Researchers are leading several programs designed to serve the sickle cell community in the United States and sub-Saharan Africa, officials at the National Heart, Lung, and Blood Institute (NHLBI) said during a recent webinar.

Dr_Microbe/Thinkstock

One program based in the United States is focused on building a registry for patients with sickle cell disease (SCD) and conducting studies designed to improve SCD care. Another program involves building “an information-sharing network and patient-powered registry” in the United States.

The programs in sub-Saharan Africa were designed to establish a database of SCD patients, optimize the use of hydroxyurea in children with SCD, and aid genomic studies of SCD.

W. Keith Hoots, MD, director of the Division of Blood Diseases and Resources at NHLBI, began the webinar with an overview of the programs in sub-Saharan Africa. He described four programs with sites in nine countries (Angola, Cameroon, Democratic Republic of Congo, Ghana, Kenya, Nigeria, South Africa, Tanzania, and Uganda).

SPARCO and SADaCC

Dr. Hoots outlined the scope the Sickle Pan-African Research Consortium (SPARCO) and the Sickle Africa Data Coordinating Center (SADaCC), both part of the Sickle In Africa consortium.

A major goal of SPARCO and SADaCC is to create a Research Electronic Data Capture database that encompasses SCD patients in sub-Saharan Africa. As of April 2019, the database included 6,578 patients. The target is 13,000 patients.

Other goals of SPARCO and SADaCC are to “harmonize” SCD phenotype definitions and ontologies, create clinical guidelines for SCD management in sub-Saharan Africa, plan future cohort studies, and develop programs for newborn screening, infection prevention, and increased use of hydroxyurea.

“So far, they’re well along in establishing a registry and a database system,” Dr. Hoots said. “They’ve agreed on the database elements, phenotype definitions, and ontologies, they’ve developed some regionally appropriate clinical management guidelines, and they’ve begun skills development on the ground at all respective sites.”

REACH

Another program Dr. Hoots discussed is Realizing Effectiveness Across Continents With Hydroxyurea (REACH), a phase 1/2 pilot study of hydroxyurea in children (aged 1-10 years) with SCD in sub-Saharan Africa.

The goals of REACH are to determine the optimal dose of hydroxyurea in this population; teach African physicians how to administer hydroxyurea; assess the safety, feasibility, and benefits of hydroxyurea; study variability in response to hydroxyurea; gather data for the Research Electronic Data Capture database; and establish a research infrastructure for future collaborations.

Results from more than 600 children enrolled in REACH were presented at the 2018 annual meeting of the American Society of Hematology and simultaneously published in the New England Journal of Medicine (N Engl J Med. 2019 Jan 10; 380[2]:121-31).

SickleGenAfrica

SickleGenAfrica is part of the H3Africa consortium and aims to “build capacity for genomic research in Africa,” Dr. Hoots said.

Under this program, researchers will conduct three studies to test the hypothesis that genetic variation affects the defense against hemolysis and organ damage in patients with SCD. The researchers will study existing cohorts of SCD patients including children and adults.

Other goals of SickleGenAfrica are to establish a molecular hematology and sickle cell mouse core, an SCD biorepository core, a bioinformatics core, and an administrative core for the coordination of activities. The program will also be used to train “future science leaders” in SCD research, Dr. Hoots said.
 

SCDIC

Cheryl Anne Boyce, PhD, chief of the Implementation Science Branch at the Center for Translation Research and Implementation Science at NHLBI, discussed the United States–based Sickle Cell Disease Implementation Consortium (SCDIC).

“The goals of the consortium are to develop a registry in collaboration with other centers and the NHLBI, as well as a needs-based community assessment of the barriers to care for subjects with sickle cell disease,” Dr. Boyce said. “We also wanted to design implementation research studies that address the identified barriers to care.”

Dr. Boyce said the SCDIC’s registry is open to patients aged 15-45 years who have a confirmed SCD diagnosis, speak English, and are able to consent to and complete a survey. The registry has enrolled almost 2,400 patients from eight centers over 18 months.

The SCDIC has also performed a needs assessment that prompted the development of three implementation research studies. The first study involves using mobile health interventions to, ideally, increase patient adherence to hydroxyurea and improve provider knowledge of hydroxyurea.

With the second study, researchers aim to improve the care of SCD patients in the emergency department by using an inpatient portal. The goals of the third study are to establish a standard definition for unaffiliated patients, conduct a needs assessment for this group, and develop an intervention that can provide these patients with guideline-based SCD care.

Get Connected

Kim Smith-Whitley, MD, director of the Comprehensive Sickle Cell Center at the Children’s Hospital of Philadelphia and a board member of the Sickle Cell Disease Association of America (SCDAA), described Get Connected, “an information-sharing network and patient-powered registry” created by SCDAA.

Dr. Smith-Whitley said one purpose of Get Connected is to provide a network that facilitates “the distribution of information related to clinical care, research, health services, health policy, and advocacy.”

The network is open to families living with SCD and sickle cell trait, SCDAA member organizations, health care providers, clinical researchers, and community-based organizations.

Get Connected also includes a registry for SCD patients that stores information on their diagnosis and treatment, as well as online communities that can be used to share information and provide psychosocial support.

Thus far, Get Connected has enrolled 6,329 individuals. This includes 5,100 children and adults with SCD, 652 children and adults with sickle cell trait, and 577 nonpatients.

The webinar presenters did not disclose any conflicts of interest.

jensmith@mdedge.com

Researchers are leading several programs designed to serve the sickle cell community in the United States and sub-Saharan Africa, officials at the National Heart, Lung, and Blood Institute (NHLBI) said during a recent webinar.

Dr_Microbe/Thinkstock

One program based in the United States is focused on building a registry for patients with sickle cell disease (SCD) and conducting studies designed to improve SCD care. Another program involves building “an information-sharing network and patient-powered registry” in the United States.

The programs in sub-Saharan Africa were designed to establish a database of SCD patients, optimize the use of hydroxyurea in children with SCD, and aid genomic studies of SCD.

W. Keith Hoots, MD, director of the Division of Blood Diseases and Resources at NHLBI, began the webinar with an overview of the programs in sub-Saharan Africa. He described four programs with sites in nine countries (Angola, Cameroon, Democratic Republic of Congo, Ghana, Kenya, Nigeria, South Africa, Tanzania, and Uganda).

SPARCO and SADaCC

Dr. Hoots outlined the scope the Sickle Pan-African Research Consortium (SPARCO) and the Sickle Africa Data Coordinating Center (SADaCC), both part of the Sickle In Africa consortium.

A major goal of SPARCO and SADaCC is to create a Research Electronic Data Capture database that encompasses SCD patients in sub-Saharan Africa. As of April 2019, the database included 6,578 patients. The target is 13,000 patients.

Other goals of SPARCO and SADaCC are to “harmonize” SCD phenotype definitions and ontologies, create clinical guidelines for SCD management in sub-Saharan Africa, plan future cohort studies, and develop programs for newborn screening, infection prevention, and increased use of hydroxyurea.

“So far, they’re well along in establishing a registry and a database system,” Dr. Hoots said. “They’ve agreed on the database elements, phenotype definitions, and ontologies, they’ve developed some regionally appropriate clinical management guidelines, and they’ve begun skills development on the ground at all respective sites.”

REACH

Another program Dr. Hoots discussed is Realizing Effectiveness Across Continents With Hydroxyurea (REACH), a phase 1/2 pilot study of hydroxyurea in children (aged 1-10 years) with SCD in sub-Saharan Africa.

The goals of REACH are to determine the optimal dose of hydroxyurea in this population; teach African physicians how to administer hydroxyurea; assess the safety, feasibility, and benefits of hydroxyurea; study variability in response to hydroxyurea; gather data for the Research Electronic Data Capture database; and establish a research infrastructure for future collaborations.

Results from more than 600 children enrolled in REACH were presented at the 2018 annual meeting of the American Society of Hematology and simultaneously published in the New England Journal of Medicine (N Engl J Med. 2019 Jan 10; 380[2]:121-31).

SickleGenAfrica

SickleGenAfrica is part of the H3Africa consortium and aims to “build capacity for genomic research in Africa,” Dr. Hoots said.

Under this program, researchers will conduct three studies to test the hypothesis that genetic variation affects the defense against hemolysis and organ damage in patients with SCD. The researchers will study existing cohorts of SCD patients including children and adults.

Other goals of SickleGenAfrica are to establish a molecular hematology and sickle cell mouse core, an SCD biorepository core, a bioinformatics core, and an administrative core for the coordination of activities. The program will also be used to train “future science leaders” in SCD research, Dr. Hoots said.
 

SCDIC

Cheryl Anne Boyce, PhD, chief of the Implementation Science Branch at the Center for Translation Research and Implementation Science at NHLBI, discussed the United States–based Sickle Cell Disease Implementation Consortium (SCDIC).

“The goals of the consortium are to develop a registry in collaboration with other centers and the NHLBI, as well as a needs-based community assessment of the barriers to care for subjects with sickle cell disease,” Dr. Boyce said. “We also wanted to design implementation research studies that address the identified barriers to care.”

Dr. Boyce said the SCDIC’s registry is open to patients aged 15-45 years who have a confirmed SCD diagnosis, speak English, and are able to consent to and complete a survey. The registry has enrolled almost 2,400 patients from eight centers over 18 months.

The SCDIC has also performed a needs assessment that prompted the development of three implementation research studies. The first study involves using mobile health interventions to, ideally, increase patient adherence to hydroxyurea and improve provider knowledge of hydroxyurea.

With the second study, researchers aim to improve the care of SCD patients in the emergency department by using an inpatient portal. The goals of the third study are to establish a standard definition for unaffiliated patients, conduct a needs assessment for this group, and develop an intervention that can provide these patients with guideline-based SCD care.

Get Connected

Kim Smith-Whitley, MD, director of the Comprehensive Sickle Cell Center at the Children’s Hospital of Philadelphia and a board member of the Sickle Cell Disease Association of America (SCDAA), described Get Connected, “an information-sharing network and patient-powered registry” created by SCDAA.

Dr. Smith-Whitley said one purpose of Get Connected is to provide a network that facilitates “the distribution of information related to clinical care, research, health services, health policy, and advocacy.”

The network is open to families living with SCD and sickle cell trait, SCDAA member organizations, health care providers, clinical researchers, and community-based organizations.

Get Connected also includes a registry for SCD patients that stores information on their diagnosis and treatment, as well as online communities that can be used to share information and provide psychosocial support.

Thus far, Get Connected has enrolled 6,329 individuals. This includes 5,100 children and adults with SCD, 652 children and adults with sickle cell trait, and 577 nonpatients.

The webinar presenters did not disclose any conflicts of interest.

jensmith@mdedge.com

Researchers are leading several programs designed to serve the sickle cell community in the United States and sub-Saharan Africa, officials at the National Heart, Lung, and Blood Institute (NHLBI) said during a recent webinar.

Dr_Microbe/Thinkstock

One program based in the United States is focused on building a registry for patients with sickle cell disease (SCD) and conducting studies designed to improve SCD care. Another program involves building “an information-sharing network and patient-powered registry” in the United States.

The programs in sub-Saharan Africa were designed to establish a database of SCD patients, optimize the use of hydroxyurea in children with SCD, and aid genomic studies of SCD.

W. Keith Hoots, MD, director of the Division of Blood Diseases and Resources at NHLBI, began the webinar with an overview of the programs in sub-Saharan Africa. He described four programs with sites in nine countries (Angola, Cameroon, Democratic Republic of Congo, Ghana, Kenya, Nigeria, South Africa, Tanzania, and Uganda).

SPARCO and SADaCC

Dr. Hoots outlined the scope the Sickle Pan-African Research Consortium (SPARCO) and the Sickle Africa Data Coordinating Center (SADaCC), both part of the Sickle In Africa consortium.

A major goal of SPARCO and SADaCC is to create a Research Electronic Data Capture database that encompasses SCD patients in sub-Saharan Africa. As of April 2019, the database included 6,578 patients. The target is 13,000 patients.

Other goals of SPARCO and SADaCC are to “harmonize” SCD phenotype definitions and ontologies, create clinical guidelines for SCD management in sub-Saharan Africa, plan future cohort studies, and develop programs for newborn screening, infection prevention, and increased use of hydroxyurea.

“So far, they’re well along in establishing a registry and a database system,” Dr. Hoots said. “They’ve agreed on the database elements, phenotype definitions, and ontologies, they’ve developed some regionally appropriate clinical management guidelines, and they’ve begun skills development on the ground at all respective sites.”

REACH

Another program Dr. Hoots discussed is Realizing Effectiveness Across Continents With Hydroxyurea (REACH), a phase 1/2 pilot study of hydroxyurea in children (aged 1-10 years) with SCD in sub-Saharan Africa.

The goals of REACH are to determine the optimal dose of hydroxyurea in this population; teach African physicians how to administer hydroxyurea; assess the safety, feasibility, and benefits of hydroxyurea; study variability in response to hydroxyurea; gather data for the Research Electronic Data Capture database; and establish a research infrastructure for future collaborations.

Results from more than 600 children enrolled in REACH were presented at the 2018 annual meeting of the American Society of Hematology and simultaneously published in the New England Journal of Medicine (N Engl J Med. 2019 Jan 10; 380[2]:121-31).

SickleGenAfrica

SickleGenAfrica is part of the H3Africa consortium and aims to “build capacity for genomic research in Africa,” Dr. Hoots said.

Under this program, researchers will conduct three studies to test the hypothesis that genetic variation affects the defense against hemolysis and organ damage in patients with SCD. The researchers will study existing cohorts of SCD patients including children and adults.

Other goals of SickleGenAfrica are to establish a molecular hematology and sickle cell mouse core, an SCD biorepository core, a bioinformatics core, and an administrative core for the coordination of activities. The program will also be used to train “future science leaders” in SCD research, Dr. Hoots said.
 

SCDIC

Cheryl Anne Boyce, PhD, chief of the Implementation Science Branch at the Center for Translation Research and Implementation Science at NHLBI, discussed the United States–based Sickle Cell Disease Implementation Consortium (SCDIC).

“The goals of the consortium are to develop a registry in collaboration with other centers and the NHLBI, as well as a needs-based community assessment of the barriers to care for subjects with sickle cell disease,” Dr. Boyce said. “We also wanted to design implementation research studies that address the identified barriers to care.”

Dr. Boyce said the SCDIC’s registry is open to patients aged 15-45 years who have a confirmed SCD diagnosis, speak English, and are able to consent to and complete a survey. The registry has enrolled almost 2,400 patients from eight centers over 18 months.

The SCDIC has also performed a needs assessment that prompted the development of three implementation research studies. The first study involves using mobile health interventions to, ideally, increase patient adherence to hydroxyurea and improve provider knowledge of hydroxyurea.

With the second study, researchers aim to improve the care of SCD patients in the emergency department by using an inpatient portal. The goals of the third study are to establish a standard definition for unaffiliated patients, conduct a needs assessment for this group, and develop an intervention that can provide these patients with guideline-based SCD care.

Get Connected

Kim Smith-Whitley, MD, director of the Comprehensive Sickle Cell Center at the Children’s Hospital of Philadelphia and a board member of the Sickle Cell Disease Association of America (SCDAA), described Get Connected, “an information-sharing network and patient-powered registry” created by SCDAA.

Dr. Smith-Whitley said one purpose of Get Connected is to provide a network that facilitates “the distribution of information related to clinical care, research, health services, health policy, and advocacy.”

The network is open to families living with SCD and sickle cell trait, SCDAA member organizations, health care providers, clinical researchers, and community-based organizations.

Get Connected also includes a registry for SCD patients that stores information on their diagnosis and treatment, as well as online communities that can be used to share information and provide psychosocial support.

Thus far, Get Connected has enrolled 6,329 individuals. This includes 5,100 children and adults with SCD, 652 children and adults with sickle cell trait, and 577 nonpatients.

The webinar presenters did not disclose any conflicts of interest.

jensmith@mdedge.com

Young people who have received allogeneic hematopoietic stem cell transplants (HSCTs) are more likely to wear hats, sunscreen and other sun protection, but still intentionally tan and experience sunburn at the same rate as their peers, new research suggests.

Yuri Arcurs/Fotolia

In a survey‐based, cross‐sectional cohort study, researchers compared sun-protection behaviors and sun exposure in 85 children aged 21 years and younger who had undergone HSCT and 85 age-, sex-, and skin type–matched controls. The findings were published in Pediatric Dermatology.

HSCT recipients have a higher risk of long-term complications such as skin cancer, for which sun exposure is a major modifiable environmental risk factor.

“Therefore, consistent sun avoidance and protection as well as regular dermatologic evaluations are important for HSCT recipients,” wrote Edward B. Li, PhD, from Harvard Medical School, Boston, and coauthors.

The survey found no significant difference between the transplant and control group in the amount of intentional sun exposure, such as the amount of time spent outside on weekdays and weekends during the peak sun intensity hours of 10 a.m. and 4 p.m. More than one in five transplant recipients (21.2%) reported spending at least 3 hours a day outside between 10 a.m. and 4 p.m. on weekdays, as did 36.5% of transplant recipients on weekends.

There were also no significant differences between the two groups in terms of time spent tanning, either in the sun or in a tanning bed. Additionally, a similar number of transplant recipients and controls experienced one or more red or painful sunburns in the past year (25.9% vs. 27.1%).

However, transplant patients did practice better sun protection behaviors than did the control group, with 60% reporting that they always wore sunscreen, compared with 29.4% of controls. The transplant recipients were also significantly more likely to wear sunglasses and a hat and to stay in the shade or use an umbrella.

“While these data may reflect that HSCT patients are not practicing adequate sun avoidance, it may also suggest that these long‐term survivors are able to enjoy being outdoors as much as their peers and have a similar desire to have a tanned appearance,” the researchers wrote. “While a healthy and active lifestyle should be encouraged for all children, our results emphasize the need for pediatric HSCT survivors to be educated on their increased risk for UV‐related skin cancers, counseled on avoidance of intentional tanning, and advised on the importance of sun protection behaviors in an effort to improve long-term outcomes.”

The researchers noted that transplant recipients were significantly more likely to have had a full body skin exam from a health care professional than were individuals in the control group (61.2% vs. 4.7%) and were more likely to have done a self-check or been checked by a partner in the previous year.

The study was supported by the Society for Pediatric Dermatology, the Dermatology Foundation, the National Institutes of Health, and the Dr. Miriam and Sheldon G. Adelson Medical Research Foundation. One author declared a financial interest in a company developing a dermatological product. No other conflicts of interest were declared.

SOURCE: Li EB et al. Pediatr Dermatol. 2019 Aug 13. doi: 10.1111/pde.13984.

Young people who have received allogeneic hematopoietic stem cell transplants (HSCTs) are more likely to wear hats, sunscreen and other sun protection, but still intentionally tan and experience sunburn at the same rate as their peers, new research suggests.

Yuri Arcurs/Fotolia

In a survey‐based, cross‐sectional cohort study, researchers compared sun-protection behaviors and sun exposure in 85 children aged 21 years and younger who had undergone HSCT and 85 age-, sex-, and skin type–matched controls. The findings were published in Pediatric Dermatology.

HSCT recipients have a higher risk of long-term complications such as skin cancer, for which sun exposure is a major modifiable environmental risk factor.

“Therefore, consistent sun avoidance and protection as well as regular dermatologic evaluations are important for HSCT recipients,” wrote Edward B. Li, PhD, from Harvard Medical School, Boston, and coauthors.

The survey found no significant difference between the transplant and control group in the amount of intentional sun exposure, such as the amount of time spent outside on weekdays and weekends during the peak sun intensity hours of 10 a.m. and 4 p.m. More than one in five transplant recipients (21.2%) reported spending at least 3 hours a day outside between 10 a.m. and 4 p.m. on weekdays, as did 36.5% of transplant recipients on weekends.

There were also no significant differences between the two groups in terms of time spent tanning, either in the sun or in a tanning bed. Additionally, a similar number of transplant recipients and controls experienced one or more red or painful sunburns in the past year (25.9% vs. 27.1%).

However, transplant patients did practice better sun protection behaviors than did the control group, with 60% reporting that they always wore sunscreen, compared with 29.4% of controls. The transplant recipients were also significantly more likely to wear sunglasses and a hat and to stay in the shade or use an umbrella.

“While these data may reflect that HSCT patients are not practicing adequate sun avoidance, it may also suggest that these long‐term survivors are able to enjoy being outdoors as much as their peers and have a similar desire to have a tanned appearance,” the researchers wrote. “While a healthy and active lifestyle should be encouraged for all children, our results emphasize the need for pediatric HSCT survivors to be educated on their increased risk for UV‐related skin cancers, counseled on avoidance of intentional tanning, and advised on the importance of sun protection behaviors in an effort to improve long-term outcomes.”

The researchers noted that transplant recipients were significantly more likely to have had a full body skin exam from a health care professional than were individuals in the control group (61.2% vs. 4.7%) and were more likely to have done a self-check or been checked by a partner in the previous year.

The study was supported by the Society for Pediatric Dermatology, the Dermatology Foundation, the National Institutes of Health, and the Dr. Miriam and Sheldon G. Adelson Medical Research Foundation. One author declared a financial interest in a company developing a dermatological product. No other conflicts of interest were declared.

SOURCE: Li EB et al. Pediatr Dermatol. 2019 Aug 13. doi: 10.1111/pde.13984.

Young people who have received allogeneic hematopoietic stem cell transplants (HSCTs) are more likely to wear hats, sunscreen and other sun protection, but still intentionally tan and experience sunburn at the same rate as their peers, new research suggests.

Yuri Arcurs/Fotolia

In a survey‐based, cross‐sectional cohort study, researchers compared sun-protection behaviors and sun exposure in 85 children aged 21 years and younger who had undergone HSCT and 85 age-, sex-, and skin type–matched controls. The findings were published in Pediatric Dermatology.

HSCT recipients have a higher risk of long-term complications such as skin cancer, for which sun exposure is a major modifiable environmental risk factor.

“Therefore, consistent sun avoidance and protection as well as regular dermatologic evaluations are important for HSCT recipients,” wrote Edward B. Li, PhD, from Harvard Medical School, Boston, and coauthors.

The survey found no significant difference between the transplant and control group in the amount of intentional sun exposure, such as the amount of time spent outside on weekdays and weekends during the peak sun intensity hours of 10 a.m. and 4 p.m. More than one in five transplant recipients (21.2%) reported spending at least 3 hours a day outside between 10 a.m. and 4 p.m. on weekdays, as did 36.5% of transplant recipients on weekends.

There were also no significant differences between the two groups in terms of time spent tanning, either in the sun or in a tanning bed. Additionally, a similar number of transplant recipients and controls experienced one or more red or painful sunburns in the past year (25.9% vs. 27.1%).

However, transplant patients did practice better sun protection behaviors than did the control group, with 60% reporting that they always wore sunscreen, compared with 29.4% of controls. The transplant recipients were also significantly more likely to wear sunglasses and a hat and to stay in the shade or use an umbrella.

“While these data may reflect that HSCT patients are not practicing adequate sun avoidance, it may also suggest that these long‐term survivors are able to enjoy being outdoors as much as their peers and have a similar desire to have a tanned appearance,” the researchers wrote. “While a healthy and active lifestyle should be encouraged for all children, our results emphasize the need for pediatric HSCT survivors to be educated on their increased risk for UV‐related skin cancers, counseled on avoidance of intentional tanning, and advised on the importance of sun protection behaviors in an effort to improve long-term outcomes.”

The researchers noted that transplant recipients were significantly more likely to have had a full body skin exam from a health care professional than were individuals in the control group (61.2% vs. 4.7%) and were more likely to have done a self-check or been checked by a partner in the previous year.

The study was supported by the Society for Pediatric Dermatology, the Dermatology Foundation, the National Institutes of Health, and the Dr. Miriam and Sheldon G. Adelson Medical Research Foundation. One author declared a financial interest in a company developing a dermatological product. No other conflicts of interest were declared.

SOURCE: Li EB et al. Pediatr Dermatol. 2019 Aug 13. doi: 10.1111/pde.13984.

BARCELONA – Molecular testing to guide treatment in patients with lung cancer remains underused, and awareness of related evidence-based guidelines is suboptimal, results of an international survey suggest.

Sharon Worcester/ MDedge News

Overall, 61% of the 2,537 respondents from 102 countries and across multiple relevant medical specialties reported that molecular testing rates in their country were less than 50%, with the lowest rates reported in Latin America. And 33% of those requesting molecular testing said they were unaware of the most updated guidelines supporting the use of such testing in lung cancer, Matthew Smeltzer, PhD, of the University of Memphis (Tenn.) reported during a press conference at the World Conference on Lung Cancer.

The findings from the International Association for the Study of Lung Cancer (IASLC) Global Survey on Molecular Testing in Lung Cancer also showed that 41% of respondents who perform or interpret molecular testing assays report being dissatisfied with the conditions of molecular testing in their country, 17% said they feel that patients are not satisfied, and 35% said they aren’t sure about the state of testing in their country.

Specific concerns reported by respondents included trouble understanding results, the time it takes to receive the results, and the reliability of samples.

The top five barriers to molecular testing included cost, quality, access, awareness, and time, Dr. Smeltzer said at the meeting which is sponsored by the IASLC.

“These five were the same five top barriers in each region of the world,” he said, noting that the ordering of the barriers differed somewhat among regions.

The survey included a seven-question introduction, with 32 additional questions for respondents who request tests and treat patients, 45 questions on performing and interpreting assays, and 24 questions on tissue acquisition. Additionally, all respondents were asked to list barriers that impede their country’s ability to offer molecular testing.

“I’d say we got a pretty good geographic distribution of responses; 56% of these responses were from developing countries, 44% from developed countries,” he said, noting that medical oncologists constituted the highest percentage of respondents, followed by pulmonologists, thoracic surgeons, pathologists, and other scientists.

When asked specifically what would prompt molecular testing, respondents most often listed adenocarcinoma, never-smoker status, female gender, and young age, Dr. Smeltzer said.

“Overall, we’re still finding that many in the lung cancer community are not satisfied with the current state of molecular testing. We’ve got suboptimal awareness of the evidence-based guidelines. We have barriers that remain to molecular testing, which we’ve identified, and [we’re] recommending continuous education around molecular testing, and that should be intensified on a national and international level to ensure that patients receive optimal therapy,” he concluded.

The IASLC survey was funded by AstraZeneca. Dr. Smeltzer reported receiving research support from the Bristol Myers Squibb Foundation.

sworcester@mdedge.com

BARCELONA – Molecular testing to guide treatment in patients with lung cancer remains underused, and awareness of related evidence-based guidelines is suboptimal, results of an international survey suggest.

Sharon Worcester/ MDedge News

Overall, 61% of the 2,537 respondents from 102 countries and across multiple relevant medical specialties reported that molecular testing rates in their country were less than 50%, with the lowest rates reported in Latin America. And 33% of those requesting molecular testing said they were unaware of the most updated guidelines supporting the use of such testing in lung cancer, Matthew Smeltzer, PhD, of the University of Memphis (Tenn.) reported during a press conference at the World Conference on Lung Cancer.

The findings from the International Association for the Study of Lung Cancer (IASLC) Global Survey on Molecular Testing in Lung Cancer also showed that 41% of respondents who perform or interpret molecular testing assays report being dissatisfied with the conditions of molecular testing in their country, 17% said they feel that patients are not satisfied, and 35% said they aren’t sure about the state of testing in their country.

Specific concerns reported by respondents included trouble understanding results, the time it takes to receive the results, and the reliability of samples.

The top five barriers to molecular testing included cost, quality, access, awareness, and time, Dr. Smeltzer said at the meeting which is sponsored by the IASLC.

“These five were the same five top barriers in each region of the world,” he said, noting that the ordering of the barriers differed somewhat among regions.

The survey included a seven-question introduction, with 32 additional questions for respondents who request tests and treat patients, 45 questions on performing and interpreting assays, and 24 questions on tissue acquisition. Additionally, all respondents were asked to list barriers that impede their country’s ability to offer molecular testing.

“I’d say we got a pretty good geographic distribution of responses; 56% of these responses were from developing countries, 44% from developed countries,” he said, noting that medical oncologists constituted the highest percentage of respondents, followed by pulmonologists, thoracic surgeons, pathologists, and other scientists.

When asked specifically what would prompt molecular testing, respondents most often listed adenocarcinoma, never-smoker status, female gender, and young age, Dr. Smeltzer said.

“Overall, we’re still finding that many in the lung cancer community are not satisfied with the current state of molecular testing. We’ve got suboptimal awareness of the evidence-based guidelines. We have barriers that remain to molecular testing, which we’ve identified, and [we’re] recommending continuous education around molecular testing, and that should be intensified on a national and international level to ensure that patients receive optimal therapy,” he concluded.

The IASLC survey was funded by AstraZeneca. Dr. Smeltzer reported receiving research support from the Bristol Myers Squibb Foundation.

sworcester@mdedge.com

BARCELONA – Molecular testing to guide treatment in patients with lung cancer remains underused, and awareness of related evidence-based guidelines is suboptimal, results of an international survey suggest.

Sharon Worcester/ MDedge News

Overall, 61% of the 2,537 respondents from 102 countries and across multiple relevant medical specialties reported that molecular testing rates in their country were less than 50%, with the lowest rates reported in Latin America. And 33% of those requesting molecular testing said they were unaware of the most updated guidelines supporting the use of such testing in lung cancer, Matthew Smeltzer, PhD, of the University of Memphis (Tenn.) reported during a press conference at the World Conference on Lung Cancer.

The findings from the International Association for the Study of Lung Cancer (IASLC) Global Survey on Molecular Testing in Lung Cancer also showed that 41% of respondents who perform or interpret molecular testing assays report being dissatisfied with the conditions of molecular testing in their country, 17% said they feel that patients are not satisfied, and 35% said they aren’t sure about the state of testing in their country.

Specific concerns reported by respondents included trouble understanding results, the time it takes to receive the results, and the reliability of samples.

The top five barriers to molecular testing included cost, quality, access, awareness, and time, Dr. Smeltzer said at the meeting which is sponsored by the IASLC.

“These five were the same five top barriers in each region of the world,” he said, noting that the ordering of the barriers differed somewhat among regions.

The survey included a seven-question introduction, with 32 additional questions for respondents who request tests and treat patients, 45 questions on performing and interpreting assays, and 24 questions on tissue acquisition. Additionally, all respondents were asked to list barriers that impede their country’s ability to offer molecular testing.

“I’d say we got a pretty good geographic distribution of responses; 56% of these responses were from developing countries, 44% from developed countries,” he said, noting that medical oncologists constituted the highest percentage of respondents, followed by pulmonologists, thoracic surgeons, pathologists, and other scientists.

When asked specifically what would prompt molecular testing, respondents most often listed adenocarcinoma, never-smoker status, female gender, and young age, Dr. Smeltzer said.

“Overall, we’re still finding that many in the lung cancer community are not satisfied with the current state of molecular testing. We’ve got suboptimal awareness of the evidence-based guidelines. We have barriers that remain to molecular testing, which we’ve identified, and [we’re] recommending continuous education around molecular testing, and that should be intensified on a national and international level to ensure that patients receive optimal therapy,” he concluded.

The IASLC survey was funded by AstraZeneca. Dr. Smeltzer reported receiving research support from the Bristol Myers Squibb Foundation.

sworcester@mdedge.com

BARCELONA – All cancer patients should be screened for tobacco use and advised about the benefits of tobacco cessation, and evidence-based tobacco cessation assistance should be provided to those who continue using tobacco, according to a new declaration from the International Association for the Study of Lung Cancer (IASLC).

Sharon Worcester/ MDedge News

Such assistance should be “routinely and integrally incorporated into multidisciplinary cancer care for the patients and their family members,” according to the declaration, which was developed by the IASLC Tobacco Control and Smoking Cessation Committee in part to increase physician involvement in tobacco control and was officially released at the World Conference on Lung Cancer.

“The consequences of smoking continuation by cancer patients and cancer survivors are many,” presenting author Jacek Jassem, MD, PhD, professor and head of the department of oncology and radiotherapy at the Medical University of Gdansk (Poland), said during a press conference at the meeting, which is sponsored by the IASLC.

Smoking after a cancer diagnosis increases cancer-related and overall mortality, the risk of developing additional cancers, and the risk of treatment toxicity, he explained.

“And of course [continued smoking is] associated with much higher treatment costs, mostly due to complications,” he added.

The IASLC addresses both the challenges and opportunities associated with tobacco cessation, including the fact that most cancer patients who smoke continue to do so during and after treatment and that health care providers often don’t provide patients with cessation assistance to help them quit.

In addition to the screening and tobacco cessation assistance recommendations, the declaration also states the following:

• Educational programs regarding cancer management should include tobacco cessation training, empathetic communication around the history of tobacco use and cessation, and utilization of existing evidence-based tobacco cessation resources.
• Smoking cessation counseling and treatment should be a reimbursable service.
• Smoking status, both initially and during the study, should be a required data element for all prospective clinical studies.
• Clinical trials of patients with cancer should consider designs that could also determine the most effective tobacco cessation interventions.

Dr. Jassem is on the speakers bureau for MSD, Takeda, BMS, Astra-Zeneca, and Pfizer.

sworcester@mdedge.com

BARCELONA – All cancer patients should be screened for tobacco use and advised about the benefits of tobacco cessation, and evidence-based tobacco cessation assistance should be provided to those who continue using tobacco, according to a new declaration from the International Association for the Study of Lung Cancer (IASLC).

Sharon Worcester/ MDedge News

Such assistance should be “routinely and integrally incorporated into multidisciplinary cancer care for the patients and their family members,” according to the declaration, which was developed by the IASLC Tobacco Control and Smoking Cessation Committee in part to increase physician involvement in tobacco control and was officially released at the World Conference on Lung Cancer.

“The consequences of smoking continuation by cancer patients and cancer survivors are many,” presenting author Jacek Jassem, MD, PhD, professor and head of the department of oncology and radiotherapy at the Medical University of Gdansk (Poland), said during a press conference at the meeting, which is sponsored by the IASLC.

Smoking after a cancer diagnosis increases cancer-related and overall mortality, the risk of developing additional cancers, and the risk of treatment toxicity, he explained.

“And of course [continued smoking is] associated with much higher treatment costs, mostly due to complications,” he added.

The IASLC addresses both the challenges and opportunities associated with tobacco cessation, including the fact that most cancer patients who smoke continue to do so during and after treatment and that health care providers often don’t provide patients with cessation assistance to help them quit.

In addition to the screening and tobacco cessation assistance recommendations, the declaration also states the following:

• Educational programs regarding cancer management should include tobacco cessation training, empathetic communication around the history of tobacco use and cessation, and utilization of existing evidence-based tobacco cessation resources.
• Smoking cessation counseling and treatment should be a reimbursable service.
• Smoking status, both initially and during the study, should be a required data element for all prospective clinical studies.
• Clinical trials of patients with cancer should consider designs that could also determine the most effective tobacco cessation interventions.

Dr. Jassem is on the speakers bureau for MSD, Takeda, BMS, Astra-Zeneca, and Pfizer.

sworcester@mdedge.com

BARCELONA – All cancer patients should be screened for tobacco use and advised about the benefits of tobacco cessation, and evidence-based tobacco cessation assistance should be provided to those who continue using tobacco, according to a new declaration from the International Association for the Study of Lung Cancer (IASLC).

Sharon Worcester/ MDedge News

Such assistance should be “routinely and integrally incorporated into multidisciplinary cancer care for the patients and their family members,” according to the declaration, which was developed by the IASLC Tobacco Control and Smoking Cessation Committee in part to increase physician involvement in tobacco control and was officially released at the World Conference on Lung Cancer.

“The consequences of smoking continuation by cancer patients and cancer survivors are many,” presenting author Jacek Jassem, MD, PhD, professor and head of the department of oncology and radiotherapy at the Medical University of Gdansk (Poland), said during a press conference at the meeting, which is sponsored by the IASLC.

Smoking after a cancer diagnosis increases cancer-related and overall mortality, the risk of developing additional cancers, and the risk of treatment toxicity, he explained.

“And of course [continued smoking is] associated with much higher treatment costs, mostly due to complications,” he added.

The IASLC addresses both the challenges and opportunities associated with tobacco cessation, including the fact that most cancer patients who smoke continue to do so during and after treatment and that health care providers often don’t provide patients with cessation assistance to help them quit.

In addition to the screening and tobacco cessation assistance recommendations, the declaration also states the following:

• Educational programs regarding cancer management should include tobacco cessation training, empathetic communication around the history of tobacco use and cessation, and utilization of existing evidence-based tobacco cessation resources.
• Smoking cessation counseling and treatment should be a reimbursable service.
• Smoking status, both initially and during the study, should be a required data element for all prospective clinical studies.
• Clinical trials of patients with cancer should consider designs that could also determine the most effective tobacco cessation interventions.

Dr. Jassem is on the speakers bureau for MSD, Takeda, BMS, Astra-Zeneca, and Pfizer.

sworcester@mdedge.com

Immune dysregulation is a substantial feature of hidradenitis suppurativa in African American patients, according to data from 16 adults.

The etiology of hidradenitis suppurativa (HS) remains unknown, but neutrophils are prominent in affected areas of skin, wrote Angel S. Byrd, MD, PhD, of Johns Hopkins University, Baltimore, and colleagues.

“Among the several functions of neutrophils, these cells have the ability to form neutrophil extracellular traps (NETs) after exposure to certain microbes or sterile stimuli,” the researchers noted. These NETs are weblike structures that have been shown to activate several types of immune responses and promote inflammation.

In a study published in Science Translational Medicine, the researchers analyzed biospecimens from African American HS patients. They determined that NET structures were present in the lesional skin of HS patients, but not in control skin. Additionally, serum from HS patients did not properly degrade healthy control NETs in an in vitro examination.

A significant correlation (P less than .0001) occurred between the amount of NETs released in hidradenitis suppurativa lesions and hidradenitis suppurativa severity.

“Together, these results indicate that NET formation is enhanced in HS, both systemically and in lesional skin, in association with disease severity and that NET degradation mechanisms may be impaired in this disease,” the researchers wrote.

The researchers also found that total immunoglobulin G was significantly increased in the sera of HS patients, compared with that of healthy volunteers (P = .0011), and that enzymes involved in skin citrullination were elevated in the skin of HS patients, compared with controls’ skin.

Previous studies have shown that NETs can promote type I interferon (IFN) signatures in the blood of patients with autoimmune and chronic inflammatory conditions, and in this study, several type I IFN–regulated genes had increased expression in HS skin, including IFI44L, MX1, CXCL10, RSAD2, and IFI27.

The study findings were limited by the small sample size and homogenous population, the researchers noted. Future research should include “the role that neutrophil/type I IFN dysregulation plays in the clinical manifestations of the disease, and how these abnormalities in immune responses regulate other innate and adaptive immune cell types in HS,” which could affect the development of new treatments, they wrote.

The study was supported in part by the National Institutes of Health, the Johns Hopkins School of Medicine Physician Scientist Training Program, and the Danby Hidradenitis Suppurativa Foundation. Dr. Byrd disclosed participating in the Johns Hopkins Ethnic Skin Fellowship, which was supported in part by Valeant. Dr. Byrd also disclosed serving as a former investigator for Eli Lilly.

SOURCE: Byrd AS et al. Sci Transl Med. 2019 Sep 4. doi: 10.1126/scitranslmed.aav5908.

Immune dysregulation is a substantial feature of hidradenitis suppurativa in African American patients, according to data from 16 adults.

The etiology of hidradenitis suppurativa (HS) remains unknown, but neutrophils are prominent in affected areas of skin, wrote Angel S. Byrd, MD, PhD, of Johns Hopkins University, Baltimore, and colleagues.

“Among the several functions of neutrophils, these cells have the ability to form neutrophil extracellular traps (NETs) after exposure to certain microbes or sterile stimuli,” the researchers noted. These NETs are weblike structures that have been shown to activate several types of immune responses and promote inflammation.

In a study published in Science Translational Medicine, the researchers analyzed biospecimens from African American HS patients. They determined that NET structures were present in the lesional skin of HS patients, but not in control skin. Additionally, serum from HS patients did not properly degrade healthy control NETs in an in vitro examination.

A significant correlation (P less than .0001) occurred between the amount of NETs released in hidradenitis suppurativa lesions and hidradenitis suppurativa severity.

“Together, these results indicate that NET formation is enhanced in HS, both systemically and in lesional skin, in association with disease severity and that NET degradation mechanisms may be impaired in this disease,” the researchers wrote.

The researchers also found that total immunoglobulin G was significantly increased in the sera of HS patients, compared with that of healthy volunteers (P = .0011), and that enzymes involved in skin citrullination were elevated in the skin of HS patients, compared with controls’ skin.

Previous studies have shown that NETs can promote type I interferon (IFN) signatures in the blood of patients with autoimmune and chronic inflammatory conditions, and in this study, several type I IFN–regulated genes had increased expression in HS skin, including IFI44L, MX1, CXCL10, RSAD2, and IFI27.

The study findings were limited by the small sample size and homogenous population, the researchers noted. Future research should include “the role that neutrophil/type I IFN dysregulation plays in the clinical manifestations of the disease, and how these abnormalities in immune responses regulate other innate and adaptive immune cell types in HS,” which could affect the development of new treatments, they wrote.

The study was supported in part by the National Institutes of Health, the Johns Hopkins School of Medicine Physician Scientist Training Program, and the Danby Hidradenitis Suppurativa Foundation. Dr. Byrd disclosed participating in the Johns Hopkins Ethnic Skin Fellowship, which was supported in part by Valeant. Dr. Byrd also disclosed serving as a former investigator for Eli Lilly.

SOURCE: Byrd AS et al. Sci Transl Med. 2019 Sep 4. doi: 10.1126/scitranslmed.aav5908.

Immune dysregulation is a substantial feature of hidradenitis suppurativa in African American patients, according to data from 16 adults.

The etiology of hidradenitis suppurativa (HS) remains unknown, but neutrophils are prominent in affected areas of skin, wrote Angel S. Byrd, MD, PhD, of Johns Hopkins University, Baltimore, and colleagues.

“Among the several functions of neutrophils, these cells have the ability to form neutrophil extracellular traps (NETs) after exposure to certain microbes or sterile stimuli,” the researchers noted. These NETs are weblike structures that have been shown to activate several types of immune responses and promote inflammation.

In a study published in Science Translational Medicine, the researchers analyzed biospecimens from African American HS patients. They determined that NET structures were present in the lesional skin of HS patients, but not in control skin. Additionally, serum from HS patients did not properly degrade healthy control NETs in an in vitro examination.

A significant correlation (P less than .0001) occurred between the amount of NETs released in hidradenitis suppurativa lesions and hidradenitis suppurativa severity.

“Together, these results indicate that NET formation is enhanced in HS, both systemically and in lesional skin, in association with disease severity and that NET degradation mechanisms may be impaired in this disease,” the researchers wrote.

The researchers also found that total immunoglobulin G was significantly increased in the sera of HS patients, compared with that of healthy volunteers (P = .0011), and that enzymes involved in skin citrullination were elevated in the skin of HS patients, compared with controls’ skin.

Previous studies have shown that NETs can promote type I interferon (IFN) signatures in the blood of patients with autoimmune and chronic inflammatory conditions, and in this study, several type I IFN–regulated genes had increased expression in HS skin, including IFI44L, MX1, CXCL10, RSAD2, and IFI27.

The study findings were limited by the small sample size and homogenous population, the researchers noted. Future research should include “the role that neutrophil/type I IFN dysregulation plays in the clinical manifestations of the disease, and how these abnormalities in immune responses regulate other innate and adaptive immune cell types in HS,” which could affect the development of new treatments, they wrote.

The study was supported in part by the National Institutes of Health, the Johns Hopkins School of Medicine Physician Scientist Training Program, and the Danby Hidradenitis Suppurativa Foundation. Dr. Byrd disclosed participating in the Johns Hopkins Ethnic Skin Fellowship, which was supported in part by Valeant. Dr. Byrd also disclosed serving as a former investigator for Eli Lilly.

SOURCE: Byrd AS et al. Sci Transl Med. 2019 Sep 4. doi: 10.1126/scitranslmed.aav5908.

For patients with localized renal cell carcinoma (RCC), stereotactic ablative body radiotherapy (SABR) may be an effective alternative to surgery, according to findings from a retrospective study.

Patients with smaller tumors and nonmetastatic disease achieved the best outcomes with SABR, reported lead author Rodney E. Wegner, MD, of Allegheny Health Network Cancer Institute, Pittsburgh, and colleagues.

“Radiation therapy is often overlooked in [RCC] as historic preclinical data reported RCC as being relatively radioresistant to external beam radiation at conventional doses,” the investigators wrote in Advances in Radiation Oncology. However, SABR may be able to overcome this resistance by delivering “highly conformal dose escalated radiation,” the investigators noted, citing two recent reports from the International Radiosurgery Oncology Consortium for Kidney (IROCK) that showed promising results (J Urol. 2019 Jun;201[6]:1097-104 and Cancer. 2018 Mar 1;124[5]:934-42).

The present study included 347 patients with RCC from the National Cancer Database who were treated with SABR and not surgery. Most patients (94%) did not have systemic therapy. Similar proportions lacked lymph node involvement (97%) or distant metastasis (93%). About three-quarters of patients (76%) had T1 disease. The median SABR dose was 45 Gy, ranging from 35 to 54 Gy, most frequently given in three fractions.

After a median follow-up of 36 months, ranging from 1 to 156 months, median overall survival across all patients was 58 months. SABR was most effective for patients with nonmetastatic disease who had smaller tumors.

An inverse correlation between tumor size and overall survival was apparent given that patients with tumors 2.5 cm or smaller had the longest median overall survival, at 92 months, with decrements in survival as tumors got larger. Survival dropped to 88 months for tumors 2.6-3.5 cm, 44 months for tumors 3.5-5.0 cm, and finally to 26 months for tumors larger than 5.0 cm. In addition to tumor size and metastatic disease, age was a risk factor for shorter survival.

“The results presented demonstrate excellent post-SABR outcomes, with median overall survival in the range of 7-8 years for smaller lesions,” the investigators wrote. “This is particularly impressive considering that many of these patients were likely medically inoperable.”

The researchers noted that most of kidney SABR is done at academic centers, which highlights the importance of appropriate technology and training for delivering this treatment.

“Further prospective research is needed to verify its safety and efficacy,” the investigators concluded.

No external funding was provided for the project and the investigators reported no conflicts of interest.

SOURCE: Wegner RE et al. Adv Rad Onc. 2019 Aug 8. doi: 10.1016/j.adro.2019.07.018.
 

For patients with localized renal cell carcinoma (RCC), stereotactic ablative body radiotherapy (SABR) may be an effective alternative to surgery, according to findings from a retrospective study.

Patients with smaller tumors and nonmetastatic disease achieved the best outcomes with SABR, reported lead author Rodney E. Wegner, MD, of Allegheny Health Network Cancer Institute, Pittsburgh, and colleagues.

“Radiation therapy is often overlooked in [RCC] as historic preclinical data reported RCC as being relatively radioresistant to external beam radiation at conventional doses,” the investigators wrote in Advances in Radiation Oncology. However, SABR may be able to overcome this resistance by delivering “highly conformal dose escalated radiation,” the investigators noted, citing two recent reports from the International Radiosurgery Oncology Consortium for Kidney (IROCK) that showed promising results (J Urol. 2019 Jun;201[6]:1097-104 and Cancer. 2018 Mar 1;124[5]:934-42).

The present study included 347 patients with RCC from the National Cancer Database who were treated with SABR and not surgery. Most patients (94%) did not have systemic therapy. Similar proportions lacked lymph node involvement (97%) or distant metastasis (93%). About three-quarters of patients (76%) had T1 disease. The median SABR dose was 45 Gy, ranging from 35 to 54 Gy, most frequently given in three fractions.

After a median follow-up of 36 months, ranging from 1 to 156 months, median overall survival across all patients was 58 months. SABR was most effective for patients with nonmetastatic disease who had smaller tumors.

An inverse correlation between tumor size and overall survival was apparent given that patients with tumors 2.5 cm or smaller had the longest median overall survival, at 92 months, with decrements in survival as tumors got larger. Survival dropped to 88 months for tumors 2.6-3.5 cm, 44 months for tumors 3.5-5.0 cm, and finally to 26 months for tumors larger than 5.0 cm. In addition to tumor size and metastatic disease, age was a risk factor for shorter survival.

“The results presented demonstrate excellent post-SABR outcomes, with median overall survival in the range of 7-8 years for smaller lesions,” the investigators wrote. “This is particularly impressive considering that many of these patients were likely medically inoperable.”

The researchers noted that most of kidney SABR is done at academic centers, which highlights the importance of appropriate technology and training for delivering this treatment.

“Further prospective research is needed to verify its safety and efficacy,” the investigators concluded.

No external funding was provided for the project and the investigators reported no conflicts of interest.

SOURCE: Wegner RE et al. Adv Rad Onc. 2019 Aug 8. doi: 10.1016/j.adro.2019.07.018.
 

For patients with localized renal cell carcinoma (RCC), stereotactic ablative body radiotherapy (SABR) may be an effective alternative to surgery, according to findings from a retrospective study.

Patients with smaller tumors and nonmetastatic disease achieved the best outcomes with SABR, reported lead author Rodney E. Wegner, MD, of Allegheny Health Network Cancer Institute, Pittsburgh, and colleagues.

“Radiation therapy is often overlooked in [RCC] as historic preclinical data reported RCC as being relatively radioresistant to external beam radiation at conventional doses,” the investigators wrote in Advances in Radiation Oncology. However, SABR may be able to overcome this resistance by delivering “highly conformal dose escalated radiation,” the investigators noted, citing two recent reports from the International Radiosurgery Oncology Consortium for Kidney (IROCK) that showed promising results (J Urol. 2019 Jun;201[6]:1097-104 and Cancer. 2018 Mar 1;124[5]:934-42).

The present study included 347 patients with RCC from the National Cancer Database who were treated with SABR and not surgery. Most patients (94%) did not have systemic therapy. Similar proportions lacked lymph node involvement (97%) or distant metastasis (93%). About three-quarters of patients (76%) had T1 disease. The median SABR dose was 45 Gy, ranging from 35 to 54 Gy, most frequently given in three fractions.

After a median follow-up of 36 months, ranging from 1 to 156 months, median overall survival across all patients was 58 months. SABR was most effective for patients with nonmetastatic disease who had smaller tumors.

An inverse correlation between tumor size and overall survival was apparent given that patients with tumors 2.5 cm or smaller had the longest median overall survival, at 92 months, with decrements in survival as tumors got larger. Survival dropped to 88 months for tumors 2.6-3.5 cm, 44 months for tumors 3.5-5.0 cm, and finally to 26 months for tumors larger than 5.0 cm. In addition to tumor size and metastatic disease, age was a risk factor for shorter survival.

“The results presented demonstrate excellent post-SABR outcomes, with median overall survival in the range of 7-8 years for smaller lesions,” the investigators wrote. “This is particularly impressive considering that many of these patients were likely medically inoperable.”

The researchers noted that most of kidney SABR is done at academic centers, which highlights the importance of appropriate technology and training for delivering this treatment.

“Further prospective research is needed to verify its safety and efficacy,” the investigators concluded.

No external funding was provided for the project and the investigators reported no conflicts of interest.

SOURCE: Wegner RE et al. Adv Rad Onc. 2019 Aug 8. doi: 10.1016/j.adro.2019.07.018.
 

NEW ORLEANS – An arm cuff that’s one size too small in children will artificially elevate blood pressure 5 mm Hg, while one size too large will drop it 5 mm Hg, according to investigators from Columbia University in New York.

M. Alexander Otto/MDedge News

There are five cuff sizes in pediatrics, depending on a child’s arm circumference. Ideally, it’s measured beforehand so the right cuff size is used, but that doesn’t always happen in everyday practice.

Sometimes, clinicians just estimate arm size before choosing a cuff or opt for the medium-sized cuff in most kids; other times, the correct size has gone missing, said lead investigator Ruchi Gupta Mahajan, MD, a pediatric nephrology fellow at Columbia.

For those situations, she and her colleagues wanted to quantify how much the wrong cuff size throws off blood pressure readings in children, something that’s been done before in adult medicine, but not in pediatrics.

The idea was to give clinicians a decent estimate of true blood pressure even when the cuff isn’t quite right, something that’s particularly important with an increasing emphasis on catching hypertension as early as possible in children, she said.

After her subjects sat quietly for 10 minutes, Dr. Mahajan took automated blood pressure readings on 137 children; once with the right cuff size, once with a cuff one size too small, and once with a cuff one size too big, with a minute apart between readings.

The children were aged 4-12 years old and were in the office for wellness visits. None of them had heart or kidney disease, and none were on steroids or any other medications that affect blood pressure. There were a few more boys than girls, and almost all the children were Hispanic.

Overall, systolic blood pressure was an average of 5 mm Hg less with the larger cuff and 5 mm Hg more with the smaller cuff. The finding was the same in both girls and boys, and it held across age groups and in under, over, and normal weight children.

“I was really surprised there was no difference between ages, 4-12 years of age, its just a single number: 5. [Even] if [you] don’t have the appropriate cuff size,” the finding means that it’s still possible to have a good estimate of blood pressure, Dr. Mahajan said at the joint scientific sessions of the American Heart Association (AHA) Council on Hypertension, AHA Council on Kidney in Cardiovascular Disease, and American Society of Hypertension.

Meanwhile, cuff size didn’t have any statistically significant effect on diastolic pressure.

There was no outside funding for the study and Dr. Mahajan reported having no disclosures.

aotto@mdedge.com

SOURCE: Mahajan RG et al. Joint Hypertension 2019, Abstract P182.

NEW ORLEANS – An arm cuff that’s one size too small in children will artificially elevate blood pressure 5 mm Hg, while one size too large will drop it 5 mm Hg, according to investigators from Columbia University in New York.

M. Alexander Otto/MDedge News

There are five cuff sizes in pediatrics, depending on a child’s arm circumference. Ideally, it’s measured beforehand so the right cuff size is used, but that doesn’t always happen in everyday practice.

Sometimes, clinicians just estimate arm size before choosing a cuff or opt for the medium-sized cuff in most kids; other times, the correct size has gone missing, said lead investigator Ruchi Gupta Mahajan, MD, a pediatric nephrology fellow at Columbia.

For those situations, she and her colleagues wanted to quantify how much the wrong cuff size throws off blood pressure readings in children, something that’s been done before in adult medicine, but not in pediatrics.

The idea was to give clinicians a decent estimate of true blood pressure even when the cuff isn’t quite right, something that’s particularly important with an increasing emphasis on catching hypertension as early as possible in children, she said.

After her subjects sat quietly for 10 minutes, Dr. Mahajan took automated blood pressure readings on 137 children; once with the right cuff size, once with a cuff one size too small, and once with a cuff one size too big, with a minute apart between readings.

The children were aged 4-12 years old and were in the office for wellness visits. None of them had heart or kidney disease, and none were on steroids or any other medications that affect blood pressure. There were a few more boys than girls, and almost all the children were Hispanic.

Overall, systolic blood pressure was an average of 5 mm Hg less with the larger cuff and 5 mm Hg more with the smaller cuff. The finding was the same in both girls and boys, and it held across age groups and in under, over, and normal weight children.

“I was really surprised there was no difference between ages, 4-12 years of age, its just a single number: 5. [Even] if [you] don’t have the appropriate cuff size,” the finding means that it’s still possible to have a good estimate of blood pressure, Dr. Mahajan said at the joint scientific sessions of the American Heart Association (AHA) Council on Hypertension, AHA Council on Kidney in Cardiovascular Disease, and American Society of Hypertension.

Meanwhile, cuff size didn’t have any statistically significant effect on diastolic pressure.

There was no outside funding for the study and Dr. Mahajan reported having no disclosures.

aotto@mdedge.com

SOURCE: Mahajan RG et al. Joint Hypertension 2019, Abstract P182.

NEW ORLEANS – An arm cuff that’s one size too small in children will artificially elevate blood pressure 5 mm Hg, while one size too large will drop it 5 mm Hg, according to investigators from Columbia University in New York.

M. Alexander Otto/MDedge News

There are five cuff sizes in pediatrics, depending on a child’s arm circumference. Ideally, it’s measured beforehand so the right cuff size is used, but that doesn’t always happen in everyday practice.

Sometimes, clinicians just estimate arm size before choosing a cuff or opt for the medium-sized cuff in most kids; other times, the correct size has gone missing, said lead investigator Ruchi Gupta Mahajan, MD, a pediatric nephrology fellow at Columbia.

For those situations, she and her colleagues wanted to quantify how much the wrong cuff size throws off blood pressure readings in children, something that’s been done before in adult medicine, but not in pediatrics.

The idea was to give clinicians a decent estimate of true blood pressure even when the cuff isn’t quite right, something that’s particularly important with an increasing emphasis on catching hypertension as early as possible in children, she said.

After her subjects sat quietly for 10 minutes, Dr. Mahajan took automated blood pressure readings on 137 children; once with the right cuff size, once with a cuff one size too small, and once with a cuff one size too big, with a minute apart between readings.

The children were aged 4-12 years old and were in the office for wellness visits. None of them had heart or kidney disease, and none were on steroids or any other medications that affect blood pressure. There were a few more boys than girls, and almost all the children were Hispanic.

Overall, systolic blood pressure was an average of 5 mm Hg less with the larger cuff and 5 mm Hg more with the smaller cuff. The finding was the same in both girls and boys, and it held across age groups and in under, over, and normal weight children.

“I was really surprised there was no difference between ages, 4-12 years of age, its just a single number: 5. [Even] if [you] don’t have the appropriate cuff size,” the finding means that it’s still possible to have a good estimate of blood pressure, Dr. Mahajan said at the joint scientific sessions of the American Heart Association (AHA) Council on Hypertension, AHA Council on Kidney in Cardiovascular Disease, and American Society of Hypertension.

Meanwhile, cuff size didn’t have any statistically significant effect on diastolic pressure.

There was no outside funding for the study and Dr. Mahajan reported having no disclosures.

aotto@mdedge.com

SOURCE: Mahajan RG et al. Joint Hypertension 2019, Abstract P182.

NEW ORLEANS – Visceral adiposity, but not body mass index or total body fat, significantly correlated with elevated 24-hour ambulatory systolic blood pressure, greater systolic variability, and masked hypertension in a study from the University of Pennsylvania, Philadelphia.

Subjects in the highest quartile of visceral fat had a 6.3-fold greater odds of masked hypertension – normal in the office, but high at home – compared with those in the lowest quartile (95% confidence interval, 1.2-33.1).

The study findings suggest that central obesity, in particular, should trigger 24-hour ambulatory blood pressure monitoring (ABPM). “Every obese person should get a 24-hour” ABPM, but “we really need to be pushing [it] in people who have central adiposity. These are the patients ... we really need to focus on” because of the risk of masked hypertension, a “ticking time bomb” that greatly increases the risk of cardiovascular events, said lead investigator Jordana B. Cohen, MD, an assistant professor of medicine at the university.

The study also helps explain why body mass index (BMI) hasn’t been consistently linked to masked hypertension in previous studies; some studies likely included subjects with high BMIs but not central obesity.

Waist circumference, a marker of visceral adiposity, also correlated with elevated 24-hour systolic pressure and greater variability, but a trend for masked hypertension was not statistically significant, Dr. Cohen reported at the joint scientific sessions of the American Heart Association (AHA) Council on Hypertension, AHA Council on Kidney in Cardiovascular Disease, and American Society of Hypertension.

It’s long been known that visceral fat – fat around the abdominal organs – is metabolically active and associated with greater cardiovascular risk, but its relationship to blood pressure hadn’t been well described, so Dr. Cohen and her team decided to take a look.

They ran whole body dual x-ray absorptiometry scans on 96 hypertensive adults on a stable dose of one antihypertensive drug for at least 2 months and correlated the findings with ABPM. Subjects were an average of 58 years old, almost 60% were women, almost half were black, and 54% were obese, with BMIs of at least 30 kg/m².

After adjustment for age, sex, race, and antihypertensive class, the team found a significant, linear correlation between visceral fat and mean 24-hour systolic blood pressure. Patients with a visceral adiposity of about 0.1 kg/m², for instance, had a mean pressure of around 130 mm Hg, compared with patients with more than 0.6 kg/m², who had a mean of almost 150 mm Hg. Findings were similar for waist circumference over a range of 70-150 cm.

The correlations were weak (r = 0.3), but Dr. Cohen said they might improve with ongoing enrollment. Both measures also correlated with systolic variability.

Overall, the highest quartiles of waist circumference and visceral adiposity correlated with the highest mean systolic pressures and greatest variability, compared with the lowest quartiles. Visceral adiposity was the only measure significantly linked with masked hypertension. Trends in those directions for increasing BMI and total body fat mass were not statistically significant.

Mean BMI in the study was 31.7 kg/m², and mean waist circumference was 104 cm. Mean 24-hour systolic blood pressure was 135 mm Hg and mean 24-hour systolic variability was 13 mm Hg. Almost 30% of the subjects had masked hypertension. Drug classes included beta-blockers, calcium channel blockers, diuretics, ACE-inhibitors, and angiotensin receptor blockers.

Dr. Cohen plans to investigate drug response versus visceral adiposity once the recruitment goal of 150 subjects is reached.

There was no external funding, and the investigators reported that they didn’t have any relevant disclosures.

aotto@mdedge.com

SOURCE: Cohen JB et al. Joint Hypertension 2019, Abstract P2052.

NEW ORLEANS – Visceral adiposity, but not body mass index or total body fat, significantly correlated with elevated 24-hour ambulatory systolic blood pressure, greater systolic variability, and masked hypertension in a study from the University of Pennsylvania, Philadelphia.

Subjects in the highest quartile of visceral fat had a 6.3-fold greater odds of masked hypertension – normal in the office, but high at home – compared with those in the lowest quartile (95% confidence interval, 1.2-33.1).

The study findings suggest that central obesity, in particular, should trigger 24-hour ambulatory blood pressure monitoring (ABPM). “Every obese person should get a 24-hour” ABPM, but “we really need to be pushing [it] in people who have central adiposity. These are the patients ... we really need to focus on” because of the risk of masked hypertension, a “ticking time bomb” that greatly increases the risk of cardiovascular events, said lead investigator Jordana B. Cohen, MD, an assistant professor of medicine at the university.

The study also helps explain why body mass index (BMI) hasn’t been consistently linked to masked hypertension in previous studies; some studies likely included subjects with high BMIs but not central obesity.

Waist circumference, a marker of visceral adiposity, also correlated with elevated 24-hour systolic pressure and greater variability, but a trend for masked hypertension was not statistically significant, Dr. Cohen reported at the joint scientific sessions of the American Heart Association (AHA) Council on Hypertension, AHA Council on Kidney in Cardiovascular Disease, and American Society of Hypertension.

It’s long been known that visceral fat – fat around the abdominal organs – is metabolically active and associated with greater cardiovascular risk, but its relationship to blood pressure hadn’t been well described, so Dr. Cohen and her team decided to take a look.

They ran whole body dual x-ray absorptiometry scans on 96 hypertensive adults on a stable dose of one antihypertensive drug for at least 2 months and correlated the findings with ABPM. Subjects were an average of 58 years old, almost 60% were women, almost half were black, and 54% were obese, with BMIs of at least 30 kg/m².

After adjustment for age, sex, race, and antihypertensive class, the team found a significant, linear correlation between visceral fat and mean 24-hour systolic blood pressure. Patients with a visceral adiposity of about 0.1 kg/m², for instance, had a mean pressure of around 130 mm Hg, compared with patients with more than 0.6 kg/m², who had a mean of almost 150 mm Hg. Findings were similar for waist circumference over a range of 70-150 cm.

The correlations were weak (r = 0.3), but Dr. Cohen said they might improve with ongoing enrollment. Both measures also correlated with systolic variability.

Overall, the highest quartiles of waist circumference and visceral adiposity correlated with the highest mean systolic pressures and greatest variability, compared with the lowest quartiles. Visceral adiposity was the only measure significantly linked with masked hypertension. Trends in those directions for increasing BMI and total body fat mass were not statistically significant.

Mean BMI in the study was 31.7 kg/m², and mean waist circumference was 104 cm. Mean 24-hour systolic blood pressure was 135 mm Hg and mean 24-hour systolic variability was 13 mm Hg. Almost 30% of the subjects had masked hypertension. Drug classes included beta-blockers, calcium channel blockers, diuretics, ACE-inhibitors, and angiotensin receptor blockers.

Dr. Cohen plans to investigate drug response versus visceral adiposity once the recruitment goal of 150 subjects is reached.

There was no external funding, and the investigators reported that they didn’t have any relevant disclosures.

aotto@mdedge.com

SOURCE: Cohen JB et al. Joint Hypertension 2019, Abstract P2052.

NEW ORLEANS – Visceral adiposity, but not body mass index or total body fat, significantly correlated with elevated 24-hour ambulatory systolic blood pressure, greater systolic variability, and masked hypertension in a study from the University of Pennsylvania, Philadelphia.

Subjects in the highest quartile of visceral fat had a 6.3-fold greater odds of masked hypertension – normal in the office, but high at home – compared with those in the lowest quartile (95% confidence interval, 1.2-33.1).

The study findings suggest that central obesity, in particular, should trigger 24-hour ambulatory blood pressure monitoring (ABPM). “Every obese person should get a 24-hour” ABPM, but “we really need to be pushing [it] in people who have central adiposity. These are the patients ... we really need to focus on” because of the risk of masked hypertension, a “ticking time bomb” that greatly increases the risk of cardiovascular events, said lead investigator Jordana B. Cohen, MD, an assistant professor of medicine at the university.

The study also helps explain why body mass index (BMI) hasn’t been consistently linked to masked hypertension in previous studies; some studies likely included subjects with high BMIs but not central obesity.

Waist circumference, a marker of visceral adiposity, also correlated with elevated 24-hour systolic pressure and greater variability, but a trend for masked hypertension was not statistically significant, Dr. Cohen reported at the joint scientific sessions of the American Heart Association (AHA) Council on Hypertension, AHA Council on Kidney in Cardiovascular Disease, and American Society of Hypertension.

It’s long been known that visceral fat – fat around the abdominal organs – is metabolically active and associated with greater cardiovascular risk, but its relationship to blood pressure hadn’t been well described, so Dr. Cohen and her team decided to take a look.

They ran whole body dual x-ray absorptiometry scans on 96 hypertensive adults on a stable dose of one antihypertensive drug for at least 2 months and correlated the findings with ABPM. Subjects were an average of 58 years old, almost 60% were women, almost half were black, and 54% were obese, with BMIs of at least 30 kg/m².

After adjustment for age, sex, race, and antihypertensive class, the team found a significant, linear correlation between visceral fat and mean 24-hour systolic blood pressure. Patients with a visceral adiposity of about 0.1 kg/m², for instance, had a mean pressure of around 130 mm Hg, compared with patients with more than 0.6 kg/m², who had a mean of almost 150 mm Hg. Findings were similar for waist circumference over a range of 70-150 cm.

The correlations were weak (r = 0.3), but Dr. Cohen said they might improve with ongoing enrollment. Both measures also correlated with systolic variability.

Overall, the highest quartiles of waist circumference and visceral adiposity correlated with the highest mean systolic pressures and greatest variability, compared with the lowest quartiles. Visceral adiposity was the only measure significantly linked with masked hypertension. Trends in those directions for increasing BMI and total body fat mass were not statistically significant.

Mean BMI in the study was 31.7 kg/m², and mean waist circumference was 104 cm. Mean 24-hour systolic blood pressure was 135 mm Hg and mean 24-hour systolic variability was 13 mm Hg. Almost 30% of the subjects had masked hypertension. Drug classes included beta-blockers, calcium channel blockers, diuretics, ACE-inhibitors, and angiotensin receptor blockers.

Dr. Cohen plans to investigate drug response versus visceral adiposity once the recruitment goal of 150 subjects is reached.

There was no external funding, and the investigators reported that they didn’t have any relevant disclosures.

aotto@mdedge.com

SOURCE: Cohen JB et al. Joint Hypertension 2019, Abstract P2052.

AUSTIN, TEX. – The way Kelly M. Cordoro sees it, the most difficult part of managing pediatric patients with severe psoriasis is not in the logistics of prescribing a drug, it’s deciding which drug to use for which patient.

“You can look up the dosing and frequency of these drugs; all of that’s available,” she said at the annual meeting of the Society for Pediatric Dermatology. “But how do we think about which drug for which patient? What are the considerations?”

Dr. Cordoro, professor of dermatology and pediatrics at the University of California, San Francisco, described psoriasis as an autoamplifying inflammatory cascade involving innate and adaptive immunity and noted that various components of that cascade represent treatment targets. “We don’t have a true comprehension of the pathophysiology of psoriasis, but as we learn the pathways, we’re targeting them,” she said. “You can target keratinocyte proliferation with drugs like retinoids and phototherapy. You can broadly target T cells, neutrophils, and dendritic cells with methotrexate, cyclosporine, and phototherapy. The newer drugs target the cytokine milieu, including TNF [tumor necrosis factor]–alpha, IL [interleukin]–17A, IL-12, and IL-23.”

There is no one right answer for which drug to prescribe, she continued, except in the cases of certain comorbidities, contraindications, and genetic variants. “For example, if a patient has psoriatic arthritis, then you have methotrexate and all of the biologics that might be disease modifying,” she said. “If a patient has inflammatory bowel disease, it’s critical to know that IL-17 inhibitors will flare that disease, but anti-TNF and IL-12 and IL-23 inhibitors are okay. If a patient has liver and kidney disease, you want to avoid methotrexate and cyclosporine. If there’s a female of childbearing potential you want to be very cautious with using retinoids. I think the harder question for us is, How about the rest of the patients?”

In addition to a drug’s mechanism of action, patient- and family-related factors play a role in deciding which agent to use. For example, does the patient prefer an oral or an injectable agent? Is the patient able to travel to a phototherapy center? Is it feasible for the family to manage visits for lab work and direct clinical monitoring? Does the family have a high level of health literacy and are you communicating with them in ways that facilitate shared decision making?

“The best way to choose a systemic therapy is to develop an individualized assessment of overall disease burden,” said Dr. Cordoro, who is also division chief of pediatric dermatology at UCSF. “Include psychological burden and subjective data in addition to objective measures like body surface area. Look for triggers. Infants are more commonly affected by viral infections and, in a subset, monogenic forms of psoriasis such as deficiency of interleukin 1 receptor antagonist [DIRA]. In general, we try to take a conservative approach in the developing child. As children hit early adolescence and become post pubertal, you have to start thinking about the psychosocial impact [of psoriasis], and we have to start treating patients with the consideration that chronic uncontrolled inflammation can potentially lead to comorbidities down the road. We see this in adults with severe psoriasis and early onset cardiovascular disease, the so-called psoriatic march from chronic inflammation to cardiovascular disease.”

Dr. Cordoro advises clinicians to rethink the conventional “therapeutic ladder” concept and embrace the idea of “finding the right tool for the job right now.” If a patient presents with a flare from a known trigger such as a strep infection, “maybe you want to treat with something more conservative,” she said. “Once you treat, and if the trigger has been managed, they might be better. But some patients will need the most aggressive treatment right out of the gate.”

Tried and true systemic therapies for psoriasis include methotrexate, cyclosporine, acitretin, and phototherapy, but none is approved by the Food and Drug Administration for use in children. “These drugs have decades of experience behind them,” Dr. Cordoro said. “Methotrexate is slow to start but has a sustained profile, so if you can get the patient to respond, that response tends to persist. Methotrexate also prevents the formation of antidrug antibodies, which is important if you are considering use of a biologic agent later on.”

Cyclosporine is best if you need a rapid rescue drug to get the disease under control before moving on to other options. “One in four patients relapse once cyclosporine is discontinued, so the benefit may not be as sustained as with methotrexate,” she said. “Acitretin is a really nice choice when you can’t or don’t want to immunosuppress the patient, and phototherapy is good if you can get it. The advantages of systemic therapies are that they’re easy on, easy off, and you can combine medications in severe situations. Almost all of these drugs can be combined with another, with few exceptions. I would caution that over immunosuppression is the biggest risk ... so this must be done carefully and only when necessary.”

Biologic agents such as TNF inhibitors and IL-12/23 inhibitors are playing an increasing role in pediatric psoriasis. They can be expensive and difficult for some insurance plans to cover, but offer the convenience of better efficacy and less frequent lab monitoring than conventional systemics. In the United States, etanercept and ustekinumab are approved for moderate to severe pediatric plaque psoriasis in patients as young as age 4 and 12 years, respectively. TNF inhibitors have accumulated the most data in children, while data are accumulating in trials of IL-17 inhibitors, IL-23 inhibitors, and PDE4 inhibitors.

“These drugs have reassuring safety profiles; low rates of infection and adverse reactions,” Dr. Cordoro said of biologic agents. “They’ve changed the landscape completely because now the expectation is complete or near-complete clearance. In contrast to the systemic agents, which may be started and stopped repeatedly, you need to think about continuous therapy, because these drugs are immunogenic,” she noted. “Whether antibodies against them become neutralizing or not is a different case. If a patient does have antibodies, it does not mean you have to stop the drug. Dose escalation can help. Increasing frequency of use of the drug can help, but patients will develop antibodies and it may result in loss of efficacy or reactions to the drug,” she added.

“When you’re thinking about using a biologic agent, think about patients who are chronic, moderate to severe, and who will need more long-term therapy. Most importantly, treatment should be individualized, as there is no ‘one size fits all’ approach.”

Dr. Cordoro disclosed that she is a member of the Celgene Corporation Scientific Steering Committee.

dbrunk@mdedge.com

AUSTIN, TEX. – The way Kelly M. Cordoro sees it, the most difficult part of managing pediatric patients with severe psoriasis is not in the logistics of prescribing a drug, it’s deciding which drug to use for which patient.

“You can look up the dosing and frequency of these drugs; all of that’s available,” she said at the annual meeting of the Society for Pediatric Dermatology. “But how do we think about which drug for which patient? What are the considerations?”

Dr. Cordoro, professor of dermatology and pediatrics at the University of California, San Francisco, described psoriasis as an autoamplifying inflammatory cascade involving innate and adaptive immunity and noted that various components of that cascade represent treatment targets. “We don’t have a true comprehension of the pathophysiology of psoriasis, but as we learn the pathways, we’re targeting them,” she said. “You can target keratinocyte proliferation with drugs like retinoids and phototherapy. You can broadly target T cells, neutrophils, and dendritic cells with methotrexate, cyclosporine, and phototherapy. The newer drugs target the cytokine milieu, including TNF [tumor necrosis factor]–alpha, IL [interleukin]–17A, IL-12, and IL-23.”

There is no one right answer for which drug to prescribe, she continued, except in the cases of certain comorbidities, contraindications, and genetic variants. “For example, if a patient has psoriatic arthritis, then you have methotrexate and all of the biologics that might be disease modifying,” she said. “If a patient has inflammatory bowel disease, it’s critical to know that IL-17 inhibitors will flare that disease, but anti-TNF and IL-12 and IL-23 inhibitors are okay. If a patient has liver and kidney disease, you want to avoid methotrexate and cyclosporine. If there’s a female of childbearing potential you want to be very cautious with using retinoids. I think the harder question for us is, How about the rest of the patients?”

In addition to a drug’s mechanism of action, patient- and family-related factors play a role in deciding which agent to use. For example, does the patient prefer an oral or an injectable agent? Is the patient able to travel to a phototherapy center? Is it feasible for the family to manage visits for lab work and direct clinical monitoring? Does the family have a high level of health literacy and are you communicating with them in ways that facilitate shared decision making?

“The best way to choose a systemic therapy is to develop an individualized assessment of overall disease burden,” said Dr. Cordoro, who is also division chief of pediatric dermatology at UCSF. “Include psychological burden and subjective data in addition to objective measures like body surface area. Look for triggers. Infants are more commonly affected by viral infections and, in a subset, monogenic forms of psoriasis such as deficiency of interleukin 1 receptor antagonist [DIRA]. In general, we try to take a conservative approach in the developing child. As children hit early adolescence and become post pubertal, you have to start thinking about the psychosocial impact [of psoriasis], and we have to start treating patients with the consideration that chronic uncontrolled inflammation can potentially lead to comorbidities down the road. We see this in adults with severe psoriasis and early onset cardiovascular disease, the so-called psoriatic march from chronic inflammation to cardiovascular disease.”

Dr. Cordoro advises clinicians to rethink the conventional “therapeutic ladder” concept and embrace the idea of “finding the right tool for the job right now.” If a patient presents with a flare from a known trigger such as a strep infection, “maybe you want to treat with something more conservative,” she said. “Once you treat, and if the trigger has been managed, they might be better. But some patients will need the most aggressive treatment right out of the gate.”

Tried and true systemic therapies for psoriasis include methotrexate, cyclosporine, acitretin, and phototherapy, but none is approved by the Food and Drug Administration for use in children. “These drugs have decades of experience behind them,” Dr. Cordoro said. “Methotrexate is slow to start but has a sustained profile, so if you can get the patient to respond, that response tends to persist. Methotrexate also prevents the formation of antidrug antibodies, which is important if you are considering use of a biologic agent later on.”

Cyclosporine is best if you need a rapid rescue drug to get the disease under control before moving on to other options. “One in four patients relapse once cyclosporine is discontinued, so the benefit may not be as sustained as with methotrexate,” she said. “Acitretin is a really nice choice when you can’t or don’t want to immunosuppress the patient, and phototherapy is good if you can get it. The advantages of systemic therapies are that they’re easy on, easy off, and you can combine medications in severe situations. Almost all of these drugs can be combined with another, with few exceptions. I would caution that over immunosuppression is the biggest risk ... so this must be done carefully and only when necessary.”

Biologic agents such as TNF inhibitors and IL-12/23 inhibitors are playing an increasing role in pediatric psoriasis. They can be expensive and difficult for some insurance plans to cover, but offer the convenience of better efficacy and less frequent lab monitoring than conventional systemics. In the United States, etanercept and ustekinumab are approved for moderate to severe pediatric plaque psoriasis in patients as young as age 4 and 12 years, respectively. TNF inhibitors have accumulated the most data in children, while data are accumulating in trials of IL-17 inhibitors, IL-23 inhibitors, and PDE4 inhibitors.

“These drugs have reassuring safety profiles; low rates of infection and adverse reactions,” Dr. Cordoro said of biologic agents. “They’ve changed the landscape completely because now the expectation is complete or near-complete clearance. In contrast to the systemic agents, which may be started and stopped repeatedly, you need to think about continuous therapy, because these drugs are immunogenic,” she noted. “Whether antibodies against them become neutralizing or not is a different case. If a patient does have antibodies, it does not mean you have to stop the drug. Dose escalation can help. Increasing frequency of use of the drug can help, but patients will develop antibodies and it may result in loss of efficacy or reactions to the drug,” she added.

“When you’re thinking about using a biologic agent, think about patients who are chronic, moderate to severe, and who will need more long-term therapy. Most importantly, treatment should be individualized, as there is no ‘one size fits all’ approach.”

Dr. Cordoro disclosed that she is a member of the Celgene Corporation Scientific Steering Committee.

dbrunk@mdedge.com

AUSTIN, TEX. – The way Kelly M. Cordoro sees it, the most difficult part of managing pediatric patients with severe psoriasis is not in the logistics of prescribing a drug, it’s deciding which drug to use for which patient.

“You can look up the dosing and frequency of these drugs; all of that’s available,” she said at the annual meeting of the Society for Pediatric Dermatology. “But how do we think about which drug for which patient? What are the considerations?”

Dr. Cordoro, professor of dermatology and pediatrics at the University of California, San Francisco, described psoriasis as an autoamplifying inflammatory cascade involving innate and adaptive immunity and noted that various components of that cascade represent treatment targets. “We don’t have a true comprehension of the pathophysiology of psoriasis, but as we learn the pathways, we’re targeting them,” she said. “You can target keratinocyte proliferation with drugs like retinoids and phototherapy. You can broadly target T cells, neutrophils, and dendritic cells with methotrexate, cyclosporine, and phototherapy. The newer drugs target the cytokine milieu, including TNF [tumor necrosis factor]–alpha, IL [interleukin]–17A, IL-12, and IL-23.”

There is no one right answer for which drug to prescribe, she continued, except in the cases of certain comorbidities, contraindications, and genetic variants. “For example, if a patient has psoriatic arthritis, then you have methotrexate and all of the biologics that might be disease modifying,” she said. “If a patient has inflammatory bowel disease, it’s critical to know that IL-17 inhibitors will flare that disease, but anti-TNF and IL-12 and IL-23 inhibitors are okay. If a patient has liver and kidney disease, you want to avoid methotrexate and cyclosporine. If there’s a female of childbearing potential you want to be very cautious with using retinoids. I think the harder question for us is, How about the rest of the patients?”

In addition to a drug’s mechanism of action, patient- and family-related factors play a role in deciding which agent to use. For example, does the patient prefer an oral or an injectable agent? Is the patient able to travel to a phototherapy center? Is it feasible for the family to manage visits for lab work and direct clinical monitoring? Does the family have a high level of health literacy and are you communicating with them in ways that facilitate shared decision making?

“The best way to choose a systemic therapy is to develop an individualized assessment of overall disease burden,” said Dr. Cordoro, who is also division chief of pediatric dermatology at UCSF. “Include psychological burden and subjective data in addition to objective measures like body surface area. Look for triggers. Infants are more commonly affected by viral infections and, in a subset, monogenic forms of psoriasis such as deficiency of interleukin 1 receptor antagonist [DIRA]. In general, we try to take a conservative approach in the developing child. As children hit early adolescence and become post pubertal, you have to start thinking about the psychosocial impact [of psoriasis], and we have to start treating patients with the consideration that chronic uncontrolled inflammation can potentially lead to comorbidities down the road. We see this in adults with severe psoriasis and early onset cardiovascular disease, the so-called psoriatic march from chronic inflammation to cardiovascular disease.”

Dr. Cordoro advises clinicians to rethink the conventional “therapeutic ladder” concept and embrace the idea of “finding the right tool for the job right now.” If a patient presents with a flare from a known trigger such as a strep infection, “maybe you want to treat with something more conservative,” she said. “Once you treat, and if the trigger has been managed, they might be better. But some patients will need the most aggressive treatment right out of the gate.”

Tried and true systemic therapies for psoriasis include methotrexate, cyclosporine, acitretin, and phototherapy, but none is approved by the Food and Drug Administration for use in children. “These drugs have decades of experience behind them,” Dr. Cordoro said. “Methotrexate is slow to start but has a sustained profile, so if you can get the patient to respond, that response tends to persist. Methotrexate also prevents the formation of antidrug antibodies, which is important if you are considering use of a biologic agent later on.”

Cyclosporine is best if you need a rapid rescue drug to get the disease under control before moving on to other options. “One in four patients relapse once cyclosporine is discontinued, so the benefit may not be as sustained as with methotrexate,” she said. “Acitretin is a really nice choice when you can’t or don’t want to immunosuppress the patient, and phototherapy is good if you can get it. The advantages of systemic therapies are that they’re easy on, easy off, and you can combine medications in severe situations. Almost all of these drugs can be combined with another, with few exceptions. I would caution that over immunosuppression is the biggest risk ... so this must be done carefully and only when necessary.”

Biologic agents such as TNF inhibitors and IL-12/23 inhibitors are playing an increasing role in pediatric psoriasis. They can be expensive and difficult for some insurance plans to cover, but offer the convenience of better efficacy and less frequent lab monitoring than conventional systemics. In the United States, etanercept and ustekinumab are approved for moderate to severe pediatric plaque psoriasis in patients as young as age 4 and 12 years, respectively. TNF inhibitors have accumulated the most data in children, while data are accumulating in trials of IL-17 inhibitors, IL-23 inhibitors, and PDE4 inhibitors.

“These drugs have reassuring safety profiles; low rates of infection and adverse reactions,” Dr. Cordoro said of biologic agents. “They’ve changed the landscape completely because now the expectation is complete or near-complete clearance. In contrast to the systemic agents, which may be started and stopped repeatedly, you need to think about continuous therapy, because these drugs are immunogenic,” she noted. “Whether antibodies against them become neutralizing or not is a different case. If a patient does have antibodies, it does not mean you have to stop the drug. Dose escalation can help. Increasing frequency of use of the drug can help, but patients will develop antibodies and it may result in loss of efficacy or reactions to the drug,” she added.

“When you’re thinking about using a biologic agent, think about patients who are chronic, moderate to severe, and who will need more long-term therapy. Most importantly, treatment should be individualized, as there is no ‘one size fits all’ approach.”

Dr. Cordoro disclosed that she is a member of the Celgene Corporation Scientific Steering Committee.

dbrunk@mdedge.com

In older patients with newly diagnosed mantle cell lymphoma (MCL), first-line therapy with rituximab- and bendamustine-based regimens significantly reduced 1-year mortality rates versus chemotherapy alone, according to a retrospective analysis.

Wikimedia Commons/TexasPathologistMSW/CC-ASA 4.0 International

“This study evaluated the comparative effectiveness of [rituximab, bortezomib, or bendamustine] in elderly patients newly diagnosed with MCL,” wrote Shuangshuang Fu, PhD, of the University of Texas MD Anderson Cancer Center, Houston, and colleagues. The findings were reported in Clinical Lymphoma, Myeloma & Leukemia.

The researchers studied population-based data from the Surveillance, Epidemiology, and End Results (SEER)-Medicare linked database. They identified all patients over age 65 years who received a new diagnosis of MCL between Jan. 1, 1999, and Dec. 31, 2013.

The study cohort included a total of 1,215 patients. Participants were classified into four different groups according to treatment regimen: chemotherapy alone, rituximab plus or minus chemotherapy, bendamustine plus or minus chemotherapy, and bortezomib plus or minus chemotherapy.

At 1-year follow-up, the team analyzed various mortality outcomes, including MCL-specific, all-cause, and noncancer mortality. The bortezomib results were not included in the primary analysis because of small sample size, according to the researchers.

After multivariable analysis, Dr. Fu and colleagues found that 1-year all-cause mortality rate was significantly lower for patients receiving rituximab-based regimens, compared with chemotherapy alone (hazard ratio, 0.38; 95% confidence interval, 0.25-0.59). There was a similar decline for MCL-specific mortality (HR, 0.38; 95% CI, 0.24-0.60).

The 1-year MCL-specific mortality was also significantly reduced in the bendamustine group, compared with chemotherapy alone (HR, 0.49; 95% CI, 0.24-0.99).

“Our findings comparing rituximab with chemotherapy alone further confirmed the benefit of adding rituximab to chemotherapy in newly diagnosed older MCL patients,” they wrote.

The researchers acknowledged that a key limitation of the study was the observational design. As a result, selection bias and unmeasured confounding could have influenced the results.

“Future studies evaluating the comparative effectiveness of those newly approved novel agents for MCL patients were warranted as more data are available,” they concluded.

The study was funded by the Duncan Family Institute, the Cancer Prevention Research Institute of Texas, and the National Institutes of Health. The authors reported having no conflicts of interest.

SOURCE: Fu S et al. Clin Lymphoma Myeloma Leuk. 2019 Aug 30. doi: 10.1016/j.clml.2019.08.014.

In older patients with newly diagnosed mantle cell lymphoma (MCL), first-line therapy with rituximab- and bendamustine-based regimens significantly reduced 1-year mortality rates versus chemotherapy alone, according to a retrospective analysis.

Wikimedia Commons/TexasPathologistMSW/CC-ASA 4.0 International

“This study evaluated the comparative effectiveness of [rituximab, bortezomib, or bendamustine] in elderly patients newly diagnosed with MCL,” wrote Shuangshuang Fu, PhD, of the University of Texas MD Anderson Cancer Center, Houston, and colleagues. The findings were reported in Clinical Lymphoma, Myeloma & Leukemia.

The researchers studied population-based data from the Surveillance, Epidemiology, and End Results (SEER)-Medicare linked database. They identified all patients over age 65 years who received a new diagnosis of MCL between Jan. 1, 1999, and Dec. 31, 2013.

The study cohort included a total of 1,215 patients. Participants were classified into four different groups according to treatment regimen: chemotherapy alone, rituximab plus or minus chemotherapy, bendamustine plus or minus chemotherapy, and bortezomib plus or minus chemotherapy.

At 1-year follow-up, the team analyzed various mortality outcomes, including MCL-specific, all-cause, and noncancer mortality. The bortezomib results were not included in the primary analysis because of small sample size, according to the researchers.

After multivariable analysis, Dr. Fu and colleagues found that 1-year all-cause mortality rate was significantly lower for patients receiving rituximab-based regimens, compared with chemotherapy alone (hazard ratio, 0.38; 95% confidence interval, 0.25-0.59). There was a similar decline for MCL-specific mortality (HR, 0.38; 95% CI, 0.24-0.60).

The 1-year MCL-specific mortality was also significantly reduced in the bendamustine group, compared with chemotherapy alone (HR, 0.49; 95% CI, 0.24-0.99).

“Our findings comparing rituximab with chemotherapy alone further confirmed the benefit of adding rituximab to chemotherapy in newly diagnosed older MCL patients,” they wrote.

The researchers acknowledged that a key limitation of the study was the observational design. As a result, selection bias and unmeasured confounding could have influenced the results.

“Future studies evaluating the comparative effectiveness of those newly approved novel agents for MCL patients were warranted as more data are available,” they concluded.

The study was funded by the Duncan Family Institute, the Cancer Prevention Research Institute of Texas, and the National Institutes of Health. The authors reported having no conflicts of interest.

SOURCE: Fu S et al. Clin Lymphoma Myeloma Leuk. 2019 Aug 30. doi: 10.1016/j.clml.2019.08.014.

In older patients with newly diagnosed mantle cell lymphoma (MCL), first-line therapy with rituximab- and bendamustine-based regimens significantly reduced 1-year mortality rates versus chemotherapy alone, according to a retrospective analysis.

Wikimedia Commons/TexasPathologistMSW/CC-ASA 4.0 International

“This study evaluated the comparative effectiveness of [rituximab, bortezomib, or bendamustine] in elderly patients newly diagnosed with MCL,” wrote Shuangshuang Fu, PhD, of the University of Texas MD Anderson Cancer Center, Houston, and colleagues. The findings were reported in Clinical Lymphoma, Myeloma & Leukemia.

The researchers studied population-based data from the Surveillance, Epidemiology, and End Results (SEER)-Medicare linked database. They identified all patients over age 65 years who received a new diagnosis of MCL between Jan. 1, 1999, and Dec. 31, 2013.

The study cohort included a total of 1,215 patients. Participants were classified into four different groups according to treatment regimen: chemotherapy alone, rituximab plus or minus chemotherapy, bendamustine plus or minus chemotherapy, and bortezomib plus or minus chemotherapy.

At 1-year follow-up, the team analyzed various mortality outcomes, including MCL-specific, all-cause, and noncancer mortality. The bortezomib results were not included in the primary analysis because of small sample size, according to the researchers.

After multivariable analysis, Dr. Fu and colleagues found that 1-year all-cause mortality rate was significantly lower for patients receiving rituximab-based regimens, compared with chemotherapy alone (hazard ratio, 0.38; 95% confidence interval, 0.25-0.59). There was a similar decline for MCL-specific mortality (HR, 0.38; 95% CI, 0.24-0.60).

The 1-year MCL-specific mortality was also significantly reduced in the bendamustine group, compared with chemotherapy alone (HR, 0.49; 95% CI, 0.24-0.99).

“Our findings comparing rituximab with chemotherapy alone further confirmed the benefit of adding rituximab to chemotherapy in newly diagnosed older MCL patients,” they wrote.

The researchers acknowledged that a key limitation of the study was the observational design. As a result, selection bias and unmeasured confounding could have influenced the results.

“Future studies evaluating the comparative effectiveness of those newly approved novel agents for MCL patients were warranted as more data are available,” they concluded.

The study was funded by the Duncan Family Institute, the Cancer Prevention Research Institute of Texas, and the National Institutes of Health. The authors reported having no conflicts of interest.

SOURCE: Fu S et al. Clin Lymphoma Myeloma Leuk. 2019 Aug 30. doi: 10.1016/j.clml.2019.08.014.