, substance abuse, and mental health issues).⁴ Because younger generations have incorporated social media into their daily lives, the medical community can harness TikTok’s potential to disseminate accurate information to potential patients for targeted medical education.

For example, Jake Goodman, MD, MBA, and Melissa Shepard, MD, each have more than a million TikTok followers and are notable psychiatrists who post a variety of content ranging from recognizing signs of depression to reducing stigma around mental health. Similarly, Justin Puder, PhD, is a licensed psychologist who advocates for ways to overcome mental health issues. By creating diverse content with appealing strategies, spreading accurate medical knowledge, and answering common medical questions for the public, these ‘mental health influencers’ educate potential patients to create patient-centered interactions.

Given the ever-changing digital media landscape, an emphasis must be placed on understanding how adolescents respond to social media in maladaptive or adaptive ways by pointing out the common strengths and weaknesses adolescents share. While there are many pros and cons to social media platforms, it is undeniable that these platforms – such as TikTok – are here to stay. It is crucial for members of the medical community to recognize the outlets that younger generations use to express themselves and to exploit these media channels therapeutically.
 

Ms. Wong is a fourth-year medical student at the New York Institute of Technology College of Osteopathic Medicine in Old Westbury, N.Y. Dr. Chua is a psychiatrist with the department of child and adolescent psychiatry and behavioral sciences at Children’s Hospital of Philadelphia, and assistant professor of clinical psychiatry at the University of Pennsylvania, also in Philadelphia.

References

1. Gottlieb M and Dyer S. Information and Disinformation: Social Media in the COVID-19 Crisis. Acad Emerg Med. 2020 Jul;27(7):640-1. doi: 10.1111/acem.14036.

2. De Veirman M et al. Front Psychol. 2019;10:2685. doi: 10.3389/fpsyg.2019.02685.

3. Bennett N-K et al. “Parasocial Relationships: The Nature of Celebrity Fascinations.” National Register of Health Service Psychologists. https://www.findapsychologist.org/parasocial-relationships-the-nature-of-celebrity-fascinations/.

4. Eghtesadi M and Florea A. Can J Public Health. 2020 Jun;111(3):389-91. doi: 10.17269/s41997-020-00343-0.

For younger generations, TikTok is a go-to site for those who like short and catchy video clips. As a social media platform that allows concise video sharing, TikTok has over 1 billion monthly global users. Because of its platform size, a plethora of resources, and influence on media discourse, TikTok is the place for content creators to share visual media. Its cursory, condensed content delivery with videos capped at 1-minute focuses on high-yield information and rapid identification of fundamental points that are both engaging and entertaining.

Currently, on TikTok, 40 billion views are associated with the hashtag #mentalhealth. Content creators and regular users are employing this platform to share their own experiences, opinions, and strategies to overcome their struggles. While it is understandable for creators to share their personal stories that may be abusive, traumatic, or violent, they may not be prepared for their video to “go viral.”

Like any other social media platform, hateful speech such as racism, sexism, or xenophobia can accumulate on TikTok, which may cause more self-harm than self-help. Oversharing about personal strategies may lead to misconceived advice for TikTok viewers, while watching these TikTok videos can have negative mental health effects, even though there are no malicious intentions behind the creators who post these videos.

Hence, public health should pay more attention to the potential health-related implications this platform can create, as the quality of the information and the qualifications of the creators are mostly unrevealed. The concerns include undisclosed conflicts of interest, unchecked spread of misinformation, difficulty identifying source credibility, and excessive false information that viewers must filter through.^1,2

Individual TikTok users may follow accounts and interpret these content creators as therapists and the content they see as therapy. They may also believe that a close relationship with the content creator exists when it does not. Specifically, these relationships may be defined as parasocial relationships, which are one-sided relationships where one person (the TikTok viewer) extends emotional energy, interest, and time, and the other party (the content creator) is completely unaware of the other’s existence.³ Additionally, Americans who are uninsured/underinsured may turn to this diluted version of therapy to compensate for the one-on-one or group therapy they need.

While TikTok may seem like a dangerous platform to browse through or post on, its growing influence cannot be underestimated. With 41% of TikTok users between the ages of 16 and 24, this is an ideal platform to disseminate public health information pertaining to this age group (for example, safe sex practices, substance abuse, and mental health issues).⁴ Because younger generations have incorporated social media into their daily lives, the medical community can harness TikTok’s potential to disseminate accurate information to potential patients for targeted medical education.

For example, Jake Goodman, MD, MBA, and Melissa Shepard, MD, each have more than a million TikTok followers and are notable psychiatrists who post a variety of content ranging from recognizing signs of depression to reducing stigma around mental health. Similarly, Justin Puder, PhD, is a licensed psychologist who advocates for ways to overcome mental health issues. By creating diverse content with appealing strategies, spreading accurate medical knowledge, and answering common medical questions for the public, these ‘mental health influencers’ educate potential patients to create patient-centered interactions.

Given the ever-changing digital media landscape, an emphasis must be placed on understanding how adolescents respond to social media in maladaptive or adaptive ways by pointing out the common strengths and weaknesses adolescents share. While there are many pros and cons to social media platforms, it is undeniable that these platforms – such as TikTok – are here to stay. It is crucial for members of the medical community to recognize the outlets that younger generations use to express themselves and to exploit these media channels therapeutically.
 

Ms. Wong is a fourth-year medical student at the New York Institute of Technology College of Osteopathic Medicine in Old Westbury, N.Y. Dr. Chua is a psychiatrist with the department of child and adolescent psychiatry and behavioral sciences at Children’s Hospital of Philadelphia, and assistant professor of clinical psychiatry at the University of Pennsylvania, also in Philadelphia.

References

1. Gottlieb M and Dyer S. Information and Disinformation: Social Media in the COVID-19 Crisis. Acad Emerg Med. 2020 Jul;27(7):640-1. doi: 10.1111/acem.14036.

2. De Veirman M et al. Front Psychol. 2019;10:2685. doi: 10.3389/fpsyg.2019.02685.

3. Bennett N-K et al. “Parasocial Relationships: The Nature of Celebrity Fascinations.” National Register of Health Service Psychologists. https://www.findapsychologist.org/parasocial-relationships-the-nature-of-celebrity-fascinations/.

4. Eghtesadi M and Florea A. Can J Public Health. 2020 Jun;111(3):389-91. doi: 10.17269/s41997-020-00343-0.

Ibrutinib (Imbruvica) is now available for use in children aged 1-12 years who have chronic graft-versus-host disease (cGVHD), which can develop after stem cell transplantation for treatment of a blood cancer.

Specifically, the indication is for pediatric patients with cGVHD who have already been treated with one or more lines of systemic therapy. The manufacturers have also launched a new oral suspension formulation, in addition to capsules and tablets, which were already available.

Ibrutinib is already approved for use in adults with cGVHD.

The drug is also approved for use in several blood cancers, including chronic lymphocytic leukemia, mantle cell lymphoma, and Waldenström’s macroglobulinemia. All these approvals are for adult patients.

This is the first pediatric indication for the product and is “incredibly meaningful,” said Gauri Sunkersett, DO, associate medical director at AbbVie, which markets the drug together with Jansen. “As a pediatric oncologist, when my patients describe the physical pain they experience from simply hugging their parents, due to their cGVHD, the importance of researching alternative treatment options in this patient population is further validated.”

These children have already been through a lot, having been diagnosed with a leukemia or lymphoma and then undergoing chemotherapy and/or radiotherapy for a stem cell transplant. Just over half (52%-65%) of children who receive allogeneic transplants go on to develop cGVHD, in which the donor bone marrow or stem cells attack the recipient.

“Imagine going through a transplant and then being told you have a moderate to severe chronic disease that can sometimes also be life-threatening,” commented Paul A. Carpenter, MD, attending physician at Seattle Children’s Hospital. “If these children were between 1 and 12 and didn’t respond to steroid treatment, we didn’t have any rigorously studied treatment options – until now.”

The new indication was approved by the U.S. Food and Drug Administration on the basis of results from the iMAGINE trial, for which Dr. Carpenter was a principal investigator.

The phase 1/2 iMAGINE trial was an open-label, multicenter, single-arm trial conducted with 47 patients (mean age, 13 years; range, 1-19 years) with relapsed/refractory cGVHD who had received at least one prior systemic therapy. Ibrutinib was given at a dose of 420 mg orally once daily to patients aged 12 and older and at a dose of 240 mg/m2 orally once daily to patients who were younger than 12 years.

The overall response rate through week 25 was 60% (confidence interval, 95%, 44%-74%). The median duration of response was 5.3 months (95% CI, 2.8-8.8).

The safety profile was consistent with the established profile for ibrutinib. Observed adverse events in pediatric patients were consistent with those observed in adult patients with moderate to severe cGVHD, the companies noted.

The FDA noted that the most common (≥ 20%) adverse reactions, including laboratory abnormalities, were anemia, musculoskeletal pain, pyrexia, diarrhea, pneumonia, abdominal pain, stomatitis, thrombocytopenia, and headache.

Full prescribing information for ibrutinib is available here.

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

Ibrutinib (Imbruvica) is now available for use in children aged 1-12 years who have chronic graft-versus-host disease (cGVHD), which can develop after stem cell transplantation for treatment of a blood cancer.

Specifically, the indication is for pediatric patients with cGVHD who have already been treated with one or more lines of systemic therapy. The manufacturers have also launched a new oral suspension formulation, in addition to capsules and tablets, which were already available.

Ibrutinib is already approved for use in adults with cGVHD.

The drug is also approved for use in several blood cancers, including chronic lymphocytic leukemia, mantle cell lymphoma, and Waldenström’s macroglobulinemia. All these approvals are for adult patients.

This is the first pediatric indication for the product and is “incredibly meaningful,” said Gauri Sunkersett, DO, associate medical director at AbbVie, which markets the drug together with Jansen. “As a pediatric oncologist, when my patients describe the physical pain they experience from simply hugging their parents, due to their cGVHD, the importance of researching alternative treatment options in this patient population is further validated.”

These children have already been through a lot, having been diagnosed with a leukemia or lymphoma and then undergoing chemotherapy and/or radiotherapy for a stem cell transplant. Just over half (52%-65%) of children who receive allogeneic transplants go on to develop cGVHD, in which the donor bone marrow or stem cells attack the recipient.

“Imagine going through a transplant and then being told you have a moderate to severe chronic disease that can sometimes also be life-threatening,” commented Paul A. Carpenter, MD, attending physician at Seattle Children’s Hospital. “If these children were between 1 and 12 and didn’t respond to steroid treatment, we didn’t have any rigorously studied treatment options – until now.”

The new indication was approved by the U.S. Food and Drug Administration on the basis of results from the iMAGINE trial, for which Dr. Carpenter was a principal investigator.

The phase 1/2 iMAGINE trial was an open-label, multicenter, single-arm trial conducted with 47 patients (mean age, 13 years; range, 1-19 years) with relapsed/refractory cGVHD who had received at least one prior systemic therapy. Ibrutinib was given at a dose of 420 mg orally once daily to patients aged 12 and older and at a dose of 240 mg/m2 orally once daily to patients who were younger than 12 years.

The overall response rate through week 25 was 60% (confidence interval, 95%, 44%-74%). The median duration of response was 5.3 months (95% CI, 2.8-8.8).

The safety profile was consistent with the established profile for ibrutinib. Observed adverse events in pediatric patients were consistent with those observed in adult patients with moderate to severe cGVHD, the companies noted.

The FDA noted that the most common (≥ 20%) adverse reactions, including laboratory abnormalities, were anemia, musculoskeletal pain, pyrexia, diarrhea, pneumonia, abdominal pain, stomatitis, thrombocytopenia, and headache.

Full prescribing information for ibrutinib is available here.

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

Ibrutinib (Imbruvica) is now available for use in children aged 1-12 years who have chronic graft-versus-host disease (cGVHD), which can develop after stem cell transplantation for treatment of a blood cancer.

Specifically, the indication is for pediatric patients with cGVHD who have already been treated with one or more lines of systemic therapy. The manufacturers have also launched a new oral suspension formulation, in addition to capsules and tablets, which were already available.

Ibrutinib is already approved for use in adults with cGVHD.

The drug is also approved for use in several blood cancers, including chronic lymphocytic leukemia, mantle cell lymphoma, and Waldenström’s macroglobulinemia. All these approvals are for adult patients.

This is the first pediatric indication for the product and is “incredibly meaningful,” said Gauri Sunkersett, DO, associate medical director at AbbVie, which markets the drug together with Jansen. “As a pediatric oncologist, when my patients describe the physical pain they experience from simply hugging their parents, due to their cGVHD, the importance of researching alternative treatment options in this patient population is further validated.”

These children have already been through a lot, having been diagnosed with a leukemia or lymphoma and then undergoing chemotherapy and/or radiotherapy for a stem cell transplant. Just over half (52%-65%) of children who receive allogeneic transplants go on to develop cGVHD, in which the donor bone marrow or stem cells attack the recipient.

“Imagine going through a transplant and then being told you have a moderate to severe chronic disease that can sometimes also be life-threatening,” commented Paul A. Carpenter, MD, attending physician at Seattle Children’s Hospital. “If these children were between 1 and 12 and didn’t respond to steroid treatment, we didn’t have any rigorously studied treatment options – until now.”

The new indication was approved by the U.S. Food and Drug Administration on the basis of results from the iMAGINE trial, for which Dr. Carpenter was a principal investigator.

The phase 1/2 iMAGINE trial was an open-label, multicenter, single-arm trial conducted with 47 patients (mean age, 13 years; range, 1-19 years) with relapsed/refractory cGVHD who had received at least one prior systemic therapy. Ibrutinib was given at a dose of 420 mg orally once daily to patients aged 12 and older and at a dose of 240 mg/m2 orally once daily to patients who were younger than 12 years.

The overall response rate through week 25 was 60% (confidence interval, 95%, 44%-74%). The median duration of response was 5.3 months (95% CI, 2.8-8.8).

The safety profile was consistent with the established profile for ibrutinib. Observed adverse events in pediatric patients were consistent with those observed in adult patients with moderate to severe cGVHD, the companies noted.

The FDA noted that the most common (≥ 20%) adverse reactions, including laboratory abnormalities, were anemia, musculoskeletal pain, pyrexia, diarrhea, pneumonia, abdominal pain, stomatitis, thrombocytopenia, and headache.

Full prescribing information for ibrutinib is available here.

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

Monkeypox cases in the United States continue to be rare in children younger than 15, women, and in individuals older than 60, according to new data released by the Centers for Disease Control and Prevention. Men aged 26-40 make up the highest proportion of cases.

The age distribution of cases is similar to those of sexually transmitted infections, said Monica Gandhi, MD, MPH, associate chief of the division of HIV, infectious diseases, and global medicine at the University of California, San Francisco. It is most common in younger to middle-aged age groups, and less common in children and older individuals. As of Aug. 21, only 17 children younger than 15 have been diagnosed with monkeypox in the United States, and women make up fewer than 1.5% of cases.

“This data should be very reassuring to parents and to children going to back to school,” Dr. Gandhi said in an interview. After 3 months of monitoring the virus, the data suggest that monkeypox is primarily spreading in networks of men who have sex with men (MSM) through sexual activity, “and that isn’t something we worry about with school-spread illness.”

In addition to the reassuring data about children and monkeypox, the CDC released laboratory testing data, a behavioral survey of MSM, patient data on the antiviral medication tecovirimat (TPOXX), and other case demographics and symptoms.

Though the number of positive monkeypox tests have continued to rise, the test-positivity rates have declined over the past month, data show. Since July 16, the positivity rate has dipped from 54% to 23%. This trend is likely because of an increase in testing availability, said Randolph Hubach, PhD, MPH, the director of the Sexual Health Research Lab at Purdue University, West Lafayette, Ind.

“We also saw this with COVID early on with testing: it was really limited to folks who were symptomatic,” he said in an interview . “As testing ramped up in accessibility, you had a lot more negative results, but because testing was more widely available, you were able to capture more positive results.”

The data also show that case numbers continue to grow in the United States, whereas in other countries that identified cases before the United States – Spain, the United Kingdom, and France, for example – cases have been leveling off, noted Dr. Gandhi.

The CDC also shared responses from a survey of gay, bisexual, and other MSM conducted from Aug. 5-15, about how they have changed their sexual behaviors in response to the monkeypox outbreak. Half of respondents reported reduced one-time sexual encounters, 49% reported reducing sex with partners met on dating apps or at sex venues, and 48% reported reducing their number of sex partners. These responses are “heartening to see,” Dr. Gandhi said, and shows that individuals are taking proactive steps to reduce their potential exposure risk to monkeypox.

More detailed demographic data showed that Black, Hispanic, or Latinx individuals make up an increasing proportion of cases in the United States. In May, 71% of people with reported monkeypox infection were White and 29% were Black. For the week of August 8-14, about a third (31%) of monkeypox cases were in White people, 32% were in Hispanic or Latinx people, and 33% were in Black people.

The most common symptoms of monkeypox were rash (98.6%), malaise (72.7%), fever (72.1%), and chills (68.9%). Rectal pain was reported in 43.9% of patients, and 25% had rectal bleeding.

The CDC also released information on 288 patients with monkeypox treated with TPOXX under compassionate use. The median age of patients was 37 and 98.9% were male. About 40% of recipients were White, 35% were Hispanic, and about 16% were Black. This information does not include every patient treated with TPOXX, the agency said, as providers can begin treatment before submitting paperwork. As of Aug. 18, the CDC had received 400 patient intake forms for TPOXX, according to its website.

The agency has yet to release data on vaccination rates, which Dr. Hubach is eager to see. Demographic information on who is receiving vaccinations, and where, can illuminate issues with access as vaccine eligibility continues to expand. “Vaccination is probably going to be the largest tool within our toolbox to try to inhibit disease acquisition and spread,” he said.

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

Monkeypox cases in the United States continue to be rare in children younger than 15, women, and in individuals older than 60, according to new data released by the Centers for Disease Control and Prevention. Men aged 26-40 make up the highest proportion of cases.

The age distribution of cases is similar to those of sexually transmitted infections, said Monica Gandhi, MD, MPH, associate chief of the division of HIV, infectious diseases, and global medicine at the University of California, San Francisco. It is most common in younger to middle-aged age groups, and less common in children and older individuals. As of Aug. 21, only 17 children younger than 15 have been diagnosed with monkeypox in the United States, and women make up fewer than 1.5% of cases.

“This data should be very reassuring to parents and to children going to back to school,” Dr. Gandhi said in an interview. After 3 months of monitoring the virus, the data suggest that monkeypox is primarily spreading in networks of men who have sex with men (MSM) through sexual activity, “and that isn’t something we worry about with school-spread illness.”

In addition to the reassuring data about children and monkeypox, the CDC released laboratory testing data, a behavioral survey of MSM, patient data on the antiviral medication tecovirimat (TPOXX), and other case demographics and symptoms.

Though the number of positive monkeypox tests have continued to rise, the test-positivity rates have declined over the past month, data show. Since July 16, the positivity rate has dipped from 54% to 23%. This trend is likely because of an increase in testing availability, said Randolph Hubach, PhD, MPH, the director of the Sexual Health Research Lab at Purdue University, West Lafayette, Ind.

“We also saw this with COVID early on with testing: it was really limited to folks who were symptomatic,” he said in an interview . “As testing ramped up in accessibility, you had a lot more negative results, but because testing was more widely available, you were able to capture more positive results.”

The data also show that case numbers continue to grow in the United States, whereas in other countries that identified cases before the United States – Spain, the United Kingdom, and France, for example – cases have been leveling off, noted Dr. Gandhi.

The CDC also shared responses from a survey of gay, bisexual, and other MSM conducted from Aug. 5-15, about how they have changed their sexual behaviors in response to the monkeypox outbreak. Half of respondents reported reduced one-time sexual encounters, 49% reported reducing sex with partners met on dating apps or at sex venues, and 48% reported reducing their number of sex partners. These responses are “heartening to see,” Dr. Gandhi said, and shows that individuals are taking proactive steps to reduce their potential exposure risk to monkeypox.

More detailed demographic data showed that Black, Hispanic, or Latinx individuals make up an increasing proportion of cases in the United States. In May, 71% of people with reported monkeypox infection were White and 29% were Black. For the week of August 8-14, about a third (31%) of monkeypox cases were in White people, 32% were in Hispanic or Latinx people, and 33% were in Black people.

The most common symptoms of monkeypox were rash (98.6%), malaise (72.7%), fever (72.1%), and chills (68.9%). Rectal pain was reported in 43.9% of patients, and 25% had rectal bleeding.

The CDC also released information on 288 patients with monkeypox treated with TPOXX under compassionate use. The median age of patients was 37 and 98.9% were male. About 40% of recipients were White, 35% were Hispanic, and about 16% were Black. This information does not include every patient treated with TPOXX, the agency said, as providers can begin treatment before submitting paperwork. As of Aug. 18, the CDC had received 400 patient intake forms for TPOXX, according to its website.

The agency has yet to release data on vaccination rates, which Dr. Hubach is eager to see. Demographic information on who is receiving vaccinations, and where, can illuminate issues with access as vaccine eligibility continues to expand. “Vaccination is probably going to be the largest tool within our toolbox to try to inhibit disease acquisition and spread,” he said.

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

Monkeypox cases in the United States continue to be rare in children younger than 15, women, and in individuals older than 60, according to new data released by the Centers for Disease Control and Prevention. Men aged 26-40 make up the highest proportion of cases.

The age distribution of cases is similar to those of sexually transmitted infections, said Monica Gandhi, MD, MPH, associate chief of the division of HIV, infectious diseases, and global medicine at the University of California, San Francisco. It is most common in younger to middle-aged age groups, and less common in children and older individuals. As of Aug. 21, only 17 children younger than 15 have been diagnosed with monkeypox in the United States, and women make up fewer than 1.5% of cases.

“This data should be very reassuring to parents and to children going to back to school,” Dr. Gandhi said in an interview. After 3 months of monitoring the virus, the data suggest that monkeypox is primarily spreading in networks of men who have sex with men (MSM) through sexual activity, “and that isn’t something we worry about with school-spread illness.”

In addition to the reassuring data about children and monkeypox, the CDC released laboratory testing data, a behavioral survey of MSM, patient data on the antiviral medication tecovirimat (TPOXX), and other case demographics and symptoms.

Though the number of positive monkeypox tests have continued to rise, the test-positivity rates have declined over the past month, data show. Since July 16, the positivity rate has dipped from 54% to 23%. This trend is likely because of an increase in testing availability, said Randolph Hubach, PhD, MPH, the director of the Sexual Health Research Lab at Purdue University, West Lafayette, Ind.

“We also saw this with COVID early on with testing: it was really limited to folks who were symptomatic,” he said in an interview . “As testing ramped up in accessibility, you had a lot more negative results, but because testing was more widely available, you were able to capture more positive results.”

The data also show that case numbers continue to grow in the United States, whereas in other countries that identified cases before the United States – Spain, the United Kingdom, and France, for example – cases have been leveling off, noted Dr. Gandhi.

The CDC also shared responses from a survey of gay, bisexual, and other MSM conducted from Aug. 5-15, about how they have changed their sexual behaviors in response to the monkeypox outbreak. Half of respondents reported reduced one-time sexual encounters, 49% reported reducing sex with partners met on dating apps or at sex venues, and 48% reported reducing their number of sex partners. These responses are “heartening to see,” Dr. Gandhi said, and shows that individuals are taking proactive steps to reduce their potential exposure risk to monkeypox.

More detailed demographic data showed that Black, Hispanic, or Latinx individuals make up an increasing proportion of cases in the United States. In May, 71% of people with reported monkeypox infection were White and 29% were Black. For the week of August 8-14, about a third (31%) of monkeypox cases were in White people, 32% were in Hispanic or Latinx people, and 33% were in Black people.

The most common symptoms of monkeypox were rash (98.6%), malaise (72.7%), fever (72.1%), and chills (68.9%). Rectal pain was reported in 43.9% of patients, and 25% had rectal bleeding.

The CDC also released information on 288 patients with monkeypox treated with TPOXX under compassionate use. The median age of patients was 37 and 98.9% were male. About 40% of recipients were White, 35% were Hispanic, and about 16% were Black. This information does not include every patient treated with TPOXX, the agency said, as providers can begin treatment before submitting paperwork. As of Aug. 18, the CDC had received 400 patient intake forms for TPOXX, according to its website.

The agency has yet to release data on vaccination rates, which Dr. Hubach is eager to see. Demographic information on who is receiving vaccinations, and where, can illuminate issues with access as vaccine eligibility continues to expand. “Vaccination is probably going to be the largest tool within our toolbox to try to inhibit disease acquisition and spread,” he said.

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

Adherence to well-child visits in the United States increased overall over a 10-year period, but a gap of up to 20% persisted between the highest and lowest adherence groups, reflecting disparities by race and ethnicity, poverty level, geography, and insurance status.

Well-child visits are recommended to provide children with preventive health and development services, ensure immunizations, and allow parents to discuss health concerns, wrote Salam Abdus, PhD, and Thomas M. Selden, PhD, of the Agency for Healthcare Research and Quality, Rockville, Md.

“We know from prior studies that as of 2008, well-child visits were trending upward, but often fell short of recommendations among key socioeconomic groups,” they wrote.

To examine recent trends in well-child visits, the researchers conducted a cross-sectional study of data from the Medical Expenditure Panel Survey (MEPS) on children aged 0 to 18 years. The findings were published in JAMA Pediatrics.

The study population included 19,018 children in 2006 and 2007 and 17,533 children in 2016 and 2017.

Adherence was defined as the ratio of reported well-child visits divided by the recommended number of visits in a calendar year.

Overall, the mean adherence increased from 47.9% in 2006-2007 to 62.3% in 2016-2017.

However, significant gaps persisted across race and ethnicity. Notably, adherence in the Hispanic population increased by nearly 22% between the study dates, compared to a 15.3% increase among White non-Hispanic children. However, Hispanic children still trailed White children overall in 2016-2017 (58% vs. 67.8%).

The smallest increase in adherence occurred among Black non-Hispanic children (5.6%) which further widened the gap between Black and White non-Hispanic children in 2016-2017 (52.5% vs. 67.8%).

Adherence rates increased similarly for children with public and private insurance (15.5% and 13.9%, respectively), but the adherence rates for uninsured children remained stable. Adherence in 2016-2017 for children with private, public, and no insurance were 66.3%, 58.7%, and 31.1%.

Also, despite overall increases in adherence across regions, a gap of more than 20% separated the region with the highest adherence (Northeast) from the lowest (West) in both the 2006-2007 and 2016-2017 periods (69.3% vs. 38.4%, and 79.3% vs. 55.2%, respectively).

The findings show an increase in well-child visits that spanned a time period of increased recommendations, economic changes, and the impact of the Affordable Care Act, but unaddressed disparities remain, the researchers noted.

Reducing disparities and improving adherence, “will require the combined efforts of researchers, policymakers, and clinicians to improve our understanding of adherence, to implement policies improving access to care, and to increase health care professional engagement with disadvantaged communities,” they concluded.
 

Overall increases are encouraging, but barriers need attention

“Demographic data are critical to determine which groups of children need the most support for recommended well child care,” Susan Boulter, MD, of the Geisel School of Medicine at Dartmouth, Hanover, N.H., said in an interview. In the current study, “it was encouraging to see how either public or private insurance significantly increased the percentage of children receiving well child care,” she said.

The level of increased adherence to AAP-recommended guidelines for well-child visits was surprising, said Dr. Boulter. The overall increase is likely attributable in part to the increased coverage for well-child visits in the wake of the Affordable Care Act, as the study authors mention, she said.

“The gains experienced by Hispanic families were especially encouraging,” she added.

However, ongoing barriers to well-child care include “lack of adequate provider numbers and mix, transportation difficulties for patients, and lack of child care and time away from work for parents so they can complete the recommended well child visit schedule,” Dr. Boulter noted. “Provider schedules and locations of care should be improved so families would have easier access. Also, social media should have more positive well-child messages to counteract the negative messaging.”

More research is needed to examine the impact of COVID-19 on well-child visits, Dr. Boulter emphasized. “Most likely, the percentages in all groups will have changed since COVID-19 has impacted office practices,” she said. “Anxiety about COVID-19 transmissibility in the pediatric office decreased routine office visits, and skepticism about vaccines, including vaccine refusal, has significantly changed the percentage of children who have received the AAP recommended vaccines,” she explained. Ideally, the study authors will review the MEPS data again to examine changes since the COVID-19 pandemic began, she told this news organization.

The study received no outside funding. The researchers had no financial conflicts to disclose. Dr. Boulter had no financial conflicts to disclose and serves on the editorial advisory board of Pediatric News.

Adherence to well-child visits in the United States increased overall over a 10-year period, but a gap of up to 20% persisted between the highest and lowest adherence groups, reflecting disparities by race and ethnicity, poverty level, geography, and insurance status.

Well-child visits are recommended to provide children with preventive health and development services, ensure immunizations, and allow parents to discuss health concerns, wrote Salam Abdus, PhD, and Thomas M. Selden, PhD, of the Agency for Healthcare Research and Quality, Rockville, Md.

“We know from prior studies that as of 2008, well-child visits were trending upward, but often fell short of recommendations among key socioeconomic groups,” they wrote.

To examine recent trends in well-child visits, the researchers conducted a cross-sectional study of data from the Medical Expenditure Panel Survey (MEPS) on children aged 0 to 18 years. The findings were published in JAMA Pediatrics.

The study population included 19,018 children in 2006 and 2007 and 17,533 children in 2016 and 2017.

Adherence was defined as the ratio of reported well-child visits divided by the recommended number of visits in a calendar year.

Overall, the mean adherence increased from 47.9% in 2006-2007 to 62.3% in 2016-2017.

However, significant gaps persisted across race and ethnicity. Notably, adherence in the Hispanic population increased by nearly 22% between the study dates, compared to a 15.3% increase among White non-Hispanic children. However, Hispanic children still trailed White children overall in 2016-2017 (58% vs. 67.8%).

The smallest increase in adherence occurred among Black non-Hispanic children (5.6%) which further widened the gap between Black and White non-Hispanic children in 2016-2017 (52.5% vs. 67.8%).

Adherence rates increased similarly for children with public and private insurance (15.5% and 13.9%, respectively), but the adherence rates for uninsured children remained stable. Adherence in 2016-2017 for children with private, public, and no insurance were 66.3%, 58.7%, and 31.1%.

Also, despite overall increases in adherence across regions, a gap of more than 20% separated the region with the highest adherence (Northeast) from the lowest (West) in both the 2006-2007 and 2016-2017 periods (69.3% vs. 38.4%, and 79.3% vs. 55.2%, respectively).

The findings show an increase in well-child visits that spanned a time period of increased recommendations, economic changes, and the impact of the Affordable Care Act, but unaddressed disparities remain, the researchers noted.

Reducing disparities and improving adherence, “will require the combined efforts of researchers, policymakers, and clinicians to improve our understanding of adherence, to implement policies improving access to care, and to increase health care professional engagement with disadvantaged communities,” they concluded.
 

Overall increases are encouraging, but barriers need attention

“Demographic data are critical to determine which groups of children need the most support for recommended well child care,” Susan Boulter, MD, of the Geisel School of Medicine at Dartmouth, Hanover, N.H., said in an interview. In the current study, “it was encouraging to see how either public or private insurance significantly increased the percentage of children receiving well child care,” she said.

The level of increased adherence to AAP-recommended guidelines for well-child visits was surprising, said Dr. Boulter. The overall increase is likely attributable in part to the increased coverage for well-child visits in the wake of the Affordable Care Act, as the study authors mention, she said.

“The gains experienced by Hispanic families were especially encouraging,” she added.

However, ongoing barriers to well-child care include “lack of adequate provider numbers and mix, transportation difficulties for patients, and lack of child care and time away from work for parents so they can complete the recommended well child visit schedule,” Dr. Boulter noted. “Provider schedules and locations of care should be improved so families would have easier access. Also, social media should have more positive well-child messages to counteract the negative messaging.”

More research is needed to examine the impact of COVID-19 on well-child visits, Dr. Boulter emphasized. “Most likely, the percentages in all groups will have changed since COVID-19 has impacted office practices,” she said. “Anxiety about COVID-19 transmissibility in the pediatric office decreased routine office visits, and skepticism about vaccines, including vaccine refusal, has significantly changed the percentage of children who have received the AAP recommended vaccines,” she explained. Ideally, the study authors will review the MEPS data again to examine changes since the COVID-19 pandemic began, she told this news organization.

The study received no outside funding. The researchers had no financial conflicts to disclose. Dr. Boulter had no financial conflicts to disclose and serves on the editorial advisory board of Pediatric News.

Adherence to well-child visits in the United States increased overall over a 10-year period, but a gap of up to 20% persisted between the highest and lowest adherence groups, reflecting disparities by race and ethnicity, poverty level, geography, and insurance status.

Well-child visits are recommended to provide children with preventive health and development services, ensure immunizations, and allow parents to discuss health concerns, wrote Salam Abdus, PhD, and Thomas M. Selden, PhD, of the Agency for Healthcare Research and Quality, Rockville, Md.

“We know from prior studies that as of 2008, well-child visits were trending upward, but often fell short of recommendations among key socioeconomic groups,” they wrote.

To examine recent trends in well-child visits, the researchers conducted a cross-sectional study of data from the Medical Expenditure Panel Survey (MEPS) on children aged 0 to 18 years. The findings were published in JAMA Pediatrics.

The study population included 19,018 children in 2006 and 2007 and 17,533 children in 2016 and 2017.

Adherence was defined as the ratio of reported well-child visits divided by the recommended number of visits in a calendar year.

Overall, the mean adherence increased from 47.9% in 2006-2007 to 62.3% in 2016-2017.

However, significant gaps persisted across race and ethnicity. Notably, adherence in the Hispanic population increased by nearly 22% between the study dates, compared to a 15.3% increase among White non-Hispanic children. However, Hispanic children still trailed White children overall in 2016-2017 (58% vs. 67.8%).

The smallest increase in adherence occurred among Black non-Hispanic children (5.6%) which further widened the gap between Black and White non-Hispanic children in 2016-2017 (52.5% vs. 67.8%).

Adherence rates increased similarly for children with public and private insurance (15.5% and 13.9%, respectively), but the adherence rates for uninsured children remained stable. Adherence in 2016-2017 for children with private, public, and no insurance were 66.3%, 58.7%, and 31.1%.

Also, despite overall increases in adherence across regions, a gap of more than 20% separated the region with the highest adherence (Northeast) from the lowest (West) in both the 2006-2007 and 2016-2017 periods (69.3% vs. 38.4%, and 79.3% vs. 55.2%, respectively).

The findings show an increase in well-child visits that spanned a time period of increased recommendations, economic changes, and the impact of the Affordable Care Act, but unaddressed disparities remain, the researchers noted.

Reducing disparities and improving adherence, “will require the combined efforts of researchers, policymakers, and clinicians to improve our understanding of adherence, to implement policies improving access to care, and to increase health care professional engagement with disadvantaged communities,” they concluded.
 

Overall increases are encouraging, but barriers need attention

“Demographic data are critical to determine which groups of children need the most support for recommended well child care,” Susan Boulter, MD, of the Geisel School of Medicine at Dartmouth, Hanover, N.H., said in an interview. In the current study, “it was encouraging to see how either public or private insurance significantly increased the percentage of children receiving well child care,” she said.

The level of increased adherence to AAP-recommended guidelines for well-child visits was surprising, said Dr. Boulter. The overall increase is likely attributable in part to the increased coverage for well-child visits in the wake of the Affordable Care Act, as the study authors mention, she said.

“The gains experienced by Hispanic families were especially encouraging,” she added.

However, ongoing barriers to well-child care include “lack of adequate provider numbers and mix, transportation difficulties for patients, and lack of child care and time away from work for parents so they can complete the recommended well child visit schedule,” Dr. Boulter noted. “Provider schedules and locations of care should be improved so families would have easier access. Also, social media should have more positive well-child messages to counteract the negative messaging.”

More research is needed to examine the impact of COVID-19 on well-child visits, Dr. Boulter emphasized. “Most likely, the percentages in all groups will have changed since COVID-19 has impacted office practices,” she said. “Anxiety about COVID-19 transmissibility in the pediatric office decreased routine office visits, and skepticism about vaccines, including vaccine refusal, has significantly changed the percentage of children who have received the AAP recommended vaccines,” she explained. Ideally, the study authors will review the MEPS data again to examine changes since the COVID-19 pandemic began, she told this news organization.

The study received no outside funding. The researchers had no financial conflicts to disclose. Dr. Boulter had no financial conflicts to disclose and serves on the editorial advisory board of Pediatric News.

It’s back to school time, and some may be wondering what the current availability of vaccines may mean and the effects of the ever-changing COVID-19 guidelines on their children’s education and day-to-day experiences as students this year.

Unlike last school year, there are now vaccines available for all over the age of 6 months, and home rapid antigen tests are more readily available. Additionally, many have now been exposed either by infection or vaccination to the virus.

The CDC has removed the recommendations for maintaining cohorts in the K-12 population. This changing landscape along with differing levels of personal risk make it challenging to counsel families about what to expect in terms of COVID this year.

The best defense that we currently have against COVID is the vaccine. Although it seems that many are susceptible to the virus despite the vaccine, those who have been vaccinated are less susceptible to serious disease, including young children.

As older children may be heading to college, it is important

to encourage them to isolate when they have symptoms, even when they test negative for COVID as we would all like to avoid being sick in general.

Additionally, they should pay attention to the COVID risk level in their area and wear masks, particularly when indoors, as the levels increase. College students should have a plan for where they can isolate when not feeling well. If anyone does test positive for COVID, they should follow the most recent quarantine guidelines, including wearing a well fitted mask when they do begin returning to activities.
 

Monkeypox

We now have a new health concern for this school year.

Monkeypox has come onto the scene with information changing as rapidly as information previously did for COVID. With this virus, we must particularly counsel those heading away to college to be careful to limit their exposure to this disease.

Dormitories and other congregate settings are high-risk locations for the spread of monkeypox. Particularly, students headed to stay in dormitories should be counseled about avoiding:

• sexual activity with those with lesions consistent with monkeypox;
• sharing eating and drinking utensils; and
• sleeping in the same bed as or sharing bedding or towels with anyone with a diagnosis of or lesions consistent with monkeypox.

Additionally, as with prevention of all infections, it is important to frequently wash hands or use alcohol-based sanitizer before eating, and avoid touching the face after using the restroom.

Guidance for those eligible for vaccines against monkeypox seems to be quickly changing as well.

At the time of this article, CDC guidance recommends the vaccine against monkeypox for:

• those considered to be at high risk for it, including those identified by public health officials as a contact of someone with monkeypox;
• those who are aware that a sexual partner had a diagnosis of monkeypox within the past 2 weeks;
• those with multiple sex partners in the past 2 weeks in an area with known monkeypox; and
• those whose jobs may expose them to monkeypox.

Currently, the CDC recommends the vaccine JYNNEOS, a two-dose vaccine that reaches maximum protection after fourteen days. Ultimately, guidance is likely to continue to quickly change for both COVID-19 and Monkeypox throughout the fall. It is possible that new vaccinations will become available, and families and physicians alike will have many questions.

Primary care offices should ensure that someone is keeping up to date with the latest guidance to share with the office so that physicians may share accurate information with their patients.

Families should be counseled that we anticipate information about monkeypox, particularly related to vaccinations, to continue to change, as it has during all stages of the COVID pandemic.

As always, patients should be reminded to continue regular routine vaccinations, including the annual influenza vaccine.

Dr. Wheat is a family physician at Erie Family Health Center and program director of Northwestern University’s McGaw Family Medicine residency program, both in Chicago. Dr. Wheat serves on the editorial advisory board of Family Practice News. You can contact her at fpnews@mdedge.com.

It’s back to school time, and some may be wondering what the current availability of vaccines may mean and the effects of the ever-changing COVID-19 guidelines on their children’s education and day-to-day experiences as students this year.

Unlike last school year, there are now vaccines available for all over the age of 6 months, and home rapid antigen tests are more readily available. Additionally, many have now been exposed either by infection or vaccination to the virus.

The CDC has removed the recommendations for maintaining cohorts in the K-12 population. This changing landscape along with differing levels of personal risk make it challenging to counsel families about what to expect in terms of COVID this year.

The best defense that we currently have against COVID is the vaccine. Although it seems that many are susceptible to the virus despite the vaccine, those who have been vaccinated are less susceptible to serious disease, including young children.

As older children may be heading to college, it is important

to encourage them to isolate when they have symptoms, even when they test negative for COVID as we would all like to avoid being sick in general.

Additionally, they should pay attention to the COVID risk level in their area and wear masks, particularly when indoors, as the levels increase. College students should have a plan for where they can isolate when not feeling well. If anyone does test positive for COVID, they should follow the most recent quarantine guidelines, including wearing a well fitted mask when they do begin returning to activities.
 

Monkeypox

We now have a new health concern for this school year.

Monkeypox has come onto the scene with information changing as rapidly as information previously did for COVID. With this virus, we must particularly counsel those heading away to college to be careful to limit their exposure to this disease.

Dormitories and other congregate settings are high-risk locations for the spread of monkeypox. Particularly, students headed to stay in dormitories should be counseled about avoiding:

• sexual activity with those with lesions consistent with monkeypox;
• sharing eating and drinking utensils; and
• sleeping in the same bed as or sharing bedding or towels with anyone with a diagnosis of or lesions consistent with monkeypox.

Additionally, as with prevention of all infections, it is important to frequently wash hands or use alcohol-based sanitizer before eating, and avoid touching the face after using the restroom.

Guidance for those eligible for vaccines against monkeypox seems to be quickly changing as well.

At the time of this article, CDC guidance recommends the vaccine against monkeypox for:

• those considered to be at high risk for it, including those identified by public health officials as a contact of someone with monkeypox;
• those who are aware that a sexual partner had a diagnosis of monkeypox within the past 2 weeks;
• those with multiple sex partners in the past 2 weeks in an area with known monkeypox; and
• those whose jobs may expose them to monkeypox.

Currently, the CDC recommends the vaccine JYNNEOS, a two-dose vaccine that reaches maximum protection after fourteen days. Ultimately, guidance is likely to continue to quickly change for both COVID-19 and Monkeypox throughout the fall. It is possible that new vaccinations will become available, and families and physicians alike will have many questions.

Primary care offices should ensure that someone is keeping up to date with the latest guidance to share with the office so that physicians may share accurate information with their patients.

Families should be counseled that we anticipate information about monkeypox, particularly related to vaccinations, to continue to change, as it has during all stages of the COVID pandemic.

As always, patients should be reminded to continue regular routine vaccinations, including the annual influenza vaccine.

Dr. Wheat is a family physician at Erie Family Health Center and program director of Northwestern University’s McGaw Family Medicine residency program, both in Chicago. Dr. Wheat serves on the editorial advisory board of Family Practice News. You can contact her at fpnews@mdedge.com.

It’s back to school time, and some may be wondering what the current availability of vaccines may mean and the effects of the ever-changing COVID-19 guidelines on their children’s education and day-to-day experiences as students this year.

Unlike last school year, there are now vaccines available for all over the age of 6 months, and home rapid antigen tests are more readily available. Additionally, many have now been exposed either by infection or vaccination to the virus.

The CDC has removed the recommendations for maintaining cohorts in the K-12 population. This changing landscape along with differing levels of personal risk make it challenging to counsel families about what to expect in terms of COVID this year.

The best defense that we currently have against COVID is the vaccine. Although it seems that many are susceptible to the virus despite the vaccine, those who have been vaccinated are less susceptible to serious disease, including young children.

As older children may be heading to college, it is important

to encourage them to isolate when they have symptoms, even when they test negative for COVID as we would all like to avoid being sick in general.

Additionally, they should pay attention to the COVID risk level in their area and wear masks, particularly when indoors, as the levels increase. College students should have a plan for where they can isolate when not feeling well. If anyone does test positive for COVID, they should follow the most recent quarantine guidelines, including wearing a well fitted mask when they do begin returning to activities.
 

Monkeypox

We now have a new health concern for this school year.

Monkeypox has come onto the scene with information changing as rapidly as information previously did for COVID. With this virus, we must particularly counsel those heading away to college to be careful to limit their exposure to this disease.

Dormitories and other congregate settings are high-risk locations for the spread of monkeypox. Particularly, students headed to stay in dormitories should be counseled about avoiding:

• sexual activity with those with lesions consistent with monkeypox;
• sharing eating and drinking utensils; and
• sleeping in the same bed as or sharing bedding or towels with anyone with a diagnosis of or lesions consistent with monkeypox.

Additionally, as with prevention of all infections, it is important to frequently wash hands or use alcohol-based sanitizer before eating, and avoid touching the face after using the restroom.

Guidance for those eligible for vaccines against monkeypox seems to be quickly changing as well.

At the time of this article, CDC guidance recommends the vaccine against monkeypox for:

• those considered to be at high risk for it, including those identified by public health officials as a contact of someone with monkeypox;
• those who are aware that a sexual partner had a diagnosis of monkeypox within the past 2 weeks;
• those with multiple sex partners in the past 2 weeks in an area with known monkeypox; and
• those whose jobs may expose them to monkeypox.

Currently, the CDC recommends the vaccine JYNNEOS, a two-dose vaccine that reaches maximum protection after fourteen days. Ultimately, guidance is likely to continue to quickly change for both COVID-19 and Monkeypox throughout the fall. It is possible that new vaccinations will become available, and families and physicians alike will have many questions.

Primary care offices should ensure that someone is keeping up to date with the latest guidance to share with the office so that physicians may share accurate information with their patients.

Families should be counseled that we anticipate information about monkeypox, particularly related to vaccinations, to continue to change, as it has during all stages of the COVID pandemic.

As always, patients should be reminded to continue regular routine vaccinations, including the annual influenza vaccine.

Dr. Wheat is a family physician at Erie Family Health Center and program director of Northwestern University’s McGaw Family Medicine residency program, both in Chicago. Dr. Wheat serves on the editorial advisory board of Family Practice News. You can contact her at fpnews@mdedge.com.

The Food and Drug Administration has approved use of the Omnipod 5 automated insulin delivery system (Insulet Corp) for children aged 2 years and older with type 1 diabetes, the company announced on Aug. 22.

Omnipod 5 was originally cleared for use in individuals age 6 and older in Jan. 2022, as previously reported by this news organization. It is the third semi-automated closed-loop system approved in the United States but the first that is tubing-free. It integrates with the Dexcom G6 continuous glucose monitor system and a compatible smartphone to automatically adjust insulin and protect against high and low glucose levels.

“We received tremendous first-hand reports of how Omnipod 5 made diabetes management easier for our pivotal trial participants, and the clinical data demonstrated impressive glycemic improvements as well,” Trang Ly, MBBS, PhD, senior vice president and medical director at Insulet, said in a news release. “This expanded indication for younger children gives us great pride, knowing we can further ease the burden of glucose management for these children and their caregivers with our simple to use, elegant, automated insulin delivery system.”

In a recent clinical trial in very young children (age 2-5.9 years) with type 1 diabetes, Jennifer L. Sherr, MD, PhD, and colleagues found that the Omnipod 5 lowered A1c by 0.55 percentage points and reduced time in hypoglycemia (< 70 mg/dL) by 0.27%. According to their findings, published in Diabetes Care, time spent in target glucose range (70-180 mg/dL) increased by 11%, or by 2.6 hours more per day, in children in the study.

According to the release, the Omnipod 5 can now be prescribed to patients with insurance coverage. Patients can access their prescription through the pharmacy.

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

The Food and Drug Administration has approved use of the Omnipod 5 automated insulin delivery system (Insulet Corp) for children aged 2 years and older with type 1 diabetes, the company announced on Aug. 22.

Omnipod 5 was originally cleared for use in individuals age 6 and older in Jan. 2022, as previously reported by this news organization. It is the third semi-automated closed-loop system approved in the United States but the first that is tubing-free. It integrates with the Dexcom G6 continuous glucose monitor system and a compatible smartphone to automatically adjust insulin and protect against high and low glucose levels.

“We received tremendous first-hand reports of how Omnipod 5 made diabetes management easier for our pivotal trial participants, and the clinical data demonstrated impressive glycemic improvements as well,” Trang Ly, MBBS, PhD, senior vice president and medical director at Insulet, said in a news release. “This expanded indication for younger children gives us great pride, knowing we can further ease the burden of glucose management for these children and their caregivers with our simple to use, elegant, automated insulin delivery system.”

In a recent clinical trial in very young children (age 2-5.9 years) with type 1 diabetes, Jennifer L. Sherr, MD, PhD, and colleagues found that the Omnipod 5 lowered A1c by 0.55 percentage points and reduced time in hypoglycemia (< 70 mg/dL) by 0.27%. According to their findings, published in Diabetes Care, time spent in target glucose range (70-180 mg/dL) increased by 11%, or by 2.6 hours more per day, in children in the study.

According to the release, the Omnipod 5 can now be prescribed to patients with insurance coverage. Patients can access their prescription through the pharmacy.

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

The Food and Drug Administration has approved use of the Omnipod 5 automated insulin delivery system (Insulet Corp) for children aged 2 years and older with type 1 diabetes, the company announced on Aug. 22.

Omnipod 5 was originally cleared for use in individuals age 6 and older in Jan. 2022, as previously reported by this news organization. It is the third semi-automated closed-loop system approved in the United States but the first that is tubing-free. It integrates with the Dexcom G6 continuous glucose monitor system and a compatible smartphone to automatically adjust insulin and protect against high and low glucose levels.

“We received tremendous first-hand reports of how Omnipod 5 made diabetes management easier for our pivotal trial participants, and the clinical data demonstrated impressive glycemic improvements as well,” Trang Ly, MBBS, PhD, senior vice president and medical director at Insulet, said in a news release. “This expanded indication for younger children gives us great pride, knowing we can further ease the burden of glucose management for these children and their caregivers with our simple to use, elegant, automated insulin delivery system.”

In a recent clinical trial in very young children (age 2-5.9 years) with type 1 diabetes, Jennifer L. Sherr, MD, PhD, and colleagues found that the Omnipod 5 lowered A1c by 0.55 percentage points and reduced time in hypoglycemia (< 70 mg/dL) by 0.27%. According to their findings, published in Diabetes Care, time spent in target glucose range (70-180 mg/dL) increased by 11%, or by 2.6 hours more per day, in children in the study.

According to the release, the Omnipod 5 can now be prescribed to patients with insurance coverage. Patients can access their prescription through the pharmacy.

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

Researchers have identified 72 genes very strongly linked to autism spectrum disorders and more than 250 other genes with a strong link to ASD, according to a study published in Nature Genetics. The findings, based on analysis of more than 150,000 people’s genetics, arose from a collaboration of five research groups whose work included comparisons of ASD cohorts with separate cohorts of individuals with developmental delay or schizophrenia.

“We know that many genes, when mutated, contribute to autism,” and this study brought together “multiple types of mutations in a wide array of samples to get a much richer sense of the genes and genetic architecture involved in autism and other neurodevelopmental conditions,” co–senior author Joseph D. Buxbaum, PhD, director of the Seaver Autism Center for Research and Treatment at Mount Sinai and a professor at the Icahn School of Medicine at Mount Sinai, both in New York, said in a prepared statement. “This is significant in that we now have more insights as to the biology of the brain changes that underlie autism and more potential targets for treatment.”

Glen Elliott, PhD, MD, a clinical professor of psychiatry at Stanford (Calif.) University who was not involved in the study, said the paper is important paper for informing clinicians of where the basic research is headed. “We’re still in for a long road” before it bears fruit in terms of therapeutics. The value of studies like these, that investigate which genes are most associated with ASD, is that they may lead toward understanding the pathways in the brain that give rise to certain symptoms of ASD, which can then become therapeutic targets, Dr. Elliott said.
 

Investigating large cohorts

The researchers analyzed genetic exome sequencing data from 33 ASD cohorts with a total of 63,237 people and then compared these data with another cohort of people with developmental delay and a cohort of people with schizophrenia. The combined ASD cohorts included 15,036 individuals with ASD, 28,522 parents, and 5,492 unaffected siblings. The remaining participants were 5,591 people with ASD and 8,597 matched controls from case control studies.

In the ASD cohorts, the researchers identified 72 genes that were associated with ASD. De novo variants were eight times more likely in cases (4%) than in controls (0.5%). Ten genes occurred at least twice in ASD cases but never occurred in unaffected siblings.

Then the researchers integrated these ASD genetic data with a cohort of 91,605 people that included 31,058 people with developmental delay and their parents. Substantial overlap with gene mutations existed between these two cohorts: 70.1% of the genes related to developmental delay appeared linked to risk for ASD, and 86.6% of genes associated with ASD risk also had associations with developmental delay. Overall, the researchers identified 373 genes strongly associated with ASD and/or developmental delay and 664 genes with a likely association.

“Isolating genes that exert a greater effect on ASD than they do on other developmental delays has remained challenging due to the frequent comorbidity of these phenotypes,” wrote lead author Jack M. Fu, of Massachusetts General Hospital and Harvard Medical School, both in Boston, and colleagues. “Still, an estimated 13.4% of the transmission and de novo association–ASD genes show little evidence for association in the developmental delay cohort.”
 

ASD, developmental delay, and schizophrenia

When the researchers compared the cells where the genetic mutations occurred in fetal brains, they found that genes associated with developmental delay more often occurred in less differentiated cell types – less mature cells in the developmental process. Gene mutations associated with ASD, on the other hand, occurred in more mature cell types, particularly in maturing excitatory neurons and related cells.

”Our results are consistent with developmental delay-predominant genes being expressed earlier in development and in less differentiated cells than ASD-predominant genes,” they wrote.

The researchers also compared the specific gene mutations found in these two cohorts with a previously published set of 244 genes associated with schizophrenia. Of these, 234 genes are among those with a transmission and de novo association to ASD and/or developmental delay. Of the 72 genes linked to ASD, eight appear in the set of genes linked to schizophrenia, and 61 were associated with developmental delay, though these two subsets do not overlap each other much.

“The ASD-schizophrenia overlap was significantly enriched, while the developmental delay-schizophrenia overlap was not,” they reported. ”Together, these data suggest that one subset of ASD risk genes may overlap developmental delay while a different subset overlaps schizophrenia.”
 

Chasing therapy targets by backtracking through genes

The findings are a substantial step forward in understanding the potential genetic contribution to ASD, but they also highlight the challenges of eventually trying to use this information in a clinically meaningful way.

“Given the substantial overlap between the genes implicated in neurodevelopmental disorders writ large and those implicated directly in ASD, disentangling the relative impact of individual genes on neurodevelopment and phenotypic spectra is a daunting yet important challenge,” the researchers wrote. “To identify the key neurobiological features of ASD will likely require convergence of evidence from many ASD genes and studies.”

Dr. Elliott said the biggest takeaway from this study is a better understanding of how the paradigm has shifted away from finding “one gene” for autism or a cure based on genetics and more toward understanding the pathophysiology of symptoms that can point to therapies for better management of the condition.

“Basic researchers have completely changed the strategy for trying to understand the biology of major disorders,” including, in this case, autism, Dr. Elliott said. “The intent is to try to find the underlying systems [in the brain] by backtracking through genes. Meanwhile, given that scientists have made substantial progress in identifying genes that have specific effects on brain development, “the hope is that will mesh with this kind of research, to begin to identify systems that might ultimately be targets for treating.”

The end goal is to be able to offer targeted approaches, based on the pathways causing a symptom, which can be linked backward to a gene.

”So this is not going to offer an immediate cure – it’s probably not going to offer a cure at all – but it may actually lead to much more targeted medications than we currently have for specific types of symptoms within the autism spectrum,” Dr. Elliott said. “What they’re trying to do, ultimately, is to say, when this system is really badly affected because of a genetic abnormality, even though that genetic abnormality is very rare, it leads to these specific kinds of symptoms. If we can find out the neuroregulators underlying that change, then that would be the target, even if that gene were not present.”

The research was funded by the Simons Foundation for Autism Research Initiative, the SPARK project, the National Human Genome Research Institute Home, the National Institute of Mental Health, the National Institute of Child Health and Development, AMED, and the Beatrice and Samuel Seaver Foundation. Five authors reported financial disclosures linked to Desitin, Roche, BioMarin, BrigeBio Pharma, Illumina, Levo Therapeutics, and Microsoft.

Researchers have identified 72 genes very strongly linked to autism spectrum disorders and more than 250 other genes with a strong link to ASD, according to a study published in Nature Genetics. The findings, based on analysis of more than 150,000 people’s genetics, arose from a collaboration of five research groups whose work included comparisons of ASD cohorts with separate cohorts of individuals with developmental delay or schizophrenia.

“We know that many genes, when mutated, contribute to autism,” and this study brought together “multiple types of mutations in a wide array of samples to get a much richer sense of the genes and genetic architecture involved in autism and other neurodevelopmental conditions,” co–senior author Joseph D. Buxbaum, PhD, director of the Seaver Autism Center for Research and Treatment at Mount Sinai and a professor at the Icahn School of Medicine at Mount Sinai, both in New York, said in a prepared statement. “This is significant in that we now have more insights as to the biology of the brain changes that underlie autism and more potential targets for treatment.”

Glen Elliott, PhD, MD, a clinical professor of psychiatry at Stanford (Calif.) University who was not involved in the study, said the paper is important paper for informing clinicians of where the basic research is headed. “We’re still in for a long road” before it bears fruit in terms of therapeutics. The value of studies like these, that investigate which genes are most associated with ASD, is that they may lead toward understanding the pathways in the brain that give rise to certain symptoms of ASD, which can then become therapeutic targets, Dr. Elliott said.
 

Investigating large cohorts

The researchers analyzed genetic exome sequencing data from 33 ASD cohorts with a total of 63,237 people and then compared these data with another cohort of people with developmental delay and a cohort of people with schizophrenia. The combined ASD cohorts included 15,036 individuals with ASD, 28,522 parents, and 5,492 unaffected siblings. The remaining participants were 5,591 people with ASD and 8,597 matched controls from case control studies.

In the ASD cohorts, the researchers identified 72 genes that were associated with ASD. De novo variants were eight times more likely in cases (4%) than in controls (0.5%). Ten genes occurred at least twice in ASD cases but never occurred in unaffected siblings.

Then the researchers integrated these ASD genetic data with a cohort of 91,605 people that included 31,058 people with developmental delay and their parents. Substantial overlap with gene mutations existed between these two cohorts: 70.1% of the genes related to developmental delay appeared linked to risk for ASD, and 86.6% of genes associated with ASD risk also had associations with developmental delay. Overall, the researchers identified 373 genes strongly associated with ASD and/or developmental delay and 664 genes with a likely association.

“Isolating genes that exert a greater effect on ASD than they do on other developmental delays has remained challenging due to the frequent comorbidity of these phenotypes,” wrote lead author Jack M. Fu, of Massachusetts General Hospital and Harvard Medical School, both in Boston, and colleagues. “Still, an estimated 13.4% of the transmission and de novo association–ASD genes show little evidence for association in the developmental delay cohort.”
 

ASD, developmental delay, and schizophrenia

When the researchers compared the cells where the genetic mutations occurred in fetal brains, they found that genes associated with developmental delay more often occurred in less differentiated cell types – less mature cells in the developmental process. Gene mutations associated with ASD, on the other hand, occurred in more mature cell types, particularly in maturing excitatory neurons and related cells.

”Our results are consistent with developmental delay-predominant genes being expressed earlier in development and in less differentiated cells than ASD-predominant genes,” they wrote.

The researchers also compared the specific gene mutations found in these two cohorts with a previously published set of 244 genes associated with schizophrenia. Of these, 234 genes are among those with a transmission and de novo association to ASD and/or developmental delay. Of the 72 genes linked to ASD, eight appear in the set of genes linked to schizophrenia, and 61 were associated with developmental delay, though these two subsets do not overlap each other much.

“The ASD-schizophrenia overlap was significantly enriched, while the developmental delay-schizophrenia overlap was not,” they reported. ”Together, these data suggest that one subset of ASD risk genes may overlap developmental delay while a different subset overlaps schizophrenia.”
 

Chasing therapy targets by backtracking through genes

The findings are a substantial step forward in understanding the potential genetic contribution to ASD, but they also highlight the challenges of eventually trying to use this information in a clinically meaningful way.

“Given the substantial overlap between the genes implicated in neurodevelopmental disorders writ large and those implicated directly in ASD, disentangling the relative impact of individual genes on neurodevelopment and phenotypic spectra is a daunting yet important challenge,” the researchers wrote. “To identify the key neurobiological features of ASD will likely require convergence of evidence from many ASD genes and studies.”

Dr. Elliott said the biggest takeaway from this study is a better understanding of how the paradigm has shifted away from finding “one gene” for autism or a cure based on genetics and more toward understanding the pathophysiology of symptoms that can point to therapies for better management of the condition.

“Basic researchers have completely changed the strategy for trying to understand the biology of major disorders,” including, in this case, autism, Dr. Elliott said. “The intent is to try to find the underlying systems [in the brain] by backtracking through genes. Meanwhile, given that scientists have made substantial progress in identifying genes that have specific effects on brain development, “the hope is that will mesh with this kind of research, to begin to identify systems that might ultimately be targets for treating.”

The end goal is to be able to offer targeted approaches, based on the pathways causing a symptom, which can be linked backward to a gene.

”So this is not going to offer an immediate cure – it’s probably not going to offer a cure at all – but it may actually lead to much more targeted medications than we currently have for specific types of symptoms within the autism spectrum,” Dr. Elliott said. “What they’re trying to do, ultimately, is to say, when this system is really badly affected because of a genetic abnormality, even though that genetic abnormality is very rare, it leads to these specific kinds of symptoms. If we can find out the neuroregulators underlying that change, then that would be the target, even if that gene were not present.”

The research was funded by the Simons Foundation for Autism Research Initiative, the SPARK project, the National Human Genome Research Institute Home, the National Institute of Mental Health, the National Institute of Child Health and Development, AMED, and the Beatrice and Samuel Seaver Foundation. Five authors reported financial disclosures linked to Desitin, Roche, BioMarin, BrigeBio Pharma, Illumina, Levo Therapeutics, and Microsoft.

Researchers have identified 72 genes very strongly linked to autism spectrum disorders and more than 250 other genes with a strong link to ASD, according to a study published in Nature Genetics. The findings, based on analysis of more than 150,000 people’s genetics, arose from a collaboration of five research groups whose work included comparisons of ASD cohorts with separate cohorts of individuals with developmental delay or schizophrenia.

“We know that many genes, when mutated, contribute to autism,” and this study brought together “multiple types of mutations in a wide array of samples to get a much richer sense of the genes and genetic architecture involved in autism and other neurodevelopmental conditions,” co–senior author Joseph D. Buxbaum, PhD, director of the Seaver Autism Center for Research and Treatment at Mount Sinai and a professor at the Icahn School of Medicine at Mount Sinai, both in New York, said in a prepared statement. “This is significant in that we now have more insights as to the biology of the brain changes that underlie autism and more potential targets for treatment.”

Glen Elliott, PhD, MD, a clinical professor of psychiatry at Stanford (Calif.) University who was not involved in the study, said the paper is important paper for informing clinicians of where the basic research is headed. “We’re still in for a long road” before it bears fruit in terms of therapeutics. The value of studies like these, that investigate which genes are most associated with ASD, is that they may lead toward understanding the pathways in the brain that give rise to certain symptoms of ASD, which can then become therapeutic targets, Dr. Elliott said.
 

Investigating large cohorts

The researchers analyzed genetic exome sequencing data from 33 ASD cohorts with a total of 63,237 people and then compared these data with another cohort of people with developmental delay and a cohort of people with schizophrenia. The combined ASD cohorts included 15,036 individuals with ASD, 28,522 parents, and 5,492 unaffected siblings. The remaining participants were 5,591 people with ASD and 8,597 matched controls from case control studies.

In the ASD cohorts, the researchers identified 72 genes that were associated with ASD. De novo variants were eight times more likely in cases (4%) than in controls (0.5%). Ten genes occurred at least twice in ASD cases but never occurred in unaffected siblings.

Then the researchers integrated these ASD genetic data with a cohort of 91,605 people that included 31,058 people with developmental delay and their parents. Substantial overlap with gene mutations existed between these two cohorts: 70.1% of the genes related to developmental delay appeared linked to risk for ASD, and 86.6% of genes associated with ASD risk also had associations with developmental delay. Overall, the researchers identified 373 genes strongly associated with ASD and/or developmental delay and 664 genes with a likely association.

“Isolating genes that exert a greater effect on ASD than they do on other developmental delays has remained challenging due to the frequent comorbidity of these phenotypes,” wrote lead author Jack M. Fu, of Massachusetts General Hospital and Harvard Medical School, both in Boston, and colleagues. “Still, an estimated 13.4% of the transmission and de novo association–ASD genes show little evidence for association in the developmental delay cohort.”
 

ASD, developmental delay, and schizophrenia

When the researchers compared the cells where the genetic mutations occurred in fetal brains, they found that genes associated with developmental delay more often occurred in less differentiated cell types – less mature cells in the developmental process. Gene mutations associated with ASD, on the other hand, occurred in more mature cell types, particularly in maturing excitatory neurons and related cells.

”Our results are consistent with developmental delay-predominant genes being expressed earlier in development and in less differentiated cells than ASD-predominant genes,” they wrote.

The researchers also compared the specific gene mutations found in these two cohorts with a previously published set of 244 genes associated with schizophrenia. Of these, 234 genes are among those with a transmission and de novo association to ASD and/or developmental delay. Of the 72 genes linked to ASD, eight appear in the set of genes linked to schizophrenia, and 61 were associated with developmental delay, though these two subsets do not overlap each other much.

“The ASD-schizophrenia overlap was significantly enriched, while the developmental delay-schizophrenia overlap was not,” they reported. ”Together, these data suggest that one subset of ASD risk genes may overlap developmental delay while a different subset overlaps schizophrenia.”
 

Chasing therapy targets by backtracking through genes

The findings are a substantial step forward in understanding the potential genetic contribution to ASD, but they also highlight the challenges of eventually trying to use this information in a clinically meaningful way.

“Given the substantial overlap between the genes implicated in neurodevelopmental disorders writ large and those implicated directly in ASD, disentangling the relative impact of individual genes on neurodevelopment and phenotypic spectra is a daunting yet important challenge,” the researchers wrote. “To identify the key neurobiological features of ASD will likely require convergence of evidence from many ASD genes and studies.”

Dr. Elliott said the biggest takeaway from this study is a better understanding of how the paradigm has shifted away from finding “one gene” for autism or a cure based on genetics and more toward understanding the pathophysiology of symptoms that can point to therapies for better management of the condition.

“Basic researchers have completely changed the strategy for trying to understand the biology of major disorders,” including, in this case, autism, Dr. Elliott said. “The intent is to try to find the underlying systems [in the brain] by backtracking through genes. Meanwhile, given that scientists have made substantial progress in identifying genes that have specific effects on brain development, “the hope is that will mesh with this kind of research, to begin to identify systems that might ultimately be targets for treating.”

The end goal is to be able to offer targeted approaches, based on the pathways causing a symptom, which can be linked backward to a gene.

”So this is not going to offer an immediate cure – it’s probably not going to offer a cure at all – but it may actually lead to much more targeted medications than we currently have for specific types of symptoms within the autism spectrum,” Dr. Elliott said. “What they’re trying to do, ultimately, is to say, when this system is really badly affected because of a genetic abnormality, even though that genetic abnormality is very rare, it leads to these specific kinds of symptoms. If we can find out the neuroregulators underlying that change, then that would be the target, even if that gene were not present.”

The research was funded by the Simons Foundation for Autism Research Initiative, the SPARK project, the National Human Genome Research Institute Home, the National Institute of Mental Health, the National Institute of Child Health and Development, AMED, and the Beatrice and Samuel Seaver Foundation. Five authors reported financial disclosures linked to Desitin, Roche, BioMarin, BrigeBio Pharma, Illumina, Levo Therapeutics, and Microsoft.

Pfizer has sent an application to the Food and Drug Administration for emergency use authorization of its updated COVID-19 booster vaccine for the fall of 2022, the company announced on Aug. 22.

The vaccine, which is adapted for the BA.4 and BA.5 Omicron variants, would be meant for ages 12 and older. If authorized by the FDA, the doses could ship as soon as September.

“Having rapidly scaled up production, we are positioned to immediately begin distribution of the bivalent Omicron BA.4/BA.5 boosters, if authorized, to help protect individuals and families as we prepare for potential fall and winter surges,” Albert Bourla, PhD, Pfizer’s chairman and CEO, said in the statement.

Earlier this year, the FDA ordered vaccine makers such as Pfizer and Moderna to update their shots to target BA.4 and BA.5, which are better at escaping immunity from earlier vaccines and previous infections.

The United States has a contract to buy 105 million of the Pfizer doses and 66 million of the Moderna doses, according to The Associated Press. Moderna is expected to file its FDA application soon as well.

The new shots target both the original spike protein on the coronavirus and the spike mutations carried by BA.4 and BA.5. For now, BA.5 is causing 89% of new infections in the United States, followed by BA.4.6 with 6.3% and BA.4 with 4.3%, according to the latest Centers for Disease Control and Prevention data.

There’s no way to tell if BA.5 will still be the dominant strain this winter or if new variant will replace it, the AP reported. But public health officials have supported the updated boosters as a way to target the most recent strains and increase immunity again.

On Aug. 15, Great Britain became the first country to authorize another one of Moderna’s updated vaccines, which adds protection against BA.1, or the original Omicron strain that became dominant in the winter of 2021-2022. European regulators are considering this shot, the AP reported, but the United States opted not to use this version since new Omicron variants have become dominant.

To approve the latest Pfizer shot, the FDA will rely on scientific testing of prior updates to the vaccine, rather than the newest boosters, to decide whether to fast-track the updated shots for fall, the AP reported. This method is like how flu vaccines are updated each year without large studies that take months.

Previously, Pfizer announced results from a study that found the earlier Omicron update significantly boosted antibodies capable of fighting the BA.1 variant and provided some protection against BA.4 and BA.5. The company’s latest FDA application contains that data and animal testing on the newest booster, the AP reported.

Pfizer will start a trial using the BA.4/BA.5 booster in coming weeks to get more data on how well the latest shot works. Moderna has begun a similar study.

The full results from these studies won’t be available before a fall booster campaign, which is why the FDA and public health officials have called for an updated shot to be ready for distribution in September.

“It’s clear that none of these vaccines are going to completely prevent infection,” Rachel Presti, MD, a researcher with the Moderna trial and an infectious diseases specialist at Washington University in St. Louis, told the AP.

But previous studies of variant booster candidates have shown that “you still get a broader immune response giving a variant booster than giving the same booster,” she said.

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

Pfizer has sent an application to the Food and Drug Administration for emergency use authorization of its updated COVID-19 booster vaccine for the fall of 2022, the company announced on Aug. 22.

The vaccine, which is adapted for the BA.4 and BA.5 Omicron variants, would be meant for ages 12 and older. If authorized by the FDA, the doses could ship as soon as September.

“Having rapidly scaled up production, we are positioned to immediately begin distribution of the bivalent Omicron BA.4/BA.5 boosters, if authorized, to help protect individuals and families as we prepare for potential fall and winter surges,” Albert Bourla, PhD, Pfizer’s chairman and CEO, said in the statement.

Earlier this year, the FDA ordered vaccine makers such as Pfizer and Moderna to update their shots to target BA.4 and BA.5, which are better at escaping immunity from earlier vaccines and previous infections.

The United States has a contract to buy 105 million of the Pfizer doses and 66 million of the Moderna doses, according to The Associated Press. Moderna is expected to file its FDA application soon as well.

The new shots target both the original spike protein on the coronavirus and the spike mutations carried by BA.4 and BA.5. For now, BA.5 is causing 89% of new infections in the United States, followed by BA.4.6 with 6.3% and BA.4 with 4.3%, according to the latest Centers for Disease Control and Prevention data.

There’s no way to tell if BA.5 will still be the dominant strain this winter or if new variant will replace it, the AP reported. But public health officials have supported the updated boosters as a way to target the most recent strains and increase immunity again.

On Aug. 15, Great Britain became the first country to authorize another one of Moderna’s updated vaccines, which adds protection against BA.1, or the original Omicron strain that became dominant in the winter of 2021-2022. European regulators are considering this shot, the AP reported, but the United States opted not to use this version since new Omicron variants have become dominant.

To approve the latest Pfizer shot, the FDA will rely on scientific testing of prior updates to the vaccine, rather than the newest boosters, to decide whether to fast-track the updated shots for fall, the AP reported. This method is like how flu vaccines are updated each year without large studies that take months.

Previously, Pfizer announced results from a study that found the earlier Omicron update significantly boosted antibodies capable of fighting the BA.1 variant and provided some protection against BA.4 and BA.5. The company’s latest FDA application contains that data and animal testing on the newest booster, the AP reported.

Pfizer will start a trial using the BA.4/BA.5 booster in coming weeks to get more data on how well the latest shot works. Moderna has begun a similar study.

The full results from these studies won’t be available before a fall booster campaign, which is why the FDA and public health officials have called for an updated shot to be ready for distribution in September.

“It’s clear that none of these vaccines are going to completely prevent infection,” Rachel Presti, MD, a researcher with the Moderna trial and an infectious diseases specialist at Washington University in St. Louis, told the AP.

But previous studies of variant booster candidates have shown that “you still get a broader immune response giving a variant booster than giving the same booster,” she said.

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

Pfizer has sent an application to the Food and Drug Administration for emergency use authorization of its updated COVID-19 booster vaccine for the fall of 2022, the company announced on Aug. 22.

The vaccine, which is adapted for the BA.4 and BA.5 Omicron variants, would be meant for ages 12 and older. If authorized by the FDA, the doses could ship as soon as September.

“Having rapidly scaled up production, we are positioned to immediately begin distribution of the bivalent Omicron BA.4/BA.5 boosters, if authorized, to help protect individuals and families as we prepare for potential fall and winter surges,” Albert Bourla, PhD, Pfizer’s chairman and CEO, said in the statement.

Earlier this year, the FDA ordered vaccine makers such as Pfizer and Moderna to update their shots to target BA.4 and BA.5, which are better at escaping immunity from earlier vaccines and previous infections.

The United States has a contract to buy 105 million of the Pfizer doses and 66 million of the Moderna doses, according to The Associated Press. Moderna is expected to file its FDA application soon as well.

The new shots target both the original spike protein on the coronavirus and the spike mutations carried by BA.4 and BA.5. For now, BA.5 is causing 89% of new infections in the United States, followed by BA.4.6 with 6.3% and BA.4 with 4.3%, according to the latest Centers for Disease Control and Prevention data.

There’s no way to tell if BA.5 will still be the dominant strain this winter or if new variant will replace it, the AP reported. But public health officials have supported the updated boosters as a way to target the most recent strains and increase immunity again.

On Aug. 15, Great Britain became the first country to authorize another one of Moderna’s updated vaccines, which adds protection against BA.1, or the original Omicron strain that became dominant in the winter of 2021-2022. European regulators are considering this shot, the AP reported, but the United States opted not to use this version since new Omicron variants have become dominant.

To approve the latest Pfizer shot, the FDA will rely on scientific testing of prior updates to the vaccine, rather than the newest boosters, to decide whether to fast-track the updated shots for fall, the AP reported. This method is like how flu vaccines are updated each year without large studies that take months.

Previously, Pfizer announced results from a study that found the earlier Omicron update significantly boosted antibodies capable of fighting the BA.1 variant and provided some protection against BA.4 and BA.5. The company’s latest FDA application contains that data and animal testing on the newest booster, the AP reported.

Pfizer will start a trial using the BA.4/BA.5 booster in coming weeks to get more data on how well the latest shot works. Moderna has begun a similar study.

The full results from these studies won’t be available before a fall booster campaign, which is why the FDA and public health officials have called for an updated shot to be ready for distribution in September.

“It’s clear that none of these vaccines are going to completely prevent infection,” Rachel Presti, MD, a researcher with the Moderna trial and an infectious diseases specialist at Washington University in St. Louis, told the AP.

But previous studies of variant booster candidates have shown that “you still get a broader immune response giving a variant booster than giving the same booster,” she said.

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

Two new studies offer insights into leukemia survival rates in the United States. From 2000 to 2014, a drop in mortality among children spurred a rise in 5-year leukemia survival rates among patients aged 0-24. But adolescents and young adults who survive 5 years after diagnosis face an ongoing higher risk of death, recent research revealed, and their long-term survival is lower compared to that of the general population.

“Outcomes are improving. However, additional efforts, support, and resources are needed to further improve short- and long-term survival for acute leukemia survivors. Targeted efforts focused on populations that face greater disparities in their survival are needed to move the needle faster,” Michael Roth, MD, codirector of the Adolescent and Young Adult Oncology Program at the University of Texas M.D. Anderson Cancer Center, said in an interview.

In one study, released in The Lancet Child & Adolescent Health, an international team of researchers tracked survival outcomes from various types of leukemia in 61 nations. The study focused on the years 2000-2014 and followed patients aged 0-24.

“Age-standardized 5-year net survival in children, adolescents, and young adults for all leukemias combined during 2010-14 varied widely, ranging from 46% in Mexico to more than 85% in Canada, Cyprus, Belgium, Denmark, Finland, and Australia,” the researchers wrote. “Throughout 2000-14, survival from all leukemias combined remained consistently higher for children than adolescents and young adults, and minimal improvement was seen for adolescents and young adults in most countries.”

The U.S. data came from 41 states that cover 86% of the nation’s population, lead author Naomi Ssenyonga, a research fellow at London School of Hygiene & Tropical Medicine, said in an interview.

The 5-year survival rate for acute lymphoid leukemia (ALL) rose from 80% during 2000-2004 to 86% during 2010-2014. Survival in patients with acute myeloid leukemia (AML) was lower than for other subtypes: 66% in 2010-2014 vs. 57% in 2000-2004.

In regard to all leukemias, “we noted a steady increase in the U.S. of 6 percentage points in 5-year survival, up from 77% for patients diagnosed during 2000-2004 to 83% for those diagnosed during 2010-2014,” Ms. Ssenyonga said. “The gains were largely driven by the improvements seen among children.”

Why haven’t adolescents and young adults gained as much ground in survival?

“They often have unique clinical needs,” Ms. Ssenyonga said. “Over the past few years, adolescents and young adults with leukemia in some parts of the world, including the U.S., have increasingly been treated under pediatric protocols. This has led to higher survival. However, this approach has not been adopted consistently, and survival for adolescents and young adults with leukemia is still generally lower than survival for children.”

Gwen Nichols, MD, chief medical officer of the Leukemia & Lymphoma Society, agreed that pediatric treatment protocols hold promise as treatments for young adults. However, “because we arbitrarily set an age cutoff for being an adult, many of these patients are treated by an adult [nonpediatric] hematologist/oncologist, and some patients in the 20-39 age group do not receive the more intensive treatment regimens given to children,” she said in an interview.

In another study, published in Cancer Epidemiology, Biomarkers, & Prevention, M.D. Anderson Cancer Center’s Dr. Roth and colleagues tracked 1,938 patients with ALL and 2,350 with AML who were diagnosed at ages 15-39 from 1980 to 2009. All lived at least 5 years after diagnosis. In both groups, about 58% were White, and most of the rest were Hispanic. The median age of diagnosis for ALL was 23 (range: 15-39) and 28 years for AML (range: 15-39).

“For ALL, 10-year survival for those diagnosed in the 1980s, 1990s, and 2000s was 83%, 88%, and 88%, respectively,” the researchers reported. “Ten-year survival for AML was 82%, 90%, and 90% for those diagnosed in the 1980s, 1990s, and 2000s, respectively.”

“Early mortality within 10 years of diagnosis was mostly secondary to leukemia progressing or recurring. We believe that later mortality is secondary to the development of late side effects from their cancer treatment,” Dr. Roth said.

He noted that many adolescents and young adults with ALL or AML receive stem-cell transplants. “This treatment approach is effective. However, it is associated with short- and long-term toxicity that impacts patients’ health for many years after treatment.”

Indeed, up to 80% of acute leukemia survivors have significant health complications after therapy, said the Leukemia & Lymphoma Society’s Dr. Nichols, who wasn’t surprised by the findings. According to the society, “even when treatments are effective, more than 70% of childhood cancer survivors have a chronic health condition and 42% have a severe, disabling or life-threatening condition 30 years after diagnosis.”

“It would be interesting to understand the male predominance better,” she added, noting that the study found that male patients had worse long-term survival than females (survival time ratio: 0.61, 95% confidence interval, 0.45-0.82). “While it is tempting to suggest it is due to difference in cardiac disease, I am not aware of data to support why there is this survival difference.”

What’s next? “In ALL, we now have a number of new modalities to treat high-risk and relapsed disease such as antibodies and CAR-T,” Dr. Nichols said. “We anticipate that 5-year survival can improve utilizing these modalities due to getting more patients into remission, hopefully while reducing chemotherapeutic toxicity.”

Dr. Nichol’s also highlighted the society’s new genomic-led Pediatric Acute Leukemia (PedAL) Master Clinical Trial, which began enrolling children with acute leukemia in the United States and Canada this year, in an effort to transform medicine’s traditional high-level chemotherapy strategy to their care. The project was launched in collaboration with the National Cancer Institute, Children’s Oncology Group, and the European Pediatric Acute Leukemia Foundation.

As part of the screening process, the biology of each child’s cancer will be identified, and families will be encouraged to enroll them in appropriate targeted therapy trials.

“Until we are able to decrease the toxicity of leukemia regimens, we won’t see a dramatic shift in late effects and thus in morbidity and mortality,” Dr. Nichols said. “The trial is an effort to test newer, less toxic regimens to begin to change that cycle.”

The 5-year survival study was funded by Children with Cancer UK, Institut National du Cancer, La Ligue Contre le Cancer, Centers for Disease Control and Prevention, Swiss Re, Swiss Cancer Research foundation, Swiss Cancer League, Rossy Family Foundation, National Cancer Institute, and the American Cancer Society. One author reports a grant from Macmillan Cancer Support, consultancy fees from Pfizer, and unsolicited small gifts from Moondance Cancer Initiative for philanthropic work. The other authors report no disclosures.

The long-term survival study was funded by the National Cancer Institute, the Archer Foundation and LyondellBasell Industries. Dr. Roth reports no disclosures; other authors report various disclosures. Dr. Nichols reports no disclosures.

Two new studies offer insights into leukemia survival rates in the United States. From 2000 to 2014, a drop in mortality among children spurred a rise in 5-year leukemia survival rates among patients aged 0-24. But adolescents and young adults who survive 5 years after diagnosis face an ongoing higher risk of death, recent research revealed, and their long-term survival is lower compared to that of the general population.

“Outcomes are improving. However, additional efforts, support, and resources are needed to further improve short- and long-term survival for acute leukemia survivors. Targeted efforts focused on populations that face greater disparities in their survival are needed to move the needle faster,” Michael Roth, MD, codirector of the Adolescent and Young Adult Oncology Program at the University of Texas M.D. Anderson Cancer Center, said in an interview.

In one study, released in The Lancet Child & Adolescent Health, an international team of researchers tracked survival outcomes from various types of leukemia in 61 nations. The study focused on the years 2000-2014 and followed patients aged 0-24.

“Age-standardized 5-year net survival in children, adolescents, and young adults for all leukemias combined during 2010-14 varied widely, ranging from 46% in Mexico to more than 85% in Canada, Cyprus, Belgium, Denmark, Finland, and Australia,” the researchers wrote. “Throughout 2000-14, survival from all leukemias combined remained consistently higher for children than adolescents and young adults, and minimal improvement was seen for adolescents and young adults in most countries.”

The U.S. data came from 41 states that cover 86% of the nation’s population, lead author Naomi Ssenyonga, a research fellow at London School of Hygiene & Tropical Medicine, said in an interview.

The 5-year survival rate for acute lymphoid leukemia (ALL) rose from 80% during 2000-2004 to 86% during 2010-2014. Survival in patients with acute myeloid leukemia (AML) was lower than for other subtypes: 66% in 2010-2014 vs. 57% in 2000-2004.

In regard to all leukemias, “we noted a steady increase in the U.S. of 6 percentage points in 5-year survival, up from 77% for patients diagnosed during 2000-2004 to 83% for those diagnosed during 2010-2014,” Ms. Ssenyonga said. “The gains were largely driven by the improvements seen among children.”

Why haven’t adolescents and young adults gained as much ground in survival?

“They often have unique clinical needs,” Ms. Ssenyonga said. “Over the past few years, adolescents and young adults with leukemia in some parts of the world, including the U.S., have increasingly been treated under pediatric protocols. This has led to higher survival. However, this approach has not been adopted consistently, and survival for adolescents and young adults with leukemia is still generally lower than survival for children.”

Gwen Nichols, MD, chief medical officer of the Leukemia & Lymphoma Society, agreed that pediatric treatment protocols hold promise as treatments for young adults. However, “because we arbitrarily set an age cutoff for being an adult, many of these patients are treated by an adult [nonpediatric] hematologist/oncologist, and some patients in the 20-39 age group do not receive the more intensive treatment regimens given to children,” she said in an interview.

In another study, published in Cancer Epidemiology, Biomarkers, & Prevention, M.D. Anderson Cancer Center’s Dr. Roth and colleagues tracked 1,938 patients with ALL and 2,350 with AML who were diagnosed at ages 15-39 from 1980 to 2009. All lived at least 5 years after diagnosis. In both groups, about 58% were White, and most of the rest were Hispanic. The median age of diagnosis for ALL was 23 (range: 15-39) and 28 years for AML (range: 15-39).

“For ALL, 10-year survival for those diagnosed in the 1980s, 1990s, and 2000s was 83%, 88%, and 88%, respectively,” the researchers reported. “Ten-year survival for AML was 82%, 90%, and 90% for those diagnosed in the 1980s, 1990s, and 2000s, respectively.”

“Early mortality within 10 years of diagnosis was mostly secondary to leukemia progressing or recurring. We believe that later mortality is secondary to the development of late side effects from their cancer treatment,” Dr. Roth said.

He noted that many adolescents and young adults with ALL or AML receive stem-cell transplants. “This treatment approach is effective. However, it is associated with short- and long-term toxicity that impacts patients’ health for many years after treatment.”

Indeed, up to 80% of acute leukemia survivors have significant health complications after therapy, said the Leukemia & Lymphoma Society’s Dr. Nichols, who wasn’t surprised by the findings. According to the society, “even when treatments are effective, more than 70% of childhood cancer survivors have a chronic health condition and 42% have a severe, disabling or life-threatening condition 30 years after diagnosis.”

“It would be interesting to understand the male predominance better,” she added, noting that the study found that male patients had worse long-term survival than females (survival time ratio: 0.61, 95% confidence interval, 0.45-0.82). “While it is tempting to suggest it is due to difference in cardiac disease, I am not aware of data to support why there is this survival difference.”

What’s next? “In ALL, we now have a number of new modalities to treat high-risk and relapsed disease such as antibodies and CAR-T,” Dr. Nichols said. “We anticipate that 5-year survival can improve utilizing these modalities due to getting more patients into remission, hopefully while reducing chemotherapeutic toxicity.”

Dr. Nichol’s also highlighted the society’s new genomic-led Pediatric Acute Leukemia (PedAL) Master Clinical Trial, which began enrolling children with acute leukemia in the United States and Canada this year, in an effort to transform medicine’s traditional high-level chemotherapy strategy to their care. The project was launched in collaboration with the National Cancer Institute, Children’s Oncology Group, and the European Pediatric Acute Leukemia Foundation.

As part of the screening process, the biology of each child’s cancer will be identified, and families will be encouraged to enroll them in appropriate targeted therapy trials.

“Until we are able to decrease the toxicity of leukemia regimens, we won’t see a dramatic shift in late effects and thus in morbidity and mortality,” Dr. Nichols said. “The trial is an effort to test newer, less toxic regimens to begin to change that cycle.”

The 5-year survival study was funded by Children with Cancer UK, Institut National du Cancer, La Ligue Contre le Cancer, Centers for Disease Control and Prevention, Swiss Re, Swiss Cancer Research foundation, Swiss Cancer League, Rossy Family Foundation, National Cancer Institute, and the American Cancer Society. One author reports a grant from Macmillan Cancer Support, consultancy fees from Pfizer, and unsolicited small gifts from Moondance Cancer Initiative for philanthropic work. The other authors report no disclosures.

The long-term survival study was funded by the National Cancer Institute, the Archer Foundation and LyondellBasell Industries. Dr. Roth reports no disclosures; other authors report various disclosures. Dr. Nichols reports no disclosures.

Two new studies offer insights into leukemia survival rates in the United States. From 2000 to 2014, a drop in mortality among children spurred a rise in 5-year leukemia survival rates among patients aged 0-24. But adolescents and young adults who survive 5 years after diagnosis face an ongoing higher risk of death, recent research revealed, and their long-term survival is lower compared to that of the general population.

“Outcomes are improving. However, additional efforts, support, and resources are needed to further improve short- and long-term survival for acute leukemia survivors. Targeted efforts focused on populations that face greater disparities in their survival are needed to move the needle faster,” Michael Roth, MD, codirector of the Adolescent and Young Adult Oncology Program at the University of Texas M.D. Anderson Cancer Center, said in an interview.

In one study, released in The Lancet Child & Adolescent Health, an international team of researchers tracked survival outcomes from various types of leukemia in 61 nations. The study focused on the years 2000-2014 and followed patients aged 0-24.

“Age-standardized 5-year net survival in children, adolescents, and young adults for all leukemias combined during 2010-14 varied widely, ranging from 46% in Mexico to more than 85% in Canada, Cyprus, Belgium, Denmark, Finland, and Australia,” the researchers wrote. “Throughout 2000-14, survival from all leukemias combined remained consistently higher for children than adolescents and young adults, and minimal improvement was seen for adolescents and young adults in most countries.”

The U.S. data came from 41 states that cover 86% of the nation’s population, lead author Naomi Ssenyonga, a research fellow at London School of Hygiene & Tropical Medicine, said in an interview.

The 5-year survival rate for acute lymphoid leukemia (ALL) rose from 80% during 2000-2004 to 86% during 2010-2014. Survival in patients with acute myeloid leukemia (AML) was lower than for other subtypes: 66% in 2010-2014 vs. 57% in 2000-2004.

In regard to all leukemias, “we noted a steady increase in the U.S. of 6 percentage points in 5-year survival, up from 77% for patients diagnosed during 2000-2004 to 83% for those diagnosed during 2010-2014,” Ms. Ssenyonga said. “The gains were largely driven by the improvements seen among children.”

Why haven’t adolescents and young adults gained as much ground in survival?

“They often have unique clinical needs,” Ms. Ssenyonga said. “Over the past few years, adolescents and young adults with leukemia in some parts of the world, including the U.S., have increasingly been treated under pediatric protocols. This has led to higher survival. However, this approach has not been adopted consistently, and survival for adolescents and young adults with leukemia is still generally lower than survival for children.”

Gwen Nichols, MD, chief medical officer of the Leukemia & Lymphoma Society, agreed that pediatric treatment protocols hold promise as treatments for young adults. However, “because we arbitrarily set an age cutoff for being an adult, many of these patients are treated by an adult [nonpediatric] hematologist/oncologist, and some patients in the 20-39 age group do not receive the more intensive treatment regimens given to children,” she said in an interview.

In another study, published in Cancer Epidemiology, Biomarkers, & Prevention, M.D. Anderson Cancer Center’s Dr. Roth and colleagues tracked 1,938 patients with ALL and 2,350 with AML who were diagnosed at ages 15-39 from 1980 to 2009. All lived at least 5 years after diagnosis. In both groups, about 58% were White, and most of the rest were Hispanic. The median age of diagnosis for ALL was 23 (range: 15-39) and 28 years for AML (range: 15-39).

“For ALL, 10-year survival for those diagnosed in the 1980s, 1990s, and 2000s was 83%, 88%, and 88%, respectively,” the researchers reported. “Ten-year survival for AML was 82%, 90%, and 90% for those diagnosed in the 1980s, 1990s, and 2000s, respectively.”

“Early mortality within 10 years of diagnosis was mostly secondary to leukemia progressing or recurring. We believe that later mortality is secondary to the development of late side effects from their cancer treatment,” Dr. Roth said.

He noted that many adolescents and young adults with ALL or AML receive stem-cell transplants. “This treatment approach is effective. However, it is associated with short- and long-term toxicity that impacts patients’ health for many years after treatment.”

Indeed, up to 80% of acute leukemia survivors have significant health complications after therapy, said the Leukemia & Lymphoma Society’s Dr. Nichols, who wasn’t surprised by the findings. According to the society, “even when treatments are effective, more than 70% of childhood cancer survivors have a chronic health condition and 42% have a severe, disabling or life-threatening condition 30 years after diagnosis.”

“It would be interesting to understand the male predominance better,” she added, noting that the study found that male patients had worse long-term survival than females (survival time ratio: 0.61, 95% confidence interval, 0.45-0.82). “While it is tempting to suggest it is due to difference in cardiac disease, I am not aware of data to support why there is this survival difference.”

What’s next? “In ALL, we now have a number of new modalities to treat high-risk and relapsed disease such as antibodies and CAR-T,” Dr. Nichols said. “We anticipate that 5-year survival can improve utilizing these modalities due to getting more patients into remission, hopefully while reducing chemotherapeutic toxicity.”

Dr. Nichol’s also highlighted the society’s new genomic-led Pediatric Acute Leukemia (PedAL) Master Clinical Trial, which began enrolling children with acute leukemia in the United States and Canada this year, in an effort to transform medicine’s traditional high-level chemotherapy strategy to their care. The project was launched in collaboration with the National Cancer Institute, Children’s Oncology Group, and the European Pediatric Acute Leukemia Foundation.

As part of the screening process, the biology of each child’s cancer will be identified, and families will be encouraged to enroll them in appropriate targeted therapy trials.

“Until we are able to decrease the toxicity of leukemia regimens, we won’t see a dramatic shift in late effects and thus in morbidity and mortality,” Dr. Nichols said. “The trial is an effort to test newer, less toxic regimens to begin to change that cycle.”

The 5-year survival study was funded by Children with Cancer UK, Institut National du Cancer, La Ligue Contre le Cancer, Centers for Disease Control and Prevention, Swiss Re, Swiss Cancer Research foundation, Swiss Cancer League, Rossy Family Foundation, National Cancer Institute, and the American Cancer Society. One author reports a grant from Macmillan Cancer Support, consultancy fees from Pfizer, and unsolicited small gifts from Moondance Cancer Initiative for philanthropic work. The other authors report no disclosures.

The long-term survival study was funded by the National Cancer Institute, the Archer Foundation and LyondellBasell Industries. Dr. Roth reports no disclosures; other authors report various disclosures. Dr. Nichols reports no disclosures.