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Blood-brain barrier imaging could predict disease progression in bipolar
Blood-brain barrier imaging can serve as a biomarker for progression of disease in adults with bipolar disorder, results from a small study suggest.
“While the pathophysiology of bipolar disorder remains poorly understood, converging evidence points to the presence of neuroinflammation in bipolar patients,” wrote Lyna Kamintsky, a PhD candidate at Dalhousie University, Halifax, N.S., and colleagues.
The researchers examined MRI data from 36 patients with bipolar disorder and compared them with 14 matched controls. The average age of the patients was 49 years and the average duration of illness was 28 years. The study was published in NeuroImage: Clinical (2019 Oct 22. doi: 10.1016/j.nicl.2019.102049).
“Leakage rates were considered pathological when exceeding 0.02, the 95th percentile of all values in a cohort of control subjects,” the researchers said. Overall, 10 subjects (all patients with bipolar disorder) met criteria for “extensive blood-brain barrier leakage.” The researchers found that those patients also had higher rates of chronic illness, more frequent and/or severe manic episodes, and more severe anxiety, depression, and social/occupational dysfunction, compared with those without blood-brain barrier leakage.
The patients with extensive blood-brain barrier leakage also had higher body mass indexes, greater risk of cardiovascular disease, and advanced heart age. In addition, all patients in this group had comorbid insulin resistance.
The study findings were limited by the small sample size, but , the researchers said.
The study was supported by the European Union’s Seventh Framework Program, the Nova Scotia Health Research Foundation, Brain Canada, and the Brain & Behavior Research Foundation. The researchers disclosed having no financial conflicts.
SOURCE: Kamintsky L et al. NeuroImage: Clinical. 2019 Oct 22. doi: 10.1016/j.nicl.2019.102049.
Blood-brain barrier imaging can serve as a biomarker for progression of disease in adults with bipolar disorder, results from a small study suggest.
“While the pathophysiology of bipolar disorder remains poorly understood, converging evidence points to the presence of neuroinflammation in bipolar patients,” wrote Lyna Kamintsky, a PhD candidate at Dalhousie University, Halifax, N.S., and colleagues.
The researchers examined MRI data from 36 patients with bipolar disorder and compared them with 14 matched controls. The average age of the patients was 49 years and the average duration of illness was 28 years. The study was published in NeuroImage: Clinical (2019 Oct 22. doi: 10.1016/j.nicl.2019.102049).
“Leakage rates were considered pathological when exceeding 0.02, the 95th percentile of all values in a cohort of control subjects,” the researchers said. Overall, 10 subjects (all patients with bipolar disorder) met criteria for “extensive blood-brain barrier leakage.” The researchers found that those patients also had higher rates of chronic illness, more frequent and/or severe manic episodes, and more severe anxiety, depression, and social/occupational dysfunction, compared with those without blood-brain barrier leakage.
The patients with extensive blood-brain barrier leakage also had higher body mass indexes, greater risk of cardiovascular disease, and advanced heart age. In addition, all patients in this group had comorbid insulin resistance.
The study findings were limited by the small sample size, but , the researchers said.
The study was supported by the European Union’s Seventh Framework Program, the Nova Scotia Health Research Foundation, Brain Canada, and the Brain & Behavior Research Foundation. The researchers disclosed having no financial conflicts.
SOURCE: Kamintsky L et al. NeuroImage: Clinical. 2019 Oct 22. doi: 10.1016/j.nicl.2019.102049.
Blood-brain barrier imaging can serve as a biomarker for progression of disease in adults with bipolar disorder, results from a small study suggest.
“While the pathophysiology of bipolar disorder remains poorly understood, converging evidence points to the presence of neuroinflammation in bipolar patients,” wrote Lyna Kamintsky, a PhD candidate at Dalhousie University, Halifax, N.S., and colleagues.
The researchers examined MRI data from 36 patients with bipolar disorder and compared them with 14 matched controls. The average age of the patients was 49 years and the average duration of illness was 28 years. The study was published in NeuroImage: Clinical (2019 Oct 22. doi: 10.1016/j.nicl.2019.102049).
“Leakage rates were considered pathological when exceeding 0.02, the 95th percentile of all values in a cohort of control subjects,” the researchers said. Overall, 10 subjects (all patients with bipolar disorder) met criteria for “extensive blood-brain barrier leakage.” The researchers found that those patients also had higher rates of chronic illness, more frequent and/or severe manic episodes, and more severe anxiety, depression, and social/occupational dysfunction, compared with those without blood-brain barrier leakage.
The patients with extensive blood-brain barrier leakage also had higher body mass indexes, greater risk of cardiovascular disease, and advanced heart age. In addition, all patients in this group had comorbid insulin resistance.
The study findings were limited by the small sample size, but , the researchers said.
The study was supported by the European Union’s Seventh Framework Program, the Nova Scotia Health Research Foundation, Brain Canada, and the Brain & Behavior Research Foundation. The researchers disclosed having no financial conflicts.
SOURCE: Kamintsky L et al. NeuroImage: Clinical. 2019 Oct 22. doi: 10.1016/j.nicl.2019.102049.
FROM NEUROIMAGE: CLINICAL
AAD-NPF Pediatric psoriasis guideline advises on physical and mental care
Psoriasis management in children involves attention not only to treatment of the physical condition but also psychosocial wellness and quality of life, according to
Psoriasis affects approximately 1% of children, either alone or associated with comorbid conditions such as psoriatic arthritis (PsA), wrote Alan Menter, MD, of Baylor University Medical Center, Dallas, and coauthors of the guideline.
In the guideline, published in the Journal of the American Academy of Dermatology, the multidisciplinary work group identified screening tools to measure disease severity, strategies for management of comorbidities, and the safety and effectiveness of topical, systemic, and phototherapy treatments.
To assess disease severity, the work group recommended not only the use of body surface area (BSA), similar to measurement of severity in adults, but also the use of the Children’s Dermatology Life Quality Index, a 10-question quality of life survey, as BSA alone does not account for the potential negative impact of the disease on quality of life in terms of physical, emotional, social, and psychological function.
“For example, a child with psoriasis limited to the face or the entire scalp does not have severe disease based on BSA definitions, but if this involvement causes shame, social withdrawal, or bullying, it satisfies criteria for severe disease based on impact beyond the skin,” they said.
The work group stated that a variety of conditions may trigger or exacerbate psoriasis in children, including infections, cutaneous trauma, or physiological, emotional, and environmental stressors.
The majority of children with PsA develop joint inflammation before skin disease, the work group wrote. In addition, children with psoriasis are at increased risk for rheumatoid arthritis, so clinicians may need to distinguish between a combination of psoriasis and musculoskeletal issues and cases of either psoriatic or rheumatoid arthritis in young patients.
The cardiovascular risk factors associated with metabolic syndrome are greater in children with psoriasis, compared with children without psoriasis, the work group noted. In addition, pediatric psoriasis patients have a higher prevalence of obesity than children without psoriasis, and they recommended that children with psoriasis be monitored for the development of obesity, and that obese children with psoriasis should be referred for weight management.
The work group noted that data are insufficient in children to support the link between psoriasis and cardiovascular disease that has been documented in adults with psoriasis. However, “patients with pediatric psoriasis should have American Academy of Pediatrics (AAP)–recommended age-related cardiovascular screening regardless of the presence of signs or symptoms,” they said.
The guideline also recommends screening for dyslipidemia and hypertension according to AAP guidelines and educating pediatric psoriasis patients about the risk of diabetes and regularly screening for diabetes and insulin resistance in those who are obese. Overweight children with psoriasis may be screened at the provider’s discretion, they wrote. Patients with signs of inflammatory bowel disease, which also is associated with psoriasis in adults, should be considered for referral to a gastroenterologist, they noted.
Children with psoriasis should be screened regularly for mental health conditions regardless of age, and they should be asked about substance abuse, according to the guideline, and those with concerns should be referred for additional assessment and management.
The guideline divides treatment of psoriasis in children into three categories: topical, phototherapy and photochemotherapy, and systemic treatments (nonbiologic or biologic).
For topicals, the guideline recommendations include corticosteroids as an off-label therapy, as well as ultra-high-potency topical corticosteroids as monotherapy. Overall, “selection of a therapeutic routine (potency, delivery vehicle, frequency of application) should take into account sites of involvement, type and thickness of psoriasis, age of the patient, total BSA of application, anticipated occlusion, and disease acuity, among other patient-, disease-, and drug-related factors,” the authors wrote. Other topical options included in the recommendations: calcineurin inhibitors, topical vitamin D analogues, tazarotene (off label), anthralin, and coal tar.
Phototherapy has a history of use in psoriasis treatment and remains part of the current recommendations, although data in children are limited, and data on the use of phototherapy for pustular psoriasis in children are insufficient to make specific treatment and dosing recommendations, the work group noted. The researchers also noted that in-office phototherapy may not be feasible for many patients, but that in-home ultraviolet light equipment or natural sunlight in moderation could be recommended as an alternative.
The use of systemic, nonbiologic treatments for pediatric psoriasis should be “based on baseline severity of disease, subtype of psoriasis, speed of disease progression, lack of response to more conservative therapies such as topical agents and phototherapy (when appropriate), impaired physical or psychological functioning or [quality of life] due to disease extent, and the presence of comorbidities such as PsA,” the workgroup said.
Options for systemic treatment include methotrexate, cyclosporine (notably for pustular as well as plaque and erythrodermic psoriasis), and systemic retinoids. In addition, fumaric acid esters may be an option for children with moderate to severe psoriasis, with recommended clinical and laboratory monitoring.
The increasing safety and efficacy data on biologics in pediatric psoriasis patients support their consideration among first-line systemic treatments, the work group suggested. “Etanercept and ustekinumab are now [Food and Drug Administration] approved for patients with psoriasis 4 years and older and 12 years and older, respectively,” they said, and infliximab and adalimumab have been used off label in children.
The work group concluded that research and knowledge gaps about pediatric psoriasis persist and include mechanism of disease onset, development of comorbidities, and identification of ideal dosing for various treatments.
Finally, the work group emphasized the importance of collaboration between dermatologists and primary care providers for managing psoriasis in children, as well as the importance of patient education.
“Dermatologists should be mindful of the unique aspects of the emotional development of children and the social dynamics of having a visible difference,” they wrote. “Shared decision making with the patient (if age appropriate) and the caregivers is a useful approach, particularly as related to the use of off-label medications to treat severe disease,” they said.
“This is the first time that pediatric psoriasis has been discussed as an independent topic within the guideline,” said one of the guideline authors, Dawn M.R. Davis, MD, of the Mayo Clinic, Rochester, Minn., in an interview. “Children have unique physiology and psychosocial aspects to their care relative to adults. In addition, psoriasis has some clinical manifestations that are oftentimes distinctly seen in children,” she commented. “Creation of a guideline specific to children allows us to summarize the similarities and differences of disease presentation and management. It also allows an opportunity to clarify what research data (especially therapeutics) have been studied in children and their uses, safety profiles, and dosing,” she noted.
Psoriasis can be a psychosocially debilitating disease, she emphasized. “In children, for example, isolated or prominent facial involvement is common, which can be embarrassing and impact relationships.”
The take-home message for clinicians, Dr. Davis said, is to keep in mind the multisystemic nature of psoriasis. “It is not limited to the skin,” she said. “Treating a patient with psoriasis necessitates practicing whole-person care” and considering the multiple comorbidities that impact quality of life and overall health in children, as well as adults with psoriasis, she commented. “Dermatologists can empower patients and their caregivers by educating them on the multifocal, complex nature of the disease.” She added, “We have much to learn regarding psoriasis in the pediatric population. More research into therapeutics, topical and systemic, is necessary to optimize patient care.”
The guideline was based on studies published in the PubMed and MEDLINE databases from January 2011 through December 31, 2017.
Dr. Menter and Craig A. Elmets, MD, professor of dermatology, at the University of Alabama, Birmingham, were cochairs of the work group. The pediatric guideline is the latest in a multipart series of AAD-NPF guidelines on psoriasis being published this year in the Journal of the American Academy of Dermatology.
Many of the guideline authors, including lead author Dr. Menter, disclosed relationships with multiple companies; however, a minimum 51% of workgroup members had no relevant conflicts of interest in accordance with AAD policy. There was no funding source. Dr. Davis disclosed serving as an investigator for Regeneron, with no compensation.
SOURCE: Menter et al. J Am Acad Dermatol. 2019. doi: 10.1016/j.jaad.2019.08.049.
Psoriasis management in children involves attention not only to treatment of the physical condition but also psychosocial wellness and quality of life, according to
Psoriasis affects approximately 1% of children, either alone or associated with comorbid conditions such as psoriatic arthritis (PsA), wrote Alan Menter, MD, of Baylor University Medical Center, Dallas, and coauthors of the guideline.
In the guideline, published in the Journal of the American Academy of Dermatology, the multidisciplinary work group identified screening tools to measure disease severity, strategies for management of comorbidities, and the safety and effectiveness of topical, systemic, and phototherapy treatments.
To assess disease severity, the work group recommended not only the use of body surface area (BSA), similar to measurement of severity in adults, but also the use of the Children’s Dermatology Life Quality Index, a 10-question quality of life survey, as BSA alone does not account for the potential negative impact of the disease on quality of life in terms of physical, emotional, social, and psychological function.
“For example, a child with psoriasis limited to the face or the entire scalp does not have severe disease based on BSA definitions, but if this involvement causes shame, social withdrawal, or bullying, it satisfies criteria for severe disease based on impact beyond the skin,” they said.
The work group stated that a variety of conditions may trigger or exacerbate psoriasis in children, including infections, cutaneous trauma, or physiological, emotional, and environmental stressors.
The majority of children with PsA develop joint inflammation before skin disease, the work group wrote. In addition, children with psoriasis are at increased risk for rheumatoid arthritis, so clinicians may need to distinguish between a combination of psoriasis and musculoskeletal issues and cases of either psoriatic or rheumatoid arthritis in young patients.
The cardiovascular risk factors associated with metabolic syndrome are greater in children with psoriasis, compared with children without psoriasis, the work group noted. In addition, pediatric psoriasis patients have a higher prevalence of obesity than children without psoriasis, and they recommended that children with psoriasis be monitored for the development of obesity, and that obese children with psoriasis should be referred for weight management.
The work group noted that data are insufficient in children to support the link between psoriasis and cardiovascular disease that has been documented in adults with psoriasis. However, “patients with pediatric psoriasis should have American Academy of Pediatrics (AAP)–recommended age-related cardiovascular screening regardless of the presence of signs or symptoms,” they said.
The guideline also recommends screening for dyslipidemia and hypertension according to AAP guidelines and educating pediatric psoriasis patients about the risk of diabetes and regularly screening for diabetes and insulin resistance in those who are obese. Overweight children with psoriasis may be screened at the provider’s discretion, they wrote. Patients with signs of inflammatory bowel disease, which also is associated with psoriasis in adults, should be considered for referral to a gastroenterologist, they noted.
Children with psoriasis should be screened regularly for mental health conditions regardless of age, and they should be asked about substance abuse, according to the guideline, and those with concerns should be referred for additional assessment and management.
The guideline divides treatment of psoriasis in children into three categories: topical, phototherapy and photochemotherapy, and systemic treatments (nonbiologic or biologic).
For topicals, the guideline recommendations include corticosteroids as an off-label therapy, as well as ultra-high-potency topical corticosteroids as monotherapy. Overall, “selection of a therapeutic routine (potency, delivery vehicle, frequency of application) should take into account sites of involvement, type and thickness of psoriasis, age of the patient, total BSA of application, anticipated occlusion, and disease acuity, among other patient-, disease-, and drug-related factors,” the authors wrote. Other topical options included in the recommendations: calcineurin inhibitors, topical vitamin D analogues, tazarotene (off label), anthralin, and coal tar.
Phototherapy has a history of use in psoriasis treatment and remains part of the current recommendations, although data in children are limited, and data on the use of phototherapy for pustular psoriasis in children are insufficient to make specific treatment and dosing recommendations, the work group noted. The researchers also noted that in-office phototherapy may not be feasible for many patients, but that in-home ultraviolet light equipment or natural sunlight in moderation could be recommended as an alternative.
The use of systemic, nonbiologic treatments for pediatric psoriasis should be “based on baseline severity of disease, subtype of psoriasis, speed of disease progression, lack of response to more conservative therapies such as topical agents and phototherapy (when appropriate), impaired physical or psychological functioning or [quality of life] due to disease extent, and the presence of comorbidities such as PsA,” the workgroup said.
Options for systemic treatment include methotrexate, cyclosporine (notably for pustular as well as plaque and erythrodermic psoriasis), and systemic retinoids. In addition, fumaric acid esters may be an option for children with moderate to severe psoriasis, with recommended clinical and laboratory monitoring.
The increasing safety and efficacy data on biologics in pediatric psoriasis patients support their consideration among first-line systemic treatments, the work group suggested. “Etanercept and ustekinumab are now [Food and Drug Administration] approved for patients with psoriasis 4 years and older and 12 years and older, respectively,” they said, and infliximab and adalimumab have been used off label in children.
The work group concluded that research and knowledge gaps about pediatric psoriasis persist and include mechanism of disease onset, development of comorbidities, and identification of ideal dosing for various treatments.
Finally, the work group emphasized the importance of collaboration between dermatologists and primary care providers for managing psoriasis in children, as well as the importance of patient education.
“Dermatologists should be mindful of the unique aspects of the emotional development of children and the social dynamics of having a visible difference,” they wrote. “Shared decision making with the patient (if age appropriate) and the caregivers is a useful approach, particularly as related to the use of off-label medications to treat severe disease,” they said.
“This is the first time that pediatric psoriasis has been discussed as an independent topic within the guideline,” said one of the guideline authors, Dawn M.R. Davis, MD, of the Mayo Clinic, Rochester, Minn., in an interview. “Children have unique physiology and psychosocial aspects to their care relative to adults. In addition, psoriasis has some clinical manifestations that are oftentimes distinctly seen in children,” she commented. “Creation of a guideline specific to children allows us to summarize the similarities and differences of disease presentation and management. It also allows an opportunity to clarify what research data (especially therapeutics) have been studied in children and their uses, safety profiles, and dosing,” she noted.
Psoriasis can be a psychosocially debilitating disease, she emphasized. “In children, for example, isolated or prominent facial involvement is common, which can be embarrassing and impact relationships.”
The take-home message for clinicians, Dr. Davis said, is to keep in mind the multisystemic nature of psoriasis. “It is not limited to the skin,” she said. “Treating a patient with psoriasis necessitates practicing whole-person care” and considering the multiple comorbidities that impact quality of life and overall health in children, as well as adults with psoriasis, she commented. “Dermatologists can empower patients and their caregivers by educating them on the multifocal, complex nature of the disease.” She added, “We have much to learn regarding psoriasis in the pediatric population. More research into therapeutics, topical and systemic, is necessary to optimize patient care.”
The guideline was based on studies published in the PubMed and MEDLINE databases from January 2011 through December 31, 2017.
Dr. Menter and Craig A. Elmets, MD, professor of dermatology, at the University of Alabama, Birmingham, were cochairs of the work group. The pediatric guideline is the latest in a multipart series of AAD-NPF guidelines on psoriasis being published this year in the Journal of the American Academy of Dermatology.
Many of the guideline authors, including lead author Dr. Menter, disclosed relationships with multiple companies; however, a minimum 51% of workgroup members had no relevant conflicts of interest in accordance with AAD policy. There was no funding source. Dr. Davis disclosed serving as an investigator for Regeneron, with no compensation.
SOURCE: Menter et al. J Am Acad Dermatol. 2019. doi: 10.1016/j.jaad.2019.08.049.
Psoriasis management in children involves attention not only to treatment of the physical condition but also psychosocial wellness and quality of life, according to
Psoriasis affects approximately 1% of children, either alone or associated with comorbid conditions such as psoriatic arthritis (PsA), wrote Alan Menter, MD, of Baylor University Medical Center, Dallas, and coauthors of the guideline.
In the guideline, published in the Journal of the American Academy of Dermatology, the multidisciplinary work group identified screening tools to measure disease severity, strategies for management of comorbidities, and the safety and effectiveness of topical, systemic, and phototherapy treatments.
To assess disease severity, the work group recommended not only the use of body surface area (BSA), similar to measurement of severity in adults, but also the use of the Children’s Dermatology Life Quality Index, a 10-question quality of life survey, as BSA alone does not account for the potential negative impact of the disease on quality of life in terms of physical, emotional, social, and psychological function.
“For example, a child with psoriasis limited to the face or the entire scalp does not have severe disease based on BSA definitions, but if this involvement causes shame, social withdrawal, or bullying, it satisfies criteria for severe disease based on impact beyond the skin,” they said.
The work group stated that a variety of conditions may trigger or exacerbate psoriasis in children, including infections, cutaneous trauma, or physiological, emotional, and environmental stressors.
The majority of children with PsA develop joint inflammation before skin disease, the work group wrote. In addition, children with psoriasis are at increased risk for rheumatoid arthritis, so clinicians may need to distinguish between a combination of psoriasis and musculoskeletal issues and cases of either psoriatic or rheumatoid arthritis in young patients.
The cardiovascular risk factors associated with metabolic syndrome are greater in children with psoriasis, compared with children without psoriasis, the work group noted. In addition, pediatric psoriasis patients have a higher prevalence of obesity than children without psoriasis, and they recommended that children with psoriasis be monitored for the development of obesity, and that obese children with psoriasis should be referred for weight management.
The work group noted that data are insufficient in children to support the link between psoriasis and cardiovascular disease that has been documented in adults with psoriasis. However, “patients with pediatric psoriasis should have American Academy of Pediatrics (AAP)–recommended age-related cardiovascular screening regardless of the presence of signs or symptoms,” they said.
The guideline also recommends screening for dyslipidemia and hypertension according to AAP guidelines and educating pediatric psoriasis patients about the risk of diabetes and regularly screening for diabetes and insulin resistance in those who are obese. Overweight children with psoriasis may be screened at the provider’s discretion, they wrote. Patients with signs of inflammatory bowel disease, which also is associated with psoriasis in adults, should be considered for referral to a gastroenterologist, they noted.
Children with psoriasis should be screened regularly for mental health conditions regardless of age, and they should be asked about substance abuse, according to the guideline, and those with concerns should be referred for additional assessment and management.
The guideline divides treatment of psoriasis in children into three categories: topical, phototherapy and photochemotherapy, and systemic treatments (nonbiologic or biologic).
For topicals, the guideline recommendations include corticosteroids as an off-label therapy, as well as ultra-high-potency topical corticosteroids as monotherapy. Overall, “selection of a therapeutic routine (potency, delivery vehicle, frequency of application) should take into account sites of involvement, type and thickness of psoriasis, age of the patient, total BSA of application, anticipated occlusion, and disease acuity, among other patient-, disease-, and drug-related factors,” the authors wrote. Other topical options included in the recommendations: calcineurin inhibitors, topical vitamin D analogues, tazarotene (off label), anthralin, and coal tar.
Phototherapy has a history of use in psoriasis treatment and remains part of the current recommendations, although data in children are limited, and data on the use of phototherapy for pustular psoriasis in children are insufficient to make specific treatment and dosing recommendations, the work group noted. The researchers also noted that in-office phototherapy may not be feasible for many patients, but that in-home ultraviolet light equipment or natural sunlight in moderation could be recommended as an alternative.
The use of systemic, nonbiologic treatments for pediatric psoriasis should be “based on baseline severity of disease, subtype of psoriasis, speed of disease progression, lack of response to more conservative therapies such as topical agents and phototherapy (when appropriate), impaired physical or psychological functioning or [quality of life] due to disease extent, and the presence of comorbidities such as PsA,” the workgroup said.
Options for systemic treatment include methotrexate, cyclosporine (notably for pustular as well as plaque and erythrodermic psoriasis), and systemic retinoids. In addition, fumaric acid esters may be an option for children with moderate to severe psoriasis, with recommended clinical and laboratory monitoring.
The increasing safety and efficacy data on biologics in pediatric psoriasis patients support their consideration among first-line systemic treatments, the work group suggested. “Etanercept and ustekinumab are now [Food and Drug Administration] approved for patients with psoriasis 4 years and older and 12 years and older, respectively,” they said, and infliximab and adalimumab have been used off label in children.
The work group concluded that research and knowledge gaps about pediatric psoriasis persist and include mechanism of disease onset, development of comorbidities, and identification of ideal dosing for various treatments.
Finally, the work group emphasized the importance of collaboration between dermatologists and primary care providers for managing psoriasis in children, as well as the importance of patient education.
“Dermatologists should be mindful of the unique aspects of the emotional development of children and the social dynamics of having a visible difference,” they wrote. “Shared decision making with the patient (if age appropriate) and the caregivers is a useful approach, particularly as related to the use of off-label medications to treat severe disease,” they said.
“This is the first time that pediatric psoriasis has been discussed as an independent topic within the guideline,” said one of the guideline authors, Dawn M.R. Davis, MD, of the Mayo Clinic, Rochester, Minn., in an interview. “Children have unique physiology and psychosocial aspects to their care relative to adults. In addition, psoriasis has some clinical manifestations that are oftentimes distinctly seen in children,” she commented. “Creation of a guideline specific to children allows us to summarize the similarities and differences of disease presentation and management. It also allows an opportunity to clarify what research data (especially therapeutics) have been studied in children and their uses, safety profiles, and dosing,” she noted.
Psoriasis can be a psychosocially debilitating disease, she emphasized. “In children, for example, isolated or prominent facial involvement is common, which can be embarrassing and impact relationships.”
The take-home message for clinicians, Dr. Davis said, is to keep in mind the multisystemic nature of psoriasis. “It is not limited to the skin,” she said. “Treating a patient with psoriasis necessitates practicing whole-person care” and considering the multiple comorbidities that impact quality of life and overall health in children, as well as adults with psoriasis, she commented. “Dermatologists can empower patients and their caregivers by educating them on the multifocal, complex nature of the disease.” She added, “We have much to learn regarding psoriasis in the pediatric population. More research into therapeutics, topical and systemic, is necessary to optimize patient care.”
The guideline was based on studies published in the PubMed and MEDLINE databases from January 2011 through December 31, 2017.
Dr. Menter and Craig A. Elmets, MD, professor of dermatology, at the University of Alabama, Birmingham, were cochairs of the work group. The pediatric guideline is the latest in a multipart series of AAD-NPF guidelines on psoriasis being published this year in the Journal of the American Academy of Dermatology.
Many of the guideline authors, including lead author Dr. Menter, disclosed relationships with multiple companies; however, a minimum 51% of workgroup members had no relevant conflicts of interest in accordance with AAD policy. There was no funding source. Dr. Davis disclosed serving as an investigator for Regeneron, with no compensation.
SOURCE: Menter et al. J Am Acad Dermatol. 2019. doi: 10.1016/j.jaad.2019.08.049.
FROM THE JOURNAL OF THE AMERICAN ACADEMY OF DERMATOLOGY
Fenofibrate fights increased triglycerides in NASH
BOSTON – Fenofibrate is safe and effective for limiting triglyceride increases in patients with nonalcoholic steatohepatitis and advanced fibrosis, based on data from 31 adults.
Treatment of NASH with acetyl-CoA carboxylase inhibitors has been shown to improve liver fat and other liver conditions but may be associated with hyperlipidemia, according to Eric J. Lawitz, MD, of the University of Texas Health, San Antonio, and colleagues. The researchers examined the safety and effectiveness of fenofibrate to mitigate serum triglyceride increases in a study be presented in a late-breaking session at the annual meeting of the American Association for the Study of Liver Diseases.
The researchers randomized 15 patients to treatment with 48 mg of fenofibrate or 145 mg of fenofibrate once daily for 2 weeks, followed by a combination of the fenofibrate doses plus 20 mg of ACC inhibitor firsocostat daily for 24 weeks.
The median fasting triglycerides (TG) in the 48-mg and 145-mg fenofibrate groups were 218 mg/dL and 202 mg/dL, respectively. After 2 weeks, the median change in TG was +2 mg/dL in the 48-mg group and –42 mg/dL in the 145-mg group. After 24 weeks of combination therapy, TG levels were not significantly different from baseline in either group (+19 mg/dL in 48-mg group and +6 mg/dL in the 145-mg group). Significant reductions in serum alanine aminotransferase from baseline to week 24 occurred in the combined groups (median of 39 U/L vs. 27 U/L, respectively). In addition, 43% of patients overall showed at least a 30% reduction in protein density fat fraction.
Both firsocostat and fenofibrate were well tolerated, the researchers said. One treatment-emergent grade 3 TG elevation (defined as greater than 500 mg/dL) occurred in the 48-mg group at week 24. No hepatotoxicity was noted, no patients discontinued therapy because of adverse events, and no other grade 3 or 4 adverse events were reported.
“The combination of firsocostat and fenofibrate led to improvements in hepatic fat, liver biochemistry, and markers of fibrosis,” the researchers concluded in their abstract.
Lead author Dr. Lawitz disclosed financial relationships with Allergan, Akcea Therapeutics, Bristol-Myers Squibb, Boehringer Ingelheim, Gilead Sciences, Madrigal Pharmaceuticals, and Novartis.
The AGA GI Patient Center provides education to help your patients understand NASH at https://www.gastro.org/practice-guidance/gi-patient-center/topic/nonalcoholic-steatohepatitis-nash.
SOURCE: Lawitz E et al. The Liver Meeting 2019. Presentation LP5.
BOSTON – Fenofibrate is safe and effective for limiting triglyceride increases in patients with nonalcoholic steatohepatitis and advanced fibrosis, based on data from 31 adults.
Treatment of NASH with acetyl-CoA carboxylase inhibitors has been shown to improve liver fat and other liver conditions but may be associated with hyperlipidemia, according to Eric J. Lawitz, MD, of the University of Texas Health, San Antonio, and colleagues. The researchers examined the safety and effectiveness of fenofibrate to mitigate serum triglyceride increases in a study be presented in a late-breaking session at the annual meeting of the American Association for the Study of Liver Diseases.
The researchers randomized 15 patients to treatment with 48 mg of fenofibrate or 145 mg of fenofibrate once daily for 2 weeks, followed by a combination of the fenofibrate doses plus 20 mg of ACC inhibitor firsocostat daily for 24 weeks.
The median fasting triglycerides (TG) in the 48-mg and 145-mg fenofibrate groups were 218 mg/dL and 202 mg/dL, respectively. After 2 weeks, the median change in TG was +2 mg/dL in the 48-mg group and –42 mg/dL in the 145-mg group. After 24 weeks of combination therapy, TG levels were not significantly different from baseline in either group (+19 mg/dL in 48-mg group and +6 mg/dL in the 145-mg group). Significant reductions in serum alanine aminotransferase from baseline to week 24 occurred in the combined groups (median of 39 U/L vs. 27 U/L, respectively). In addition, 43% of patients overall showed at least a 30% reduction in protein density fat fraction.
Both firsocostat and fenofibrate were well tolerated, the researchers said. One treatment-emergent grade 3 TG elevation (defined as greater than 500 mg/dL) occurred in the 48-mg group at week 24. No hepatotoxicity was noted, no patients discontinued therapy because of adverse events, and no other grade 3 or 4 adverse events were reported.
“The combination of firsocostat and fenofibrate led to improvements in hepatic fat, liver biochemistry, and markers of fibrosis,” the researchers concluded in their abstract.
Lead author Dr. Lawitz disclosed financial relationships with Allergan, Akcea Therapeutics, Bristol-Myers Squibb, Boehringer Ingelheim, Gilead Sciences, Madrigal Pharmaceuticals, and Novartis.
The AGA GI Patient Center provides education to help your patients understand NASH at https://www.gastro.org/practice-guidance/gi-patient-center/topic/nonalcoholic-steatohepatitis-nash.
SOURCE: Lawitz E et al. The Liver Meeting 2019. Presentation LP5.
BOSTON – Fenofibrate is safe and effective for limiting triglyceride increases in patients with nonalcoholic steatohepatitis and advanced fibrosis, based on data from 31 adults.
Treatment of NASH with acetyl-CoA carboxylase inhibitors has been shown to improve liver fat and other liver conditions but may be associated with hyperlipidemia, according to Eric J. Lawitz, MD, of the University of Texas Health, San Antonio, and colleagues. The researchers examined the safety and effectiveness of fenofibrate to mitigate serum triglyceride increases in a study be presented in a late-breaking session at the annual meeting of the American Association for the Study of Liver Diseases.
The researchers randomized 15 patients to treatment with 48 mg of fenofibrate or 145 mg of fenofibrate once daily for 2 weeks, followed by a combination of the fenofibrate doses plus 20 mg of ACC inhibitor firsocostat daily for 24 weeks.
The median fasting triglycerides (TG) in the 48-mg and 145-mg fenofibrate groups were 218 mg/dL and 202 mg/dL, respectively. After 2 weeks, the median change in TG was +2 mg/dL in the 48-mg group and –42 mg/dL in the 145-mg group. After 24 weeks of combination therapy, TG levels were not significantly different from baseline in either group (+19 mg/dL in 48-mg group and +6 mg/dL in the 145-mg group). Significant reductions in serum alanine aminotransferase from baseline to week 24 occurred in the combined groups (median of 39 U/L vs. 27 U/L, respectively). In addition, 43% of patients overall showed at least a 30% reduction in protein density fat fraction.
Both firsocostat and fenofibrate were well tolerated, the researchers said. One treatment-emergent grade 3 TG elevation (defined as greater than 500 mg/dL) occurred in the 48-mg group at week 24. No hepatotoxicity was noted, no patients discontinued therapy because of adverse events, and no other grade 3 or 4 adverse events were reported.
“The combination of firsocostat and fenofibrate led to improvements in hepatic fat, liver biochemistry, and markers of fibrosis,” the researchers concluded in their abstract.
Lead author Dr. Lawitz disclosed financial relationships with Allergan, Akcea Therapeutics, Bristol-Myers Squibb, Boehringer Ingelheim, Gilead Sciences, Madrigal Pharmaceuticals, and Novartis.
The AGA GI Patient Center provides education to help your patients understand NASH at https://www.gastro.org/practice-guidance/gi-patient-center/topic/nonalcoholic-steatohepatitis-nash.
SOURCE: Lawitz E et al. The Liver Meeting 2019. Presentation LP5.
REPORTING FROM THE LIVER MEETING 2019
Key clinical point: Fenofibrate mitigated serum triglyceride increases in patients with NASH.
Major finding: After 24 weeks of fenofibrate treatment, 43% of patients showed a relative reduction of at least 30% in protein density fat fraction with an average of 40% at a 48 mg-dose and 47% at a 145-mg dose.
Study details: The data come from a 24-week study of 31 adults with advanced fibrosis caused by NASH.
Disclosures: Lead author Dr. Lawitz disclosed financial relationships with Allergan, Akcea Therapeutics, Bristol-Myers Squibb, Boehringer Ingelheim, Gilead Sciences, Madrigal Pharmaceuticals, and Novartis.
Source: Lawitz E et al. The Liver Meeting 2019. Presentation LP5.
New psoriatic patients may have lower serious infection risk with IL-12/23 inhibition
, according to data from a retrospective study of nearly 10,000 adults.
Biologics, though effective, can increase the risk for serious infection in psoriatic arthritis and psoriasis patients, and comparison data on the safety of various biologics are limited, wrote Xintong Li of Johns Hopkins University, Baltimore, and colleagues.
In a study published in Annals of the Rheumatic Diseases, the researchers identified 11,560 treatment episodes for 9,305 adults during January 1, 2015, through May 1, 2018, which included 6,043 episodes of psoriasis only, 1,869 episodes of psoriatic arthritis only, and 3,648 episodes of both conditions. The average age of the patients was 46 years, and 53% were men. The investigators studied the IL-12/23 inhibitor ustekinumab (Stelara), the IL-17A inhibitors secukinumab (Cosentyx) and ixekizumab (Taltz), and TNF inhibitors adalimumab (Humira), certolizumab pegol (Cimzia), etanercept (Enbrel), golimumab (Simponi), and infliximab (Remicade). They did not study the interleukin-17 (IL-17) inhibitor brodalumab (Siliq) or IL-23 inhibitors guselkumab (Tremfya), risankizumab (Skyrizi), or tildrakizumab (Ilumya) since they were FDA approved after or toward the end of the study period.
The primary outcome of serious infection, defined as hospitalization with infection as part of the diagnostic codes, occurred in 190 cases (2% of all treatment episodes); the most common serious infections were sepsis and pneumonia.
Overall, new biologics users had similar infection risks with IL-17 and TNF inhibitors, with incidence rates per 100 person-years of 3.4 and 2.2, respectively. By contrast, the incidence rate per 100 person-years was 0.9 with IL-12/23 inhibitors. Incidence rates were similar across all three biologic types for experienced biologics users.
The researchers also grouped patients by condition, including both treatment-naive and -experienced patients. Of the 156 serious infections in psoriasis patients, 26 occurred with IL-17 inhibitors, 29 with IL-12/23 inhibitors, and 101 with TNF inhibitors. Of 105 serious infections in the psoriatic arthritis group, 14 occurred with IL-17, 13 with IL-12/23, and 78 with TNF.
After adjusting for propensity scores, researchers found no evidence of increased serious infection risk for treatment with IL-17 inhibitors, compared with IL-12/23 (hazard ratio, 1.12; 95% confidence interval, 0.62-2.03) or TNF inhibitors (HR, 0.89; 95% CI, 0.48-1.66).
The study findings were limited by several factors, including the use of ICD diagnostic codes that were not fully validated in the patient population, a short follow-up period, and inclusion of only insured patients in the United States, the researchers noted.
However, the results suggest that serious infection risk may vary between patients with psoriasis and psoriatic arthritis, and between biologic-naive and -experienced patients, and that, despite the relatively small difference in absolute effect, “this potentially clinically relevant signal for reduced infections among the IL-12/23 inhibitors warrants further investigation and surveillance efforts,” they concluded. In addition, they said, the findings might guide clinicians and patients in choosing appropriate biologics for a particular condition.
The study was supported by the Center for Drug Safety and Effectiveness at the Johns Hopkins Bloomberg School of Public Health. The researchers had no financial conflicts to disclose. Ms. Li conducted the research while she was at Johns Hopkins, but is now with the University of North Carolina, Chapel Hill.
SOURCE: Li X et al. Ann Rheum Dis. 2019 Oct 31. doi: 10.1136/annrheumdis-2019-216102.
, according to data from a retrospective study of nearly 10,000 adults.
Biologics, though effective, can increase the risk for serious infection in psoriatic arthritis and psoriasis patients, and comparison data on the safety of various biologics are limited, wrote Xintong Li of Johns Hopkins University, Baltimore, and colleagues.
In a study published in Annals of the Rheumatic Diseases, the researchers identified 11,560 treatment episodes for 9,305 adults during January 1, 2015, through May 1, 2018, which included 6,043 episodes of psoriasis only, 1,869 episodes of psoriatic arthritis only, and 3,648 episodes of both conditions. The average age of the patients was 46 years, and 53% were men. The investigators studied the IL-12/23 inhibitor ustekinumab (Stelara), the IL-17A inhibitors secukinumab (Cosentyx) and ixekizumab (Taltz), and TNF inhibitors adalimumab (Humira), certolizumab pegol (Cimzia), etanercept (Enbrel), golimumab (Simponi), and infliximab (Remicade). They did not study the interleukin-17 (IL-17) inhibitor brodalumab (Siliq) or IL-23 inhibitors guselkumab (Tremfya), risankizumab (Skyrizi), or tildrakizumab (Ilumya) since they were FDA approved after or toward the end of the study period.
The primary outcome of serious infection, defined as hospitalization with infection as part of the diagnostic codes, occurred in 190 cases (2% of all treatment episodes); the most common serious infections were sepsis and pneumonia.
Overall, new biologics users had similar infection risks with IL-17 and TNF inhibitors, with incidence rates per 100 person-years of 3.4 and 2.2, respectively. By contrast, the incidence rate per 100 person-years was 0.9 with IL-12/23 inhibitors. Incidence rates were similar across all three biologic types for experienced biologics users.
The researchers also grouped patients by condition, including both treatment-naive and -experienced patients. Of the 156 serious infections in psoriasis patients, 26 occurred with IL-17 inhibitors, 29 with IL-12/23 inhibitors, and 101 with TNF inhibitors. Of 105 serious infections in the psoriatic arthritis group, 14 occurred with IL-17, 13 with IL-12/23, and 78 with TNF.
After adjusting for propensity scores, researchers found no evidence of increased serious infection risk for treatment with IL-17 inhibitors, compared with IL-12/23 (hazard ratio, 1.12; 95% confidence interval, 0.62-2.03) or TNF inhibitors (HR, 0.89; 95% CI, 0.48-1.66).
The study findings were limited by several factors, including the use of ICD diagnostic codes that were not fully validated in the patient population, a short follow-up period, and inclusion of only insured patients in the United States, the researchers noted.
However, the results suggest that serious infection risk may vary between patients with psoriasis and psoriatic arthritis, and between biologic-naive and -experienced patients, and that, despite the relatively small difference in absolute effect, “this potentially clinically relevant signal for reduced infections among the IL-12/23 inhibitors warrants further investigation and surveillance efforts,” they concluded. In addition, they said, the findings might guide clinicians and patients in choosing appropriate biologics for a particular condition.
The study was supported by the Center for Drug Safety and Effectiveness at the Johns Hopkins Bloomberg School of Public Health. The researchers had no financial conflicts to disclose. Ms. Li conducted the research while she was at Johns Hopkins, but is now with the University of North Carolina, Chapel Hill.
SOURCE: Li X et al. Ann Rheum Dis. 2019 Oct 31. doi: 10.1136/annrheumdis-2019-216102.
, according to data from a retrospective study of nearly 10,000 adults.
Biologics, though effective, can increase the risk for serious infection in psoriatic arthritis and psoriasis patients, and comparison data on the safety of various biologics are limited, wrote Xintong Li of Johns Hopkins University, Baltimore, and colleagues.
In a study published in Annals of the Rheumatic Diseases, the researchers identified 11,560 treatment episodes for 9,305 adults during January 1, 2015, through May 1, 2018, which included 6,043 episodes of psoriasis only, 1,869 episodes of psoriatic arthritis only, and 3,648 episodes of both conditions. The average age of the patients was 46 years, and 53% were men. The investigators studied the IL-12/23 inhibitor ustekinumab (Stelara), the IL-17A inhibitors secukinumab (Cosentyx) and ixekizumab (Taltz), and TNF inhibitors adalimumab (Humira), certolizumab pegol (Cimzia), etanercept (Enbrel), golimumab (Simponi), and infliximab (Remicade). They did not study the interleukin-17 (IL-17) inhibitor brodalumab (Siliq) or IL-23 inhibitors guselkumab (Tremfya), risankizumab (Skyrizi), or tildrakizumab (Ilumya) since they were FDA approved after or toward the end of the study period.
The primary outcome of serious infection, defined as hospitalization with infection as part of the diagnostic codes, occurred in 190 cases (2% of all treatment episodes); the most common serious infections were sepsis and pneumonia.
Overall, new biologics users had similar infection risks with IL-17 and TNF inhibitors, with incidence rates per 100 person-years of 3.4 and 2.2, respectively. By contrast, the incidence rate per 100 person-years was 0.9 with IL-12/23 inhibitors. Incidence rates were similar across all three biologic types for experienced biologics users.
The researchers also grouped patients by condition, including both treatment-naive and -experienced patients. Of the 156 serious infections in psoriasis patients, 26 occurred with IL-17 inhibitors, 29 with IL-12/23 inhibitors, and 101 with TNF inhibitors. Of 105 serious infections in the psoriatic arthritis group, 14 occurred with IL-17, 13 with IL-12/23, and 78 with TNF.
After adjusting for propensity scores, researchers found no evidence of increased serious infection risk for treatment with IL-17 inhibitors, compared with IL-12/23 (hazard ratio, 1.12; 95% confidence interval, 0.62-2.03) or TNF inhibitors (HR, 0.89; 95% CI, 0.48-1.66).
The study findings were limited by several factors, including the use of ICD diagnostic codes that were not fully validated in the patient population, a short follow-up period, and inclusion of only insured patients in the United States, the researchers noted.
However, the results suggest that serious infection risk may vary between patients with psoriasis and psoriatic arthritis, and between biologic-naive and -experienced patients, and that, despite the relatively small difference in absolute effect, “this potentially clinically relevant signal for reduced infections among the IL-12/23 inhibitors warrants further investigation and surveillance efforts,” they concluded. In addition, they said, the findings might guide clinicians and patients in choosing appropriate biologics for a particular condition.
The study was supported by the Center for Drug Safety and Effectiveness at the Johns Hopkins Bloomberg School of Public Health. The researchers had no financial conflicts to disclose. Ms. Li conducted the research while she was at Johns Hopkins, but is now with the University of North Carolina, Chapel Hill.
SOURCE: Li X et al. Ann Rheum Dis. 2019 Oct 31. doi: 10.1136/annrheumdis-2019-216102.
FROM ANNALS OF THE RHEUMATIC DISEASES
Families face challenges of gene therapy
WASHINGTON– Gene therapy for the treatment of rare diseases continues to develop and new products are entering the pipeline; however, more work is needed to make the gene therapy experience easier on patients and their families, according to members of a panel at the NORD Rare Diseases & Orphan Product Breakthrough Summit, held by the National Organization for Rare Disorders.
Companies developing gene therapy cite their main challenges as identifying patients, developing clinical trials, coordinating treatment and supporting families, managing reimbursement, and manufacturing the treatment, said Mark Rothera, president and CEO of Orchard Therapeutics, developer of ex vivo autologous hematopoietic stem cell gene therapy.
For families of patients with rare diseases who are undergoing gene therapy, challenges include struggles such as language barriers, lack of wifi, and separation from other family members for extended periods, according to Amy Price, mother of a gene therapy recipient, as well as principal consultant to Rarallel and an advocate for metachromatic leukodystrophy.
Ms. Price cited a survey she conducted of families with children who underwent gene therapy. She collected data from 16 families about their initial visit as part of a gene therapy trial; the trials included 14 families in Milan; 1 in Bethesda, Md.; and 1 in Paris. The average age of the patients at the start of the trial was 3 years, with a range of 8 months to 11 years. The trials were conducted between 1990 and 2018.
Families participating in the trials spent an average of 5.5 months in the city where the trial was conducted, and an average of 48 days in an isolation ward with their child at the start of the study.
The five biggest challenges were financial well-being (cited by 60% of survey respondents), social isolation/being away from support system (60%), fear of the unknown/long-term treatment diagnosis (73%), family separation (67%), and caring for other children simultaneous during the trial period (60%).
In addition, patients averaged 12 follow-up visits, and the most common secondary challenges cited in the survey included time spent at the hospital, emotional and physical stress on the patient, fear of test results and outcomes, exhaustion, time away from work and school, and travel logistics.
Other stressors include language barriers and not being in children’s hospital, Ms. Price said.
Ms. Price proposed patient-focused solutions such as addressing cultural challenges, connecting families to local resources, and providing clinical follow-up locally to reduce the burden of travel to the trial site.
WASHINGTON– Gene therapy for the treatment of rare diseases continues to develop and new products are entering the pipeline; however, more work is needed to make the gene therapy experience easier on patients and their families, according to members of a panel at the NORD Rare Diseases & Orphan Product Breakthrough Summit, held by the National Organization for Rare Disorders.
Companies developing gene therapy cite their main challenges as identifying patients, developing clinical trials, coordinating treatment and supporting families, managing reimbursement, and manufacturing the treatment, said Mark Rothera, president and CEO of Orchard Therapeutics, developer of ex vivo autologous hematopoietic stem cell gene therapy.
For families of patients with rare diseases who are undergoing gene therapy, challenges include struggles such as language barriers, lack of wifi, and separation from other family members for extended periods, according to Amy Price, mother of a gene therapy recipient, as well as principal consultant to Rarallel and an advocate for metachromatic leukodystrophy.
Ms. Price cited a survey she conducted of families with children who underwent gene therapy. She collected data from 16 families about their initial visit as part of a gene therapy trial; the trials included 14 families in Milan; 1 in Bethesda, Md.; and 1 in Paris. The average age of the patients at the start of the trial was 3 years, with a range of 8 months to 11 years. The trials were conducted between 1990 and 2018.
Families participating in the trials spent an average of 5.5 months in the city where the trial was conducted, and an average of 48 days in an isolation ward with their child at the start of the study.
The five biggest challenges were financial well-being (cited by 60% of survey respondents), social isolation/being away from support system (60%), fear of the unknown/long-term treatment diagnosis (73%), family separation (67%), and caring for other children simultaneous during the trial period (60%).
In addition, patients averaged 12 follow-up visits, and the most common secondary challenges cited in the survey included time spent at the hospital, emotional and physical stress on the patient, fear of test results and outcomes, exhaustion, time away from work and school, and travel logistics.
Other stressors include language barriers and not being in children’s hospital, Ms. Price said.
Ms. Price proposed patient-focused solutions such as addressing cultural challenges, connecting families to local resources, and providing clinical follow-up locally to reduce the burden of travel to the trial site.
WASHINGTON– Gene therapy for the treatment of rare diseases continues to develop and new products are entering the pipeline; however, more work is needed to make the gene therapy experience easier on patients and their families, according to members of a panel at the NORD Rare Diseases & Orphan Product Breakthrough Summit, held by the National Organization for Rare Disorders.
Companies developing gene therapy cite their main challenges as identifying patients, developing clinical trials, coordinating treatment and supporting families, managing reimbursement, and manufacturing the treatment, said Mark Rothera, president and CEO of Orchard Therapeutics, developer of ex vivo autologous hematopoietic stem cell gene therapy.
For families of patients with rare diseases who are undergoing gene therapy, challenges include struggles such as language barriers, lack of wifi, and separation from other family members for extended periods, according to Amy Price, mother of a gene therapy recipient, as well as principal consultant to Rarallel and an advocate for metachromatic leukodystrophy.
Ms. Price cited a survey she conducted of families with children who underwent gene therapy. She collected data from 16 families about their initial visit as part of a gene therapy trial; the trials included 14 families in Milan; 1 in Bethesda, Md.; and 1 in Paris. The average age of the patients at the start of the trial was 3 years, with a range of 8 months to 11 years. The trials were conducted between 1990 and 2018.
Families participating in the trials spent an average of 5.5 months in the city where the trial was conducted, and an average of 48 days in an isolation ward with their child at the start of the study.
The five biggest challenges were financial well-being (cited by 60% of survey respondents), social isolation/being away from support system (60%), fear of the unknown/long-term treatment diagnosis (73%), family separation (67%), and caring for other children simultaneous during the trial period (60%).
In addition, patients averaged 12 follow-up visits, and the most common secondary challenges cited in the survey included time spent at the hospital, emotional and physical stress on the patient, fear of test results and outcomes, exhaustion, time away from work and school, and travel logistics.
Other stressors include language barriers and not being in children’s hospital, Ms. Price said.
Ms. Price proposed patient-focused solutions such as addressing cultural challenges, connecting families to local resources, and providing clinical follow-up locally to reduce the burden of travel to the trial site.
EXPERT ANALYSIS FROM NORD 2019
Experts address barriers to genetic screening
WASHINGTON – Early diagnosis and intervention for genetic diseases using the latest carrier screening can allow families to be prepared and informed prior to pregnancy, said Aishwarya Arjunan, MS, MPH, a clinical product specialist for carrier screening at Myriad Women’s Health, part of a diagnostic testing company based in Salt Lake City, Utah.
“Rare diseases are responsible for 35% of deaths in the first year of life,” she said in a panel discussion at the Rare Diseases and Orphan Products Breakthrough Summit sponsored by the National Organization for Rare Disorders.
Most patients with rare diseases go through a “diagnostic odyssey” lasting an average of 8 years before they receive an accurate diagnosis, she said. During this time, data suggest that they have likely been misdiagnosed three times and have seen more than 10 specialists, she added.
Barriers to genetic screening include limited access to genetics professionals, lack of patient and provider education about screening, issues of insurance coverage and reimbursement, coding challenges, and misperceptions about the perceived impact of screening, noted Jodie Vento, manager of the Center for Rare Disease Therapy at the Children’s Hospital of Pittsburgh.
The genetic carrier screening options, often referred to as panethnic expanded carrier screening, represents a change from previous screening protocols based on ethnicity, said Ms. Arjunan. However, guidelines for screening based on ethnicity “misses a significant percentage of pregnancies affected by serious conditions and widens the health disparity gap,” she said.
By contrast, expanded carrier screening allows for standardization of care that gives couples and families information to make decisions and preparations.
Current genetic testing strategies include single gene testing, in which a single gene of interest is tested; multigene panel testing, in which a subset of clinically important genes are tested; whole-exome sequencing, in which the DNA responsible for coding proteins is tested; and whole-genome sequencing, in which the entire human genome is tested for genetic disorders.
Improving access to genetic testing involves a combination of provider education, changes in payer policies, action by advocacy groups, and adjustment of societal guidelines, said Ms. Arjunan. However, the advantages of expanded carrier screening are many and include guiding patients to expert care early and setting up plans for long-term care and follow-up, she noted. In addition, early identification through screening can help patients reduce or eliminate the diagnostic odyssey and connect with advocacy and community groups for support, she concluded.
The presenters had no financial conflicts to disclose.
WASHINGTON – Early diagnosis and intervention for genetic diseases using the latest carrier screening can allow families to be prepared and informed prior to pregnancy, said Aishwarya Arjunan, MS, MPH, a clinical product specialist for carrier screening at Myriad Women’s Health, part of a diagnostic testing company based in Salt Lake City, Utah.
“Rare diseases are responsible for 35% of deaths in the first year of life,” she said in a panel discussion at the Rare Diseases and Orphan Products Breakthrough Summit sponsored by the National Organization for Rare Disorders.
Most patients with rare diseases go through a “diagnostic odyssey” lasting an average of 8 years before they receive an accurate diagnosis, she said. During this time, data suggest that they have likely been misdiagnosed three times and have seen more than 10 specialists, she added.
Barriers to genetic screening include limited access to genetics professionals, lack of patient and provider education about screening, issues of insurance coverage and reimbursement, coding challenges, and misperceptions about the perceived impact of screening, noted Jodie Vento, manager of the Center for Rare Disease Therapy at the Children’s Hospital of Pittsburgh.
The genetic carrier screening options, often referred to as panethnic expanded carrier screening, represents a change from previous screening protocols based on ethnicity, said Ms. Arjunan. However, guidelines for screening based on ethnicity “misses a significant percentage of pregnancies affected by serious conditions and widens the health disparity gap,” she said.
By contrast, expanded carrier screening allows for standardization of care that gives couples and families information to make decisions and preparations.
Current genetic testing strategies include single gene testing, in which a single gene of interest is tested; multigene panel testing, in which a subset of clinically important genes are tested; whole-exome sequencing, in which the DNA responsible for coding proteins is tested; and whole-genome sequencing, in which the entire human genome is tested for genetic disorders.
Improving access to genetic testing involves a combination of provider education, changes in payer policies, action by advocacy groups, and adjustment of societal guidelines, said Ms. Arjunan. However, the advantages of expanded carrier screening are many and include guiding patients to expert care early and setting up plans for long-term care and follow-up, she noted. In addition, early identification through screening can help patients reduce or eliminate the diagnostic odyssey and connect with advocacy and community groups for support, she concluded.
The presenters had no financial conflicts to disclose.
WASHINGTON – Early diagnosis and intervention for genetic diseases using the latest carrier screening can allow families to be prepared and informed prior to pregnancy, said Aishwarya Arjunan, MS, MPH, a clinical product specialist for carrier screening at Myriad Women’s Health, part of a diagnostic testing company based in Salt Lake City, Utah.
“Rare diseases are responsible for 35% of deaths in the first year of life,” she said in a panel discussion at the Rare Diseases and Orphan Products Breakthrough Summit sponsored by the National Organization for Rare Disorders.
Most patients with rare diseases go through a “diagnostic odyssey” lasting an average of 8 years before they receive an accurate diagnosis, she said. During this time, data suggest that they have likely been misdiagnosed three times and have seen more than 10 specialists, she added.
Barriers to genetic screening include limited access to genetics professionals, lack of patient and provider education about screening, issues of insurance coverage and reimbursement, coding challenges, and misperceptions about the perceived impact of screening, noted Jodie Vento, manager of the Center for Rare Disease Therapy at the Children’s Hospital of Pittsburgh.
The genetic carrier screening options, often referred to as panethnic expanded carrier screening, represents a change from previous screening protocols based on ethnicity, said Ms. Arjunan. However, guidelines for screening based on ethnicity “misses a significant percentage of pregnancies affected by serious conditions and widens the health disparity gap,” she said.
By contrast, expanded carrier screening allows for standardization of care that gives couples and families information to make decisions and preparations.
Current genetic testing strategies include single gene testing, in which a single gene of interest is tested; multigene panel testing, in which a subset of clinically important genes are tested; whole-exome sequencing, in which the DNA responsible for coding proteins is tested; and whole-genome sequencing, in which the entire human genome is tested for genetic disorders.
Improving access to genetic testing involves a combination of provider education, changes in payer policies, action by advocacy groups, and adjustment of societal guidelines, said Ms. Arjunan. However, the advantages of expanded carrier screening are many and include guiding patients to expert care early and setting up plans for long-term care and follow-up, she noted. In addition, early identification through screening can help patients reduce or eliminate the diagnostic odyssey and connect with advocacy and community groups for support, she concluded.
The presenters had no financial conflicts to disclose.
EXPERT ANALYSIS FROM NORD 2019
FDA advisory committee supports birth control patch approval
Most of the committee members based their decisions on the need for additional contraceptive options for patients. However, most also expressed concerns about its efficacy and offered suggestions for product labeling that called attention to high rates of unintended pregnancies and increased risk of venous thromboembolism (VTE) in obese women.
The agency’s Bone, Reproductive and Urologic Drugs Advisory Committee reviewed safety and efficacy data for AG200-15, a combined hormonal contraceptive patch developed by Agile Therapeutics. The treatment regimen involves application of a patch to the abdomen, buttock, or upper torso, and the patch is changed weekly for 3 weeks, followed by 1 week without a patch.
Elizabeth Garner, MD, consultant and former chief medical officer of Agile, presented study data on safety and effectiveness of the patch. The key study (known as Study 23) considered by the FDA included 1,736 women aged 35 years and younger. The primary efficacy endpoint was the pregnancy rate in the women who used the patch. Women reported sexual activity and back-up contraception use in e-diaries.
A total of 68 pregnancies occurred in the study population after 15,165 evaluable cycles, yielding an overall Pearl Index of 5.83 across all weight and body mass index groups. Historically, a Pearl Index of 5 has been the standard measure for effectiveness in contraceptive products, with lower being better. The index is defined as the number of pregnancies per 100 woman-years of product use. For example, a Pearl Index of 0.1 means that 1 in 1,000 women who use the same contraceptive method for 1 year becomes pregnant.
A subgroup analysis showed reduced efficacy in women with a higher BMI. The Pearl Index for women with a BMI of less than 30 kg/m2 (defined as nonobese) was 4.34, whereas in women with a BMI of 30 kg/m2 and higher (defined as obese), the index was 8.64, nearly double that of nonobese women. No significant differences in the index were noted based on race/ethnicity.
The company described the patch as filling a niche and providing an additional alternative for women seeking a noninvasive method of contraception. It proposed a limitation of use (LOU) as part of the product label that would provide detailed information on efficacy based on the Pearl Index for the different categories of BMI and would suggest that the patch may be less effective for women with obesity. Most of the committee members favored use of a LOU statement on the label, but some noted that it might limit prescriptions to nonobese women.
The committee expressed concern over the Pearl data in the study. The FDA has never approved a contraceptive product with a Pearl Index of greater than 5, said Yun Tang, PhD, a statistical reviewer for the agency’s Office of Translational Sciences, who presented the evaluation of the effectiveness of AG200-15.
Key safety concerns raised in discussion included the risk of venous thromboembolism and the risk of unscheduled bleeding. Both of those issues were significantly more common among obese women, said Nneka McNeal-Jackson, MD, clinical reviewer for the FDA, who presented details on the safety profile and risk-benefit considerations for the patch.
Overall, in Study 23, the incidence rate of VTE was 28/10,000 women-years, with cases in five participants. Four of those were deemed related to the patch, and all occurred in obese women.
Virginia C. “Jennie” Leslie, MD, of Oregon Health and Science University, Portland, voted no to recommending approval of the patch mainly because of efficacy concerns. “My goal is to do no harm, and I have concerns regarding efficacy and giving our patients a false sense of hope,” she said.
Even those members who voted yes expressed concerns about the efficacy data and VTE risk in obese women and recommended postmarketing studies and appropriate labeling to help clinicians in shared decision making with their patients.
Esther Eisenberg, MD, of the National Institutes of Health, noted that the patch fills a need, certainly for women with a BMI less than 30 kg/m2, and suggested that use be limited to women in that lower BMI category.
Other committee members suggested that the product not be restricted based on BMI, but rather that the LOU provide clear explanations of how effectiveness decreases as BMI increases.
David J. Margolis, MD, of the University of Pennsylvania, Philadelphia, opted to abstain from voting, in part based on concerns about the study design and a lack of additional data from the company.
Most of the committee members based their decisions on the need for additional contraceptive options for patients. However, most also expressed concerns about its efficacy and offered suggestions for product labeling that called attention to high rates of unintended pregnancies and increased risk of venous thromboembolism (VTE) in obese women.
The agency’s Bone, Reproductive and Urologic Drugs Advisory Committee reviewed safety and efficacy data for AG200-15, a combined hormonal contraceptive patch developed by Agile Therapeutics. The treatment regimen involves application of a patch to the abdomen, buttock, or upper torso, and the patch is changed weekly for 3 weeks, followed by 1 week without a patch.
Elizabeth Garner, MD, consultant and former chief medical officer of Agile, presented study data on safety and effectiveness of the patch. The key study (known as Study 23) considered by the FDA included 1,736 women aged 35 years and younger. The primary efficacy endpoint was the pregnancy rate in the women who used the patch. Women reported sexual activity and back-up contraception use in e-diaries.
A total of 68 pregnancies occurred in the study population after 15,165 evaluable cycles, yielding an overall Pearl Index of 5.83 across all weight and body mass index groups. Historically, a Pearl Index of 5 has been the standard measure for effectiveness in contraceptive products, with lower being better. The index is defined as the number of pregnancies per 100 woman-years of product use. For example, a Pearl Index of 0.1 means that 1 in 1,000 women who use the same contraceptive method for 1 year becomes pregnant.
A subgroup analysis showed reduced efficacy in women with a higher BMI. The Pearl Index for women with a BMI of less than 30 kg/m2 (defined as nonobese) was 4.34, whereas in women with a BMI of 30 kg/m2 and higher (defined as obese), the index was 8.64, nearly double that of nonobese women. No significant differences in the index were noted based on race/ethnicity.
The company described the patch as filling a niche and providing an additional alternative for women seeking a noninvasive method of contraception. It proposed a limitation of use (LOU) as part of the product label that would provide detailed information on efficacy based on the Pearl Index for the different categories of BMI and would suggest that the patch may be less effective for women with obesity. Most of the committee members favored use of a LOU statement on the label, but some noted that it might limit prescriptions to nonobese women.
The committee expressed concern over the Pearl data in the study. The FDA has never approved a contraceptive product with a Pearl Index of greater than 5, said Yun Tang, PhD, a statistical reviewer for the agency’s Office of Translational Sciences, who presented the evaluation of the effectiveness of AG200-15.
Key safety concerns raised in discussion included the risk of venous thromboembolism and the risk of unscheduled bleeding. Both of those issues were significantly more common among obese women, said Nneka McNeal-Jackson, MD, clinical reviewer for the FDA, who presented details on the safety profile and risk-benefit considerations for the patch.
Overall, in Study 23, the incidence rate of VTE was 28/10,000 women-years, with cases in five participants. Four of those were deemed related to the patch, and all occurred in obese women.
Virginia C. “Jennie” Leslie, MD, of Oregon Health and Science University, Portland, voted no to recommending approval of the patch mainly because of efficacy concerns. “My goal is to do no harm, and I have concerns regarding efficacy and giving our patients a false sense of hope,” she said.
Even those members who voted yes expressed concerns about the efficacy data and VTE risk in obese women and recommended postmarketing studies and appropriate labeling to help clinicians in shared decision making with their patients.
Esther Eisenberg, MD, of the National Institutes of Health, noted that the patch fills a need, certainly for women with a BMI less than 30 kg/m2, and suggested that use be limited to women in that lower BMI category.
Other committee members suggested that the product not be restricted based on BMI, but rather that the LOU provide clear explanations of how effectiveness decreases as BMI increases.
David J. Margolis, MD, of the University of Pennsylvania, Philadelphia, opted to abstain from voting, in part based on concerns about the study design and a lack of additional data from the company.
Most of the committee members based their decisions on the need for additional contraceptive options for patients. However, most also expressed concerns about its efficacy and offered suggestions for product labeling that called attention to high rates of unintended pregnancies and increased risk of venous thromboembolism (VTE) in obese women.
The agency’s Bone, Reproductive and Urologic Drugs Advisory Committee reviewed safety and efficacy data for AG200-15, a combined hormonal contraceptive patch developed by Agile Therapeutics. The treatment regimen involves application of a patch to the abdomen, buttock, or upper torso, and the patch is changed weekly for 3 weeks, followed by 1 week without a patch.
Elizabeth Garner, MD, consultant and former chief medical officer of Agile, presented study data on safety and effectiveness of the patch. The key study (known as Study 23) considered by the FDA included 1,736 women aged 35 years and younger. The primary efficacy endpoint was the pregnancy rate in the women who used the patch. Women reported sexual activity and back-up contraception use in e-diaries.
A total of 68 pregnancies occurred in the study population after 15,165 evaluable cycles, yielding an overall Pearl Index of 5.83 across all weight and body mass index groups. Historically, a Pearl Index of 5 has been the standard measure for effectiveness in contraceptive products, with lower being better. The index is defined as the number of pregnancies per 100 woman-years of product use. For example, a Pearl Index of 0.1 means that 1 in 1,000 women who use the same contraceptive method for 1 year becomes pregnant.
A subgroup analysis showed reduced efficacy in women with a higher BMI. The Pearl Index for women with a BMI of less than 30 kg/m2 (defined as nonobese) was 4.34, whereas in women with a BMI of 30 kg/m2 and higher (defined as obese), the index was 8.64, nearly double that of nonobese women. No significant differences in the index were noted based on race/ethnicity.
The company described the patch as filling a niche and providing an additional alternative for women seeking a noninvasive method of contraception. It proposed a limitation of use (LOU) as part of the product label that would provide detailed information on efficacy based on the Pearl Index for the different categories of BMI and would suggest that the patch may be less effective for women with obesity. Most of the committee members favored use of a LOU statement on the label, but some noted that it might limit prescriptions to nonobese women.
The committee expressed concern over the Pearl data in the study. The FDA has never approved a contraceptive product with a Pearl Index of greater than 5, said Yun Tang, PhD, a statistical reviewer for the agency’s Office of Translational Sciences, who presented the evaluation of the effectiveness of AG200-15.
Key safety concerns raised in discussion included the risk of venous thromboembolism and the risk of unscheduled bleeding. Both of those issues were significantly more common among obese women, said Nneka McNeal-Jackson, MD, clinical reviewer for the FDA, who presented details on the safety profile and risk-benefit considerations for the patch.
Overall, in Study 23, the incidence rate of VTE was 28/10,000 women-years, with cases in five participants. Four of those were deemed related to the patch, and all occurred in obese women.
Virginia C. “Jennie” Leslie, MD, of Oregon Health and Science University, Portland, voted no to recommending approval of the patch mainly because of efficacy concerns. “My goal is to do no harm, and I have concerns regarding efficacy and giving our patients a false sense of hope,” she said.
Even those members who voted yes expressed concerns about the efficacy data and VTE risk in obese women and recommended postmarketing studies and appropriate labeling to help clinicians in shared decision making with their patients.
Esther Eisenberg, MD, of the National Institutes of Health, noted that the patch fills a need, certainly for women with a BMI less than 30 kg/m2, and suggested that use be limited to women in that lower BMI category.
Other committee members suggested that the product not be restricted based on BMI, but rather that the LOU provide clear explanations of how effectiveness decreases as BMI increases.
David J. Margolis, MD, of the University of Pennsylvania, Philadelphia, opted to abstain from voting, in part based on concerns about the study design and a lack of additional data from the company.
FROM THE FDA
ACIP approves child and adolescent vaccination schedule for 2020
The Centers for Disease Control and Prevention’s Advisory Committee on Immunization Practices voted unanimously to approve the child and adolescent immunization schedule for 2020.
by busy providers,” Candice Robinson, MD, MPH, of the CDC’s National Center for Immunization and Respiratory Diseases, said at the CDC’s October meeting of ACIP. Updates reflect changes in language in the adult vaccination schedule, notably the change in the definition of “contraindication.” The updated wording in the Notes substitutes “not recommended or contraindicated” instead of the word “contraindicated” only.
Another notable change was the addition of information on adolescent vaccination of children who received the meningococcal ACWY vaccine before 10 years of age. For “children in whom boosters are not recommended due to an ongoing or increased risk of meningococcal disease” (such as a healthy child traveling to an endemic area), they should receive MenACWY according to the recommended adolescent schedule. But those children for whom boosters are recommended because of increased disease risk from conditions including complement deficiency, HIV, or asplenia should “follow the booster schedule for persons at increased risk.”
Other changes include restructuring of the notes for the live attenuated influenza vaccine (LAIV) in special situations. The schedule now uses a bulleted list to show that LAIV should not be used in the following circumstances:
- Having history of severe allergic reaction to a previous vaccine or vaccine component.
- Using aspirin or a salicylate-containing medication.
- Being aged 2-4 years with a history of asthma or wheezing.
- Having immunocompromised conditions.
- Having anatomic or functional asplenia.
- Having cochlear implants.
- Experiencing cerebrospinal fluid–oropharyngeal communication.
- Having immunocompromised close contacts or caregivers.
- Being pregnant.
- Having received flu antivirals within the previous 48 hours.
In addition, language on shared clinical decision-making was added to the notes on the meningococcal B vaccine for adolescents and young adults aged 18-23 years not at increased risk. Based on shared clinical decision making, the recommendation is a “two-dose series of Bexsero at least 1 month apart” or “two-dose series of Trumenba at least 6 months apart; if dose two is administered earlier than 6 months, administer a third dose at least 4 months after dose two.”
Several vaccines’ Notes sections, including hepatitis B and meningococcal disease, added links to detailed recommendations in the corresponding issues of the CDC’s Morbidity and Mortality Weekly Report, to allow clinicians easy access to additional information.
View the current Child & Adolescent Vaccination Schedule here.
The ACIP members had no financial conflicts to disclose.
The Centers for Disease Control and Prevention’s Advisory Committee on Immunization Practices voted unanimously to approve the child and adolescent immunization schedule for 2020.
by busy providers,” Candice Robinson, MD, MPH, of the CDC’s National Center for Immunization and Respiratory Diseases, said at the CDC’s October meeting of ACIP. Updates reflect changes in language in the adult vaccination schedule, notably the change in the definition of “contraindication.” The updated wording in the Notes substitutes “not recommended or contraindicated” instead of the word “contraindicated” only.
Another notable change was the addition of information on adolescent vaccination of children who received the meningococcal ACWY vaccine before 10 years of age. For “children in whom boosters are not recommended due to an ongoing or increased risk of meningococcal disease” (such as a healthy child traveling to an endemic area), they should receive MenACWY according to the recommended adolescent schedule. But those children for whom boosters are recommended because of increased disease risk from conditions including complement deficiency, HIV, or asplenia should “follow the booster schedule for persons at increased risk.”
Other changes include restructuring of the notes for the live attenuated influenza vaccine (LAIV) in special situations. The schedule now uses a bulleted list to show that LAIV should not be used in the following circumstances:
- Having history of severe allergic reaction to a previous vaccine or vaccine component.
- Using aspirin or a salicylate-containing medication.
- Being aged 2-4 years with a history of asthma or wheezing.
- Having immunocompromised conditions.
- Having anatomic or functional asplenia.
- Having cochlear implants.
- Experiencing cerebrospinal fluid–oropharyngeal communication.
- Having immunocompromised close contacts or caregivers.
- Being pregnant.
- Having received flu antivirals within the previous 48 hours.
In addition, language on shared clinical decision-making was added to the notes on the meningococcal B vaccine for adolescents and young adults aged 18-23 years not at increased risk. Based on shared clinical decision making, the recommendation is a “two-dose series of Bexsero at least 1 month apart” or “two-dose series of Trumenba at least 6 months apart; if dose two is administered earlier than 6 months, administer a third dose at least 4 months after dose two.”
Several vaccines’ Notes sections, including hepatitis B and meningococcal disease, added links to detailed recommendations in the corresponding issues of the CDC’s Morbidity and Mortality Weekly Report, to allow clinicians easy access to additional information.
View the current Child & Adolescent Vaccination Schedule here.
The ACIP members had no financial conflicts to disclose.
The Centers for Disease Control and Prevention’s Advisory Committee on Immunization Practices voted unanimously to approve the child and adolescent immunization schedule for 2020.
by busy providers,” Candice Robinson, MD, MPH, of the CDC’s National Center for Immunization and Respiratory Diseases, said at the CDC’s October meeting of ACIP. Updates reflect changes in language in the adult vaccination schedule, notably the change in the definition of “contraindication.” The updated wording in the Notes substitutes “not recommended or contraindicated” instead of the word “contraindicated” only.
Another notable change was the addition of information on adolescent vaccination of children who received the meningococcal ACWY vaccine before 10 years of age. For “children in whom boosters are not recommended due to an ongoing or increased risk of meningococcal disease” (such as a healthy child traveling to an endemic area), they should receive MenACWY according to the recommended adolescent schedule. But those children for whom boosters are recommended because of increased disease risk from conditions including complement deficiency, HIV, or asplenia should “follow the booster schedule for persons at increased risk.”
Other changes include restructuring of the notes for the live attenuated influenza vaccine (LAIV) in special situations. The schedule now uses a bulleted list to show that LAIV should not be used in the following circumstances:
- Having history of severe allergic reaction to a previous vaccine or vaccine component.
- Using aspirin or a salicylate-containing medication.
- Being aged 2-4 years with a history of asthma or wheezing.
- Having immunocompromised conditions.
- Having anatomic or functional asplenia.
- Having cochlear implants.
- Experiencing cerebrospinal fluid–oropharyngeal communication.
- Having immunocompromised close contacts or caregivers.
- Being pregnant.
- Having received flu antivirals within the previous 48 hours.
In addition, language on shared clinical decision-making was added to the notes on the meningococcal B vaccine for adolescents and young adults aged 18-23 years not at increased risk. Based on shared clinical decision making, the recommendation is a “two-dose series of Bexsero at least 1 month apart” or “two-dose series of Trumenba at least 6 months apart; if dose two is administered earlier than 6 months, administer a third dose at least 4 months after dose two.”
Several vaccines’ Notes sections, including hepatitis B and meningococcal disease, added links to detailed recommendations in the corresponding issues of the CDC’s Morbidity and Mortality Weekly Report, to allow clinicians easy access to additional information.
View the current Child & Adolescent Vaccination Schedule here.
The ACIP members had no financial conflicts to disclose.
FROM AN ACIP MEETING
ACIP approves 2020 adult vaccination schedule
The Centers for Disease Control and Prevention’s Advisory Committee on Immunization Practices voted unanimously to approve the adult immunization schedule for 2020, although some fine-tuning may occur before publication.
“Some of the wordsmithing may be done later,” ACIP executive secretary Amanda Cohn, MD, said at the ACIP October meeting.
Key updates to the schedule included a change in wording for the definition of the red bars on the table to include “not recommended or contraindicated” instead of only the word “contraindicated.” Committee members were especially interested in changing this wording to guide clinicians in use of the live attenuated influenza vaccine because of its potential value in vaccinating health care personnel.
Other updates include language that vaccination of adolescents and young adults aged 16-23 years who are not at increased risk for meningococcal disease should be vaccinated as follows: “Based on shared clinical decision making, 2-dose series MenB-4C at least 1 month apart or 2-dose series MenB-FHbp at 0, 6 months.”
Similarly, clinical decision-making language was added to the notes for the pneumococcal polysaccharide vaccine (PPSV23) and the 13-valent pneumococcal conjugate vaccine (PCV13).
The routine vaccination calls for only one dose of PPSV23 given on or after the individual’s 65th birthday. Then, based on shared clinical decision making, a dose of PCV13 is recommended for immunocompetent individuals aged 65 years and older. The notes also state that, based on shared clinical decision making, PCV13 and PPSV23 should not be given in the same visit and, if both will be given, PCV13 should be first and should be given 1 year before PPSV23. In addition, “PPSV23 should be given at least 5 years after any previous PPSV23 dose.”
The schedule also adds shared clinical decision making to the notes on human papillomavirus vaccination for adults aged 27-45 years.
The committee members acknowledged the increasing complexity of the adult vaccination schedule, but several members agreed that it is accessible to many clinicians.
“We can’t let the perfect be the enemy of the good” said Jason Goldman, MD, liaison representing the American College of Physicians. “Those who want to learn the schedule will learn it; the health system will learn it,” even if not every specialist does.
The table “is something to draw you in,” said Sandra Fryhofer, MD, an internist who is liaison for the American Medical Association. The notes provide more details.
More specific information about contraindications for patients with cochlear implants, which also came up in the discussion, may be added to the schedule at a later date.
View the current adult vaccination schedule here.
The ACIP members had no financial conflicts to disclose.
The Centers for Disease Control and Prevention’s Advisory Committee on Immunization Practices voted unanimously to approve the adult immunization schedule for 2020, although some fine-tuning may occur before publication.
“Some of the wordsmithing may be done later,” ACIP executive secretary Amanda Cohn, MD, said at the ACIP October meeting.
Key updates to the schedule included a change in wording for the definition of the red bars on the table to include “not recommended or contraindicated” instead of only the word “contraindicated.” Committee members were especially interested in changing this wording to guide clinicians in use of the live attenuated influenza vaccine because of its potential value in vaccinating health care personnel.
Other updates include language that vaccination of adolescents and young adults aged 16-23 years who are not at increased risk for meningococcal disease should be vaccinated as follows: “Based on shared clinical decision making, 2-dose series MenB-4C at least 1 month apart or 2-dose series MenB-FHbp at 0, 6 months.”
Similarly, clinical decision-making language was added to the notes for the pneumococcal polysaccharide vaccine (PPSV23) and the 13-valent pneumococcal conjugate vaccine (PCV13).
The routine vaccination calls for only one dose of PPSV23 given on or after the individual’s 65th birthday. Then, based on shared clinical decision making, a dose of PCV13 is recommended for immunocompetent individuals aged 65 years and older. The notes also state that, based on shared clinical decision making, PCV13 and PPSV23 should not be given in the same visit and, if both will be given, PCV13 should be first and should be given 1 year before PPSV23. In addition, “PPSV23 should be given at least 5 years after any previous PPSV23 dose.”
The schedule also adds shared clinical decision making to the notes on human papillomavirus vaccination for adults aged 27-45 years.
The committee members acknowledged the increasing complexity of the adult vaccination schedule, but several members agreed that it is accessible to many clinicians.
“We can’t let the perfect be the enemy of the good” said Jason Goldman, MD, liaison representing the American College of Physicians. “Those who want to learn the schedule will learn it; the health system will learn it,” even if not every specialist does.
The table “is something to draw you in,” said Sandra Fryhofer, MD, an internist who is liaison for the American Medical Association. The notes provide more details.
More specific information about contraindications for patients with cochlear implants, which also came up in the discussion, may be added to the schedule at a later date.
View the current adult vaccination schedule here.
The ACIP members had no financial conflicts to disclose.
The Centers for Disease Control and Prevention’s Advisory Committee on Immunization Practices voted unanimously to approve the adult immunization schedule for 2020, although some fine-tuning may occur before publication.
“Some of the wordsmithing may be done later,” ACIP executive secretary Amanda Cohn, MD, said at the ACIP October meeting.
Key updates to the schedule included a change in wording for the definition of the red bars on the table to include “not recommended or contraindicated” instead of only the word “contraindicated.” Committee members were especially interested in changing this wording to guide clinicians in use of the live attenuated influenza vaccine because of its potential value in vaccinating health care personnel.
Other updates include language that vaccination of adolescents and young adults aged 16-23 years who are not at increased risk for meningococcal disease should be vaccinated as follows: “Based on shared clinical decision making, 2-dose series MenB-4C at least 1 month apart or 2-dose series MenB-FHbp at 0, 6 months.”
Similarly, clinical decision-making language was added to the notes for the pneumococcal polysaccharide vaccine (PPSV23) and the 13-valent pneumococcal conjugate vaccine (PCV13).
The routine vaccination calls for only one dose of PPSV23 given on or after the individual’s 65th birthday. Then, based on shared clinical decision making, a dose of PCV13 is recommended for immunocompetent individuals aged 65 years and older. The notes also state that, based on shared clinical decision making, PCV13 and PPSV23 should not be given in the same visit and, if both will be given, PCV13 should be first and should be given 1 year before PPSV23. In addition, “PPSV23 should be given at least 5 years after any previous PPSV23 dose.”
The schedule also adds shared clinical decision making to the notes on human papillomavirus vaccination for adults aged 27-45 years.
The committee members acknowledged the increasing complexity of the adult vaccination schedule, but several members agreed that it is accessible to many clinicians.
“We can’t let the perfect be the enemy of the good” said Jason Goldman, MD, liaison representing the American College of Physicians. “Those who want to learn the schedule will learn it; the health system will learn it,” even if not every specialist does.
The table “is something to draw you in,” said Sandra Fryhofer, MD, an internist who is liaison for the American Medical Association. The notes provide more details.
More specific information about contraindications for patients with cochlear implants, which also came up in the discussion, may be added to the schedule at a later date.
View the current adult vaccination schedule here.
The ACIP members had no financial conflicts to disclose.
ACIP plans flu review for older adults
according to data presented at a meeting of the Centers for Disease Control and Prevention’s ACIP.
Lynette Brammer of the CDC’s National Center for Immunization and Respiratory Diseases (NCIRD) presented a surveillance update of the flu season in the United States so far. Overall, the influenza A(H3N2) viruses are predominant, although dominance varies in different regions of the country, and it is too soon to predict what strain will dominate later in the season.
“While two of the four vaccine components were updated for the Southern Hemisphere, the components selected for the 2019-2020 Northern Hemisphere vaccine, at this time, look appropriate for the season,” she said.
In other flu news, Lisa Groskopf, MD, of the NCIRD discussed the influenza work group’s plans for a meta-analysis to assess the relative benefit of different vaccines for older adults, in light of the growing variety of products available.
Currently, no preferential recommendations have been made for a specific vaccine for a particular age group. “There’s a dearth of data comparing these vaccines to one another,” said Dr. Groskopf. She added that, because vaccine effectiveness varies by season, the generalizability of effectiveness data is another challenge.
The work group’s systematic review and meta-analysis is designed to compare the high-dose inactivated influenza vaccine (HD-IIV), the adjuvanted inactivated influenza vaccine (aIIV), and the recombinant influenza vaccine (RIV). The study will include adults aged 65 years and older who receive trivalent or quadrivalent HD-IIV, aIIV, or RIV, compared with those who receive another influenza vaccine, a noninfluenza control vaccine, placebo, or no vaccine. The outcomes will include data on safety and effectiveness of the vaccines, Dr. Groskopf said.
In addition to safety and effectiveness, manufacturers such as Sanofi Pasteur continue to collect data on the success of available vaccines and develop new ones. Lee-Jah Chang, MD, of Sanofi Pasteur presented results of a noninferiority study of the company’s investigational high-dose quadrivalent influenza vaccine (QIV-HD; including two prevailing B viruses) versus the high-dose trivalent influenza vaccine (TID-HD). The study was conducted at 35 sites in the United States and included 2,670 adults aged 65 years and older.
Overall, the reactogenicity profile for patients given QIV-HD was similar to that of TID-HD, and approximately 5% of patients in the QIV group reported an immediate adverse event, Dr. Chang said. However, no related deaths or related adverse events of special interest occurred in any of the study groups.
Sanofi plans to pursue licensure of the QIV-HD vaccine, with a Center for Biologics Evaluation and Research action date of Nov. 4, 2019, said Dr. Chang. If the vaccine is licensed, it should be available for purchase by health care providers in the first quarter of 2020.
The ACIP members had no financial conflicts to disclose.
according to data presented at a meeting of the Centers for Disease Control and Prevention’s ACIP.
Lynette Brammer of the CDC’s National Center for Immunization and Respiratory Diseases (NCIRD) presented a surveillance update of the flu season in the United States so far. Overall, the influenza A(H3N2) viruses are predominant, although dominance varies in different regions of the country, and it is too soon to predict what strain will dominate later in the season.
“While two of the four vaccine components were updated for the Southern Hemisphere, the components selected for the 2019-2020 Northern Hemisphere vaccine, at this time, look appropriate for the season,” she said.
In other flu news, Lisa Groskopf, MD, of the NCIRD discussed the influenza work group’s plans for a meta-analysis to assess the relative benefit of different vaccines for older adults, in light of the growing variety of products available.
Currently, no preferential recommendations have been made for a specific vaccine for a particular age group. “There’s a dearth of data comparing these vaccines to one another,” said Dr. Groskopf. She added that, because vaccine effectiveness varies by season, the generalizability of effectiveness data is another challenge.
The work group’s systematic review and meta-analysis is designed to compare the high-dose inactivated influenza vaccine (HD-IIV), the adjuvanted inactivated influenza vaccine (aIIV), and the recombinant influenza vaccine (RIV). The study will include adults aged 65 years and older who receive trivalent or quadrivalent HD-IIV, aIIV, or RIV, compared with those who receive another influenza vaccine, a noninfluenza control vaccine, placebo, or no vaccine. The outcomes will include data on safety and effectiveness of the vaccines, Dr. Groskopf said.
In addition to safety and effectiveness, manufacturers such as Sanofi Pasteur continue to collect data on the success of available vaccines and develop new ones. Lee-Jah Chang, MD, of Sanofi Pasteur presented results of a noninferiority study of the company’s investigational high-dose quadrivalent influenza vaccine (QIV-HD; including two prevailing B viruses) versus the high-dose trivalent influenza vaccine (TID-HD). The study was conducted at 35 sites in the United States and included 2,670 adults aged 65 years and older.
Overall, the reactogenicity profile for patients given QIV-HD was similar to that of TID-HD, and approximately 5% of patients in the QIV group reported an immediate adverse event, Dr. Chang said. However, no related deaths or related adverse events of special interest occurred in any of the study groups.
Sanofi plans to pursue licensure of the QIV-HD vaccine, with a Center for Biologics Evaluation and Research action date of Nov. 4, 2019, said Dr. Chang. If the vaccine is licensed, it should be available for purchase by health care providers in the first quarter of 2020.
The ACIP members had no financial conflicts to disclose.
according to data presented at a meeting of the Centers for Disease Control and Prevention’s ACIP.
Lynette Brammer of the CDC’s National Center for Immunization and Respiratory Diseases (NCIRD) presented a surveillance update of the flu season in the United States so far. Overall, the influenza A(H3N2) viruses are predominant, although dominance varies in different regions of the country, and it is too soon to predict what strain will dominate later in the season.
“While two of the four vaccine components were updated for the Southern Hemisphere, the components selected for the 2019-2020 Northern Hemisphere vaccine, at this time, look appropriate for the season,” she said.
In other flu news, Lisa Groskopf, MD, of the NCIRD discussed the influenza work group’s plans for a meta-analysis to assess the relative benefit of different vaccines for older adults, in light of the growing variety of products available.
Currently, no preferential recommendations have been made for a specific vaccine for a particular age group. “There’s a dearth of data comparing these vaccines to one another,” said Dr. Groskopf. She added that, because vaccine effectiveness varies by season, the generalizability of effectiveness data is another challenge.
The work group’s systematic review and meta-analysis is designed to compare the high-dose inactivated influenza vaccine (HD-IIV), the adjuvanted inactivated influenza vaccine (aIIV), and the recombinant influenza vaccine (RIV). The study will include adults aged 65 years and older who receive trivalent or quadrivalent HD-IIV, aIIV, or RIV, compared with those who receive another influenza vaccine, a noninfluenza control vaccine, placebo, or no vaccine. The outcomes will include data on safety and effectiveness of the vaccines, Dr. Groskopf said.
In addition to safety and effectiveness, manufacturers such as Sanofi Pasteur continue to collect data on the success of available vaccines and develop new ones. Lee-Jah Chang, MD, of Sanofi Pasteur presented results of a noninferiority study of the company’s investigational high-dose quadrivalent influenza vaccine (QIV-HD; including two prevailing B viruses) versus the high-dose trivalent influenza vaccine (TID-HD). The study was conducted at 35 sites in the United States and included 2,670 adults aged 65 years and older.
Overall, the reactogenicity profile for patients given QIV-HD was similar to that of TID-HD, and approximately 5% of patients in the QIV group reported an immediate adverse event, Dr. Chang said. However, no related deaths or related adverse events of special interest occurred in any of the study groups.
Sanofi plans to pursue licensure of the QIV-HD vaccine, with a Center for Biologics Evaluation and Research action date of Nov. 4, 2019, said Dr. Chang. If the vaccine is licensed, it should be available for purchase by health care providers in the first quarter of 2020.
The ACIP members had no financial conflicts to disclose.
REPORTING FROM AN ACIP MEETING