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Hospitalist movers and shakers – May 2019
Christina L. Andrew, DO, a medical director on the hospitalist team at McLeod Regional Medical Center in Florence, S.C., and Zeshan Anwar, MD, medical director of Evangelical Community Hospital’s hospitalist group in Lewisburg, Pa., recently were named Senior Fellows in Hospital Medicine (SFHM) by the Society of Hospital Medicine. SFHMs are dedicated to promoting excellence, innovation and improving the quality of patient care.
Dr. Andrew has been with McLeod since 2008. The board-certified internist received her medical degree from Des Moines (Iowa) University Osteopathic Medical Center and did her residency at the Cleveland Clinic. To earn SFHM status, physicians must have worked as a hospitalist for at least 5 years and be a member of SHM for 5 years, as well.
Dr. Anwar has been in his current position since 2015. He coordinates staff resources and inpatient care for the facility where he has worked since 2013. He has his medical degree from King Edward Medical University, Lahore, Pakistan, and did his residency at Bronx-Lebanon Hospital Center in New York.
Tiffany Egbe, MD, has been named to the board of directors of Refuge International, an organization that builds relationships in Guatemala that allow for medical services to be provided to an underserved population.
Dr. Egbe is a hospitalist in internal medicine at Christus Good Shepherd in Longview and Marshall, Tex. She also serves as program director of internal medicine residency for the University of Texas Health Science Center in Tyler, Tex.
Dr. Egbe earned her medical degree from the University of Alabama at Birmingham.
Il Jun Chon, MD, has been named vice president of medical affairs with WellSpan Ephrata (Pa.) Community Hospital. Dr. Chon, a hospitalist, had previously been the medical director of WellSpan Ephrata’s hospitalist services and president of the facility’s medical staff.
Dr. Chon earned his medical degree from the Medical College of Pennsylvania (now Drexel College of Medicine) and completed his residency at Thomas Jefferson University Hospital, both in Philadelphia.
Megan Hamreus, DO, recently was named chief of staff at Scripps Mercy Hospital in San Diego, Calif. Dr. Hamreus will oversee 1,000 doctors at two facilities.
Dr. Hamreus has been with Scripps Mercy for 10 years, serving as a hospitalist and a faculty member of the family medicine residence training program of Family Health Centers of San Diego.
Chief of staff is a 2-year, elected term. Among her duties, Dr. Hamreus will be Scripps Mercy’s liaison to the facilities’ administrative staff and Scripps Health’s board of trustees.
Jade Brice Roshell, MD, recently was named chief medical officer at Shelby Baptist Medical Center in Alabaster, Ala. Dr. Brice Roshell was promoted from director of the center’s hospitalist program.
In addition to her new position, Dr. Brice Roshell was named as one of 68 honorees on Becker’s 2019 list of African-American Leaders in Health.
Dr. Brice Roshell has been with Shelby Baptist since 2015. Previously, she was an internist at centers in Louisiana, Georgia, and Nebraska. Her medical degree is from Howard University in Washington, and she completed her residency at Tulane University in New Orleans.
Anju Manral, MD, recently was appointed as medical director for the University of New Mexico Student Health and Counseling Center in Albuquerque. The internist has experience as a hospitalist focused on palliative care and most recently has worked at UNM’s Family Health Clinic, providing care to patients of all ages and conditions.
Dr. Manral also serves as an assistant professor in the UNM General Internal Medicine Department and mentors UNM medical students in the health science learning community.
The Hiawatha (Kan.) Community Hospital unveiled its new hospitalist program on Feb. 12.
The program will be led by Dustin Williams, DNP. Dr. Williams will provide hospitalist and emergency medical services to patients every Tuesday through Friday, while an on-call specialist will serve as hospitalist on Saturday, Sunday, and Monday.
Christina L. Andrew, DO, a medical director on the hospitalist team at McLeod Regional Medical Center in Florence, S.C., and Zeshan Anwar, MD, medical director of Evangelical Community Hospital’s hospitalist group in Lewisburg, Pa., recently were named Senior Fellows in Hospital Medicine (SFHM) by the Society of Hospital Medicine. SFHMs are dedicated to promoting excellence, innovation and improving the quality of patient care.
Dr. Andrew has been with McLeod since 2008. The board-certified internist received her medical degree from Des Moines (Iowa) University Osteopathic Medical Center and did her residency at the Cleveland Clinic. To earn SFHM status, physicians must have worked as a hospitalist for at least 5 years and be a member of SHM for 5 years, as well.
Dr. Anwar has been in his current position since 2015. He coordinates staff resources and inpatient care for the facility where he has worked since 2013. He has his medical degree from King Edward Medical University, Lahore, Pakistan, and did his residency at Bronx-Lebanon Hospital Center in New York.
Tiffany Egbe, MD, has been named to the board of directors of Refuge International, an organization that builds relationships in Guatemala that allow for medical services to be provided to an underserved population.
Dr. Egbe is a hospitalist in internal medicine at Christus Good Shepherd in Longview and Marshall, Tex. She also serves as program director of internal medicine residency for the University of Texas Health Science Center in Tyler, Tex.
Dr. Egbe earned her medical degree from the University of Alabama at Birmingham.
Il Jun Chon, MD, has been named vice president of medical affairs with WellSpan Ephrata (Pa.) Community Hospital. Dr. Chon, a hospitalist, had previously been the medical director of WellSpan Ephrata’s hospitalist services and president of the facility’s medical staff.
Dr. Chon earned his medical degree from the Medical College of Pennsylvania (now Drexel College of Medicine) and completed his residency at Thomas Jefferson University Hospital, both in Philadelphia.
Megan Hamreus, DO, recently was named chief of staff at Scripps Mercy Hospital in San Diego, Calif. Dr. Hamreus will oversee 1,000 doctors at two facilities.
Dr. Hamreus has been with Scripps Mercy for 10 years, serving as a hospitalist and a faculty member of the family medicine residence training program of Family Health Centers of San Diego.
Chief of staff is a 2-year, elected term. Among her duties, Dr. Hamreus will be Scripps Mercy’s liaison to the facilities’ administrative staff and Scripps Health’s board of trustees.
Jade Brice Roshell, MD, recently was named chief medical officer at Shelby Baptist Medical Center in Alabaster, Ala. Dr. Brice Roshell was promoted from director of the center’s hospitalist program.
In addition to her new position, Dr. Brice Roshell was named as one of 68 honorees on Becker’s 2019 list of African-American Leaders in Health.
Dr. Brice Roshell has been with Shelby Baptist since 2015. Previously, she was an internist at centers in Louisiana, Georgia, and Nebraska. Her medical degree is from Howard University in Washington, and she completed her residency at Tulane University in New Orleans.
Anju Manral, MD, recently was appointed as medical director for the University of New Mexico Student Health and Counseling Center in Albuquerque. The internist has experience as a hospitalist focused on palliative care and most recently has worked at UNM’s Family Health Clinic, providing care to patients of all ages and conditions.
Dr. Manral also serves as an assistant professor in the UNM General Internal Medicine Department and mentors UNM medical students in the health science learning community.
The Hiawatha (Kan.) Community Hospital unveiled its new hospitalist program on Feb. 12.
The program will be led by Dustin Williams, DNP. Dr. Williams will provide hospitalist and emergency medical services to patients every Tuesday through Friday, while an on-call specialist will serve as hospitalist on Saturday, Sunday, and Monday.
Christina L. Andrew, DO, a medical director on the hospitalist team at McLeod Regional Medical Center in Florence, S.C., and Zeshan Anwar, MD, medical director of Evangelical Community Hospital’s hospitalist group in Lewisburg, Pa., recently were named Senior Fellows in Hospital Medicine (SFHM) by the Society of Hospital Medicine. SFHMs are dedicated to promoting excellence, innovation and improving the quality of patient care.
Dr. Andrew has been with McLeod since 2008. The board-certified internist received her medical degree from Des Moines (Iowa) University Osteopathic Medical Center and did her residency at the Cleveland Clinic. To earn SFHM status, physicians must have worked as a hospitalist for at least 5 years and be a member of SHM for 5 years, as well.
Dr. Anwar has been in his current position since 2015. He coordinates staff resources and inpatient care for the facility where he has worked since 2013. He has his medical degree from King Edward Medical University, Lahore, Pakistan, and did his residency at Bronx-Lebanon Hospital Center in New York.
Tiffany Egbe, MD, has been named to the board of directors of Refuge International, an organization that builds relationships in Guatemala that allow for medical services to be provided to an underserved population.
Dr. Egbe is a hospitalist in internal medicine at Christus Good Shepherd in Longview and Marshall, Tex. She also serves as program director of internal medicine residency for the University of Texas Health Science Center in Tyler, Tex.
Dr. Egbe earned her medical degree from the University of Alabama at Birmingham.
Il Jun Chon, MD, has been named vice president of medical affairs with WellSpan Ephrata (Pa.) Community Hospital. Dr. Chon, a hospitalist, had previously been the medical director of WellSpan Ephrata’s hospitalist services and president of the facility’s medical staff.
Dr. Chon earned his medical degree from the Medical College of Pennsylvania (now Drexel College of Medicine) and completed his residency at Thomas Jefferson University Hospital, both in Philadelphia.
Megan Hamreus, DO, recently was named chief of staff at Scripps Mercy Hospital in San Diego, Calif. Dr. Hamreus will oversee 1,000 doctors at two facilities.
Dr. Hamreus has been with Scripps Mercy for 10 years, serving as a hospitalist and a faculty member of the family medicine residence training program of Family Health Centers of San Diego.
Chief of staff is a 2-year, elected term. Among her duties, Dr. Hamreus will be Scripps Mercy’s liaison to the facilities’ administrative staff and Scripps Health’s board of trustees.
Jade Brice Roshell, MD, recently was named chief medical officer at Shelby Baptist Medical Center in Alabaster, Ala. Dr. Brice Roshell was promoted from director of the center’s hospitalist program.
In addition to her new position, Dr. Brice Roshell was named as one of 68 honorees on Becker’s 2019 list of African-American Leaders in Health.
Dr. Brice Roshell has been with Shelby Baptist since 2015. Previously, she was an internist at centers in Louisiana, Georgia, and Nebraska. Her medical degree is from Howard University in Washington, and she completed her residency at Tulane University in New Orleans.
Anju Manral, MD, recently was appointed as medical director for the University of New Mexico Student Health and Counseling Center in Albuquerque. The internist has experience as a hospitalist focused on palliative care and most recently has worked at UNM’s Family Health Clinic, providing care to patients of all ages and conditions.
Dr. Manral also serves as an assistant professor in the UNM General Internal Medicine Department and mentors UNM medical students in the health science learning community.
The Hiawatha (Kan.) Community Hospital unveiled its new hospitalist program on Feb. 12.
The program will be led by Dustin Williams, DNP. Dr. Williams will provide hospitalist and emergency medical services to patients every Tuesday through Friday, while an on-call specialist will serve as hospitalist on Saturday, Sunday, and Monday.
Higher AFib ablation volumes linked with better outcomes
SAN FRANCISCO – The number of atrial fibrillation (Afib) catheter ablations a hospital did a year had a substantial, independent effect on patient outcomes in a study of more than 54,000 U.S. catheter ablations performed during 2010-2014.
The results showed that the roughly one-third of studied hospitals with the lowest annual volume of catheter ablations performed, 20 or fewer, had twice the acute complication rate and twice the 30-day in-hospital mortality rate, compared with the hospitals that did 53 or more such procedures annually in patients with atrial fibrillation, Jim W. Cheung, MD, said while presenting a poster at the annual scientific sessions of the Heart Rhythm Society.
The data, taken from 1,738 U.S. hospitals during 2010-2014 and captured in the Nationwide Readmissions Database, also showed that 79% of these hospitals performed 20 or fewer catheter ablations for atrial fibrillation (AFib) annually, with 63% doing 10 or fewer cases per year during the 5 years studied.
The findings raise the question of whether U.S. guidelines for catheter ablation of AFib should specify a minimum case volume for hospital programs, and if so, how high the minimum should be. Volume thresholds are “something to think about,” or a system to designate centers of excellence, Dr. Cheung suggested in a video interview. But interest in setting volume thresholds to better insure competence is often counterbalanced by concerns about patient access, he noted.
The prevailing U.S. guidelines for catheter ablation of AFib are in a 2017 statement from the Heart Rhythm Society and several collaborating groups (J Arrhythm. 2017 Oct;33[5]:369-409). The statement focused on operator volume rather than hospital volume and said that each operator should perform “several” AFib ablation procedures each month, which is generally understood to mean at least 2 per month or at least about 25 annually, commented Hugh Calkins, MD, chair of the panel that wrote the statement and professor of medicine at Johns Hopkins Medicine in Baltimore. The major rationale for setting a suggested minimum of about 25 cases/year came largely from a 2013 report that is cited as the first study to document a volume-outcome relationship for catheter ablation of AFib (Circulation. 2013 Nov 5;128[19]:2104-12), Dr. Calkins noted. “Volume does matter,” he agreed in an interview, but no society or organization monitors hospital or operator volumes, nor takes any steps when volumes are low.
The Nationwide Readmissions Database included 54,599 patients who underwent AFib catheter ablation during 2010-2014. Dr. Cheung and his associates divided these patients into rough tertiles based on the annual procedure volumes of the hospitals that performed these ablations. The 36% of patients treated at hospitals that did 20 or fewer procedures annually were on average older and had more comorbidities than the 31% treated at hospitals in the highest-volume tertile, which performed at least 53 ablations annually. In an analysis that adjusted for these demographic and clinical differences, patients ablated at the lower-volume hospitals had a statistically significant 2.06-fold higher rate of any complication and a 2.24-fold increased rate of in-hospital mortality, either during the index hospitalization or during a 30-day hospital readmission, reported Dr. Cheung, director of clinical electrophysiology research at Weill Cornell Medical College in New York. The increased rate of complications was driven by a fivefold increased rate of cardiac perforations, a greater than doubled periprocedural stroke rate, and a roughly 50% increased rate of vascular complications, compared with the highest-volume hospitals and after adjustment for baseline differences.
Dr. Cheung has been a consultant to Abbott and Biotronik, and has received fellowship support from Biosense Webster, Biotronik, Boston Scientific, Medtronic, and St. Jude. Dr. Calkins disclosed ties to Abbott, Altathera, AtriCure, Boehringer Ingelheim, Boston Scientific, Medtronic, St. Jude, and MRI Interventions.
SOURCE: Cheung JW. HRS 2019, Abstract S-P001-123.
SAN FRANCISCO – The number of atrial fibrillation (Afib) catheter ablations a hospital did a year had a substantial, independent effect on patient outcomes in a study of more than 54,000 U.S. catheter ablations performed during 2010-2014.
The results showed that the roughly one-third of studied hospitals with the lowest annual volume of catheter ablations performed, 20 or fewer, had twice the acute complication rate and twice the 30-day in-hospital mortality rate, compared with the hospitals that did 53 or more such procedures annually in patients with atrial fibrillation, Jim W. Cheung, MD, said while presenting a poster at the annual scientific sessions of the Heart Rhythm Society.
The data, taken from 1,738 U.S. hospitals during 2010-2014 and captured in the Nationwide Readmissions Database, also showed that 79% of these hospitals performed 20 or fewer catheter ablations for atrial fibrillation (AFib) annually, with 63% doing 10 or fewer cases per year during the 5 years studied.
The findings raise the question of whether U.S. guidelines for catheter ablation of AFib should specify a minimum case volume for hospital programs, and if so, how high the minimum should be. Volume thresholds are “something to think about,” or a system to designate centers of excellence, Dr. Cheung suggested in a video interview. But interest in setting volume thresholds to better insure competence is often counterbalanced by concerns about patient access, he noted.
The prevailing U.S. guidelines for catheter ablation of AFib are in a 2017 statement from the Heart Rhythm Society and several collaborating groups (J Arrhythm. 2017 Oct;33[5]:369-409). The statement focused on operator volume rather than hospital volume and said that each operator should perform “several” AFib ablation procedures each month, which is generally understood to mean at least 2 per month or at least about 25 annually, commented Hugh Calkins, MD, chair of the panel that wrote the statement and professor of medicine at Johns Hopkins Medicine in Baltimore. The major rationale for setting a suggested minimum of about 25 cases/year came largely from a 2013 report that is cited as the first study to document a volume-outcome relationship for catheter ablation of AFib (Circulation. 2013 Nov 5;128[19]:2104-12), Dr. Calkins noted. “Volume does matter,” he agreed in an interview, but no society or organization monitors hospital or operator volumes, nor takes any steps when volumes are low.
The Nationwide Readmissions Database included 54,599 patients who underwent AFib catheter ablation during 2010-2014. Dr. Cheung and his associates divided these patients into rough tertiles based on the annual procedure volumes of the hospitals that performed these ablations. The 36% of patients treated at hospitals that did 20 or fewer procedures annually were on average older and had more comorbidities than the 31% treated at hospitals in the highest-volume tertile, which performed at least 53 ablations annually. In an analysis that adjusted for these demographic and clinical differences, patients ablated at the lower-volume hospitals had a statistically significant 2.06-fold higher rate of any complication and a 2.24-fold increased rate of in-hospital mortality, either during the index hospitalization or during a 30-day hospital readmission, reported Dr. Cheung, director of clinical electrophysiology research at Weill Cornell Medical College in New York. The increased rate of complications was driven by a fivefold increased rate of cardiac perforations, a greater than doubled periprocedural stroke rate, and a roughly 50% increased rate of vascular complications, compared with the highest-volume hospitals and after adjustment for baseline differences.
Dr. Cheung has been a consultant to Abbott and Biotronik, and has received fellowship support from Biosense Webster, Biotronik, Boston Scientific, Medtronic, and St. Jude. Dr. Calkins disclosed ties to Abbott, Altathera, AtriCure, Boehringer Ingelheim, Boston Scientific, Medtronic, St. Jude, and MRI Interventions.
SOURCE: Cheung JW. HRS 2019, Abstract S-P001-123.
SAN FRANCISCO – The number of atrial fibrillation (Afib) catheter ablations a hospital did a year had a substantial, independent effect on patient outcomes in a study of more than 54,000 U.S. catheter ablations performed during 2010-2014.
The results showed that the roughly one-third of studied hospitals with the lowest annual volume of catheter ablations performed, 20 or fewer, had twice the acute complication rate and twice the 30-day in-hospital mortality rate, compared with the hospitals that did 53 or more such procedures annually in patients with atrial fibrillation, Jim W. Cheung, MD, said while presenting a poster at the annual scientific sessions of the Heart Rhythm Society.
The data, taken from 1,738 U.S. hospitals during 2010-2014 and captured in the Nationwide Readmissions Database, also showed that 79% of these hospitals performed 20 or fewer catheter ablations for atrial fibrillation (AFib) annually, with 63% doing 10 or fewer cases per year during the 5 years studied.
The findings raise the question of whether U.S. guidelines for catheter ablation of AFib should specify a minimum case volume for hospital programs, and if so, how high the minimum should be. Volume thresholds are “something to think about,” or a system to designate centers of excellence, Dr. Cheung suggested in a video interview. But interest in setting volume thresholds to better insure competence is often counterbalanced by concerns about patient access, he noted.
The prevailing U.S. guidelines for catheter ablation of AFib are in a 2017 statement from the Heart Rhythm Society and several collaborating groups (J Arrhythm. 2017 Oct;33[5]:369-409). The statement focused on operator volume rather than hospital volume and said that each operator should perform “several” AFib ablation procedures each month, which is generally understood to mean at least 2 per month or at least about 25 annually, commented Hugh Calkins, MD, chair of the panel that wrote the statement and professor of medicine at Johns Hopkins Medicine in Baltimore. The major rationale for setting a suggested minimum of about 25 cases/year came largely from a 2013 report that is cited as the first study to document a volume-outcome relationship for catheter ablation of AFib (Circulation. 2013 Nov 5;128[19]:2104-12), Dr. Calkins noted. “Volume does matter,” he agreed in an interview, but no society or organization monitors hospital or operator volumes, nor takes any steps when volumes are low.
The Nationwide Readmissions Database included 54,599 patients who underwent AFib catheter ablation during 2010-2014. Dr. Cheung and his associates divided these patients into rough tertiles based on the annual procedure volumes of the hospitals that performed these ablations. The 36% of patients treated at hospitals that did 20 or fewer procedures annually were on average older and had more comorbidities than the 31% treated at hospitals in the highest-volume tertile, which performed at least 53 ablations annually. In an analysis that adjusted for these demographic and clinical differences, patients ablated at the lower-volume hospitals had a statistically significant 2.06-fold higher rate of any complication and a 2.24-fold increased rate of in-hospital mortality, either during the index hospitalization or during a 30-day hospital readmission, reported Dr. Cheung, director of clinical electrophysiology research at Weill Cornell Medical College in New York. The increased rate of complications was driven by a fivefold increased rate of cardiac perforations, a greater than doubled periprocedural stroke rate, and a roughly 50% increased rate of vascular complications, compared with the highest-volume hospitals and after adjustment for baseline differences.
Dr. Cheung has been a consultant to Abbott and Biotronik, and has received fellowship support from Biosense Webster, Biotronik, Boston Scientific, Medtronic, and St. Jude. Dr. Calkins disclosed ties to Abbott, Altathera, AtriCure, Boehringer Ingelheim, Boston Scientific, Medtronic, St. Jude, and MRI Interventions.
SOURCE: Cheung JW. HRS 2019, Abstract S-P001-123.
REPORTING FROM HEART RHYTHM 2019
Key clinical point: .
Major finding: Hospitals performing 20 or fewer catheter ablations annually had twice as many acute complications as hospitals doing at least 53.
Study details: Analysis of 54,599 atrial fibrillation patients who underwent catheter ablation in the Nationwide Readmissions Database.
Disclosures: Dr. Cheung has been a consultant to Abbott and Biotronik, and has received fellowship support from Biosense Webster, Biotronik, Boston Scientific, Medtronic, and St. Jude.
Source: Cheung JW et al. Heart Rhythm 2019, Abstract S-P001-123.
No exudates or fever? Age over 11? Skip strep test
BALTIMORE – In children with pharyngitis, it’s safe to skip group A Streptococcus testing if there are no exudates, children are 11 years or older, and there is either no cervical adenopathy or adenopathy without fever, according to a Boston Children’s Hospital investigation.
The prevalence of group A Streptococcus among children who meet those criteria is 13%, less than the estimated asymptomatic carriage rate of about 15%. Among 67,127 children tested for strep and treated for sore throats in a network of retail health clinics across the United States, 35% fit the profile.
Investigators led by Daniel Shapiro, MD, a pediatrics fellow at Boston Children’s, concluded that “laboratory testing for GAS [group A Streptococcus] might be safely avoided in a large proportion of patients with sore throats. In doing so, we may avoid some of the downstream effects of unnecessary antibiotic use.” Incorporating the rules into EHRs “might help physicians identify patients who are at low risk of GAS pharyngitis.”
The study team tackled a long-standing and vexing problem in general pediatrics: how to distinguish viral from GAS pharyngitis. They often present the same way, so it’s difficult to tell them apart, but important to do so to prevent misuse of antibiotics. Health care providers generally rely on rapid strep tests and other assays to make the call, but they have to be used cautiously, because asymptomatic carriers also will test positive and be at risk for unnecessary treatment, Dr. Shapiro said at the Pediatric Academic Societies annual meeting.
To try to prevent that, the Infectious Disease Society of America (IDSA) recommends against strep testing in children who present with overt viral signs, including cough, rhinorrhea, oral ulcers, and hoarseness (Clin Infect Dis. 2012 Nov 15;55[10]:1279-82).
In a previous study at Boston Children’s ED, however, Dr. Shapiro and his colleagues found that 29% of children with overt viral features were positive for GAS, suggesting that the IDSA guidelines probably go too far (Pediatrics. 2017 May;139[5]. pii: e20163403).
“One might conclude that while it’s a good rule of thumb to avoid testing patients with viral features, some of the patients with viral features really do have GAS pharyngitis, so the recommendation to forgo testing in all these kids needs a little bit of refinement,” he said.
That was the goal of the new study; the team sought to identify viral features that signaled a low risk of GAS pharyngitis and, therefore, no need for testing. Low risk was defined as less than 15%, in keeping with the asymptomatic carriage rate.
The 67,127 patients were aged 3-21 years. Their signs and symptoms were collected at the retail clinics in a standardized form. The subjects had rapid strep tests, with negative results confirmed by DNA probe or culture.
Fifty-four percent had viral features, defined in the study as cough, runny nose, or hoarseness (oral ulcers weren’t collected on the form). The overall prevalence of GAS was 35%, similar to previous studies; 39% of children with no viral features tested positive for GAS versus 26% of children with all three. Exudates and age below 11 years were strongly associated with GAS among patients with viral features.
It turned out that just 23% of children without exudates were GAS positive; the number fell to 15% when limited to children 11 years or older, and to 13% when either no cervical adenopathy or adenopathy without fever were added to the mix.
There was no industry funding, and Dr. Shapiro didn’t have any disclosures.
BALTIMORE – In children with pharyngitis, it’s safe to skip group A Streptococcus testing if there are no exudates, children are 11 years or older, and there is either no cervical adenopathy or adenopathy without fever, according to a Boston Children’s Hospital investigation.
The prevalence of group A Streptococcus among children who meet those criteria is 13%, less than the estimated asymptomatic carriage rate of about 15%. Among 67,127 children tested for strep and treated for sore throats in a network of retail health clinics across the United States, 35% fit the profile.
Investigators led by Daniel Shapiro, MD, a pediatrics fellow at Boston Children’s, concluded that “laboratory testing for GAS [group A Streptococcus] might be safely avoided in a large proportion of patients with sore throats. In doing so, we may avoid some of the downstream effects of unnecessary antibiotic use.” Incorporating the rules into EHRs “might help physicians identify patients who are at low risk of GAS pharyngitis.”
The study team tackled a long-standing and vexing problem in general pediatrics: how to distinguish viral from GAS pharyngitis. They often present the same way, so it’s difficult to tell them apart, but important to do so to prevent misuse of antibiotics. Health care providers generally rely on rapid strep tests and other assays to make the call, but they have to be used cautiously, because asymptomatic carriers also will test positive and be at risk for unnecessary treatment, Dr. Shapiro said at the Pediatric Academic Societies annual meeting.
To try to prevent that, the Infectious Disease Society of America (IDSA) recommends against strep testing in children who present with overt viral signs, including cough, rhinorrhea, oral ulcers, and hoarseness (Clin Infect Dis. 2012 Nov 15;55[10]:1279-82).
In a previous study at Boston Children’s ED, however, Dr. Shapiro and his colleagues found that 29% of children with overt viral features were positive for GAS, suggesting that the IDSA guidelines probably go too far (Pediatrics. 2017 May;139[5]. pii: e20163403).
“One might conclude that while it’s a good rule of thumb to avoid testing patients with viral features, some of the patients with viral features really do have GAS pharyngitis, so the recommendation to forgo testing in all these kids needs a little bit of refinement,” he said.
That was the goal of the new study; the team sought to identify viral features that signaled a low risk of GAS pharyngitis and, therefore, no need for testing. Low risk was defined as less than 15%, in keeping with the asymptomatic carriage rate.
The 67,127 patients were aged 3-21 years. Their signs and symptoms were collected at the retail clinics in a standardized form. The subjects had rapid strep tests, with negative results confirmed by DNA probe or culture.
Fifty-four percent had viral features, defined in the study as cough, runny nose, or hoarseness (oral ulcers weren’t collected on the form). The overall prevalence of GAS was 35%, similar to previous studies; 39% of children with no viral features tested positive for GAS versus 26% of children with all three. Exudates and age below 11 years were strongly associated with GAS among patients with viral features.
It turned out that just 23% of children without exudates were GAS positive; the number fell to 15% when limited to children 11 years or older, and to 13% when either no cervical adenopathy or adenopathy without fever were added to the mix.
There was no industry funding, and Dr. Shapiro didn’t have any disclosures.
BALTIMORE – In children with pharyngitis, it’s safe to skip group A Streptococcus testing if there are no exudates, children are 11 years or older, and there is either no cervical adenopathy or adenopathy without fever, according to a Boston Children’s Hospital investigation.
The prevalence of group A Streptococcus among children who meet those criteria is 13%, less than the estimated asymptomatic carriage rate of about 15%. Among 67,127 children tested for strep and treated for sore throats in a network of retail health clinics across the United States, 35% fit the profile.
Investigators led by Daniel Shapiro, MD, a pediatrics fellow at Boston Children’s, concluded that “laboratory testing for GAS [group A Streptococcus] might be safely avoided in a large proportion of patients with sore throats. In doing so, we may avoid some of the downstream effects of unnecessary antibiotic use.” Incorporating the rules into EHRs “might help physicians identify patients who are at low risk of GAS pharyngitis.”
The study team tackled a long-standing and vexing problem in general pediatrics: how to distinguish viral from GAS pharyngitis. They often present the same way, so it’s difficult to tell them apart, but important to do so to prevent misuse of antibiotics. Health care providers generally rely on rapid strep tests and other assays to make the call, but they have to be used cautiously, because asymptomatic carriers also will test positive and be at risk for unnecessary treatment, Dr. Shapiro said at the Pediatric Academic Societies annual meeting.
To try to prevent that, the Infectious Disease Society of America (IDSA) recommends against strep testing in children who present with overt viral signs, including cough, rhinorrhea, oral ulcers, and hoarseness (Clin Infect Dis. 2012 Nov 15;55[10]:1279-82).
In a previous study at Boston Children’s ED, however, Dr. Shapiro and his colleagues found that 29% of children with overt viral features were positive for GAS, suggesting that the IDSA guidelines probably go too far (Pediatrics. 2017 May;139[5]. pii: e20163403).
“One might conclude that while it’s a good rule of thumb to avoid testing patients with viral features, some of the patients with viral features really do have GAS pharyngitis, so the recommendation to forgo testing in all these kids needs a little bit of refinement,” he said.
That was the goal of the new study; the team sought to identify viral features that signaled a low risk of GAS pharyngitis and, therefore, no need for testing. Low risk was defined as less than 15%, in keeping with the asymptomatic carriage rate.
The 67,127 patients were aged 3-21 years. Their signs and symptoms were collected at the retail clinics in a standardized form. The subjects had rapid strep tests, with negative results confirmed by DNA probe or culture.
Fifty-four percent had viral features, defined in the study as cough, runny nose, or hoarseness (oral ulcers weren’t collected on the form). The overall prevalence of GAS was 35%, similar to previous studies; 39% of children with no viral features tested positive for GAS versus 26% of children with all three. Exudates and age below 11 years were strongly associated with GAS among patients with viral features.
It turned out that just 23% of children without exudates were GAS positive; the number fell to 15% when limited to children 11 years or older, and to 13% when either no cervical adenopathy or adenopathy without fever were added to the mix.
There was no industry funding, and Dr. Shapiro didn’t have any disclosures.
REPORTING FROM PAS 2019
Rimegepant dissolving tablets treat acute migraine in phase 3 trial
PHILADELPHIA – according to phase 3 trial results presented at the annual meeting of the American Academy of Neurology. The treatment’s efficacy is sustained for 2-48 hours, researchers reported.
Rimegepant is a small molecule calcitonin gene–related peptide receptor antagonist. A 75-mg oral tablet formulation was effective in phase 3 trials. The present study evaluated a novel, orally dissolving tablet formulation that is intended to speed the drug’s onset. The tablet’s time to peak concentration is 1.50 hours, compared with 1.99 hours for the oral tablet.
Formulation preferences
“People with migraine prefer orally dissolving tablets to oral tablets, mainly for their convenience, onset of action, and ability to be taken without drinking liquids,” said first author Richard B. Lipton, MD, of Albert Einstein College of Medicine, New York, and colleagues.
To assess the formulation’s efficacy and safety, the investigators conducted a double-blind, randomized, placebo-controlled, multicenter trial. Participants were aged at least 18 years and had migraine for at least 1 year. They had 2-8 moderate or severe migraine attacks and fewer than 15 headache days per month during the 3 months before the trial. Their preventive migraine medication doses, if any, had been stable for at least 3 months.
Coprimary efficacy endpoints were pain freedom 2 hours post dose and freedom from the most bothersome symptom at 2 hours post dose. The efficacy analyses included randomized subjects who had a qualifying migraine attack, took the study medication, and provided at least one postbaseline efficacy data point.
The investigators included 1,351 patients in their efficacy analysis – 669 who received rimegepant and 682 who received placebo. About 85% were female, and patients’ mean age was 40.2 years. They averaged 4.6 migraine attacks per month, and their most bothersome symptoms included photophobia (57%), nausea (23.5%), and phonophobia (19.3%). About 14% used preventive treatment.
Within 45 days of randomization, patients treated a migraine attack of moderate to severe intensity and completed an electronic diary predose to 48 hours post dose.
Less use of rescue medication
At 2 hours post dose, patients who received 75 mg rimegepant were more likely than patients who received placebo to achieve pain freedom (21.2% vs. 10.9%) and freedom from the most bothersome symptom (35.1% vs. 26.8%).
Numerical differences in the likelihood of pain relief between group began 15 minutes post dose, and the difference was statistically significant at 60 minutes (36.8% vs. 31.2%).
Various secondary endpoints, including ability to function normally at 2 hours post dose (38.1% vs. 25.8%), sustained pain relief from 2-48 hours (42.2% vs. 25.2%), and use of rescue medications within 24 hours (14.2% vs. 29.2%), also were statistically significant.
In the safety analysis, the most common adverse events were nausea (1.6% in the rimegepant group and 0.4% in the placebo group) and urinary tract infection (1.5% in the rimegepant group and 0.6% in the placebo group). There were no serious adverse events. “Safety and tolerability were similar to placebo,” Dr. Lipton and colleagues said.
Biohaven Pharmaceuticals, the developer of the drug, sponsored the study. Dr. Lipton has received honoraria and research support from Biohaven and holds stock in the company. Coauthors are employees of Biohaven.
PHILADELPHIA – according to phase 3 trial results presented at the annual meeting of the American Academy of Neurology. The treatment’s efficacy is sustained for 2-48 hours, researchers reported.
Rimegepant is a small molecule calcitonin gene–related peptide receptor antagonist. A 75-mg oral tablet formulation was effective in phase 3 trials. The present study evaluated a novel, orally dissolving tablet formulation that is intended to speed the drug’s onset. The tablet’s time to peak concentration is 1.50 hours, compared with 1.99 hours for the oral tablet.
Formulation preferences
“People with migraine prefer orally dissolving tablets to oral tablets, mainly for their convenience, onset of action, and ability to be taken without drinking liquids,” said first author Richard B. Lipton, MD, of Albert Einstein College of Medicine, New York, and colleagues.
To assess the formulation’s efficacy and safety, the investigators conducted a double-blind, randomized, placebo-controlled, multicenter trial. Participants were aged at least 18 years and had migraine for at least 1 year. They had 2-8 moderate or severe migraine attacks and fewer than 15 headache days per month during the 3 months before the trial. Their preventive migraine medication doses, if any, had been stable for at least 3 months.
Coprimary efficacy endpoints were pain freedom 2 hours post dose and freedom from the most bothersome symptom at 2 hours post dose. The efficacy analyses included randomized subjects who had a qualifying migraine attack, took the study medication, and provided at least one postbaseline efficacy data point.
The investigators included 1,351 patients in their efficacy analysis – 669 who received rimegepant and 682 who received placebo. About 85% were female, and patients’ mean age was 40.2 years. They averaged 4.6 migraine attacks per month, and their most bothersome symptoms included photophobia (57%), nausea (23.5%), and phonophobia (19.3%). About 14% used preventive treatment.
Within 45 days of randomization, patients treated a migraine attack of moderate to severe intensity and completed an electronic diary predose to 48 hours post dose.
Less use of rescue medication
At 2 hours post dose, patients who received 75 mg rimegepant were more likely than patients who received placebo to achieve pain freedom (21.2% vs. 10.9%) and freedom from the most bothersome symptom (35.1% vs. 26.8%).
Numerical differences in the likelihood of pain relief between group began 15 minutes post dose, and the difference was statistically significant at 60 minutes (36.8% vs. 31.2%).
Various secondary endpoints, including ability to function normally at 2 hours post dose (38.1% vs. 25.8%), sustained pain relief from 2-48 hours (42.2% vs. 25.2%), and use of rescue medications within 24 hours (14.2% vs. 29.2%), also were statistically significant.
In the safety analysis, the most common adverse events were nausea (1.6% in the rimegepant group and 0.4% in the placebo group) and urinary tract infection (1.5% in the rimegepant group and 0.6% in the placebo group). There were no serious adverse events. “Safety and tolerability were similar to placebo,” Dr. Lipton and colleagues said.
Biohaven Pharmaceuticals, the developer of the drug, sponsored the study. Dr. Lipton has received honoraria and research support from Biohaven and holds stock in the company. Coauthors are employees of Biohaven.
PHILADELPHIA – according to phase 3 trial results presented at the annual meeting of the American Academy of Neurology. The treatment’s efficacy is sustained for 2-48 hours, researchers reported.
Rimegepant is a small molecule calcitonin gene–related peptide receptor antagonist. A 75-mg oral tablet formulation was effective in phase 3 trials. The present study evaluated a novel, orally dissolving tablet formulation that is intended to speed the drug’s onset. The tablet’s time to peak concentration is 1.50 hours, compared with 1.99 hours for the oral tablet.
Formulation preferences
“People with migraine prefer orally dissolving tablets to oral tablets, mainly for their convenience, onset of action, and ability to be taken without drinking liquids,” said first author Richard B. Lipton, MD, of Albert Einstein College of Medicine, New York, and colleagues.
To assess the formulation’s efficacy and safety, the investigators conducted a double-blind, randomized, placebo-controlled, multicenter trial. Participants were aged at least 18 years and had migraine for at least 1 year. They had 2-8 moderate or severe migraine attacks and fewer than 15 headache days per month during the 3 months before the trial. Their preventive migraine medication doses, if any, had been stable for at least 3 months.
Coprimary efficacy endpoints were pain freedom 2 hours post dose and freedom from the most bothersome symptom at 2 hours post dose. The efficacy analyses included randomized subjects who had a qualifying migraine attack, took the study medication, and provided at least one postbaseline efficacy data point.
The investigators included 1,351 patients in their efficacy analysis – 669 who received rimegepant and 682 who received placebo. About 85% were female, and patients’ mean age was 40.2 years. They averaged 4.6 migraine attacks per month, and their most bothersome symptoms included photophobia (57%), nausea (23.5%), and phonophobia (19.3%). About 14% used preventive treatment.
Within 45 days of randomization, patients treated a migraine attack of moderate to severe intensity and completed an electronic diary predose to 48 hours post dose.
Less use of rescue medication
At 2 hours post dose, patients who received 75 mg rimegepant were more likely than patients who received placebo to achieve pain freedom (21.2% vs. 10.9%) and freedom from the most bothersome symptom (35.1% vs. 26.8%).
Numerical differences in the likelihood of pain relief between group began 15 minutes post dose, and the difference was statistically significant at 60 minutes (36.8% vs. 31.2%).
Various secondary endpoints, including ability to function normally at 2 hours post dose (38.1% vs. 25.8%), sustained pain relief from 2-48 hours (42.2% vs. 25.2%), and use of rescue medications within 24 hours (14.2% vs. 29.2%), also were statistically significant.
In the safety analysis, the most common adverse events were nausea (1.6% in the rimegepant group and 0.4% in the placebo group) and urinary tract infection (1.5% in the rimegepant group and 0.6% in the placebo group). There were no serious adverse events. “Safety and tolerability were similar to placebo,” Dr. Lipton and colleagues said.
Biohaven Pharmaceuticals, the developer of the drug, sponsored the study. Dr. Lipton has received honoraria and research support from Biohaven and holds stock in the company. Coauthors are employees of Biohaven.
REPORTING FROM AAN 2019
QI boosts adherence to protocol-based care for elevated blood lead levels
according to current guidelines, reported Courtney M. Brown, MD, and associates at Cincinnati Children’s Hospital and the University of Cincinnati.
The study protocol, undertaken at Cincinnati Children’s Hospital Medical Center’s pediatric primary care center, consisted of ordering multivitamins with iron and follow-up lead testing, educating families about identifying and reducing sources of lead exposure, and referring to a specialty environmental health clinic when indicated. Quality improvement and a real-time decision support program called Epic SmartPhrase was used to increase provider adherence. Results from patients aged 9-27 months who were seen at the hospital from February 2016 to June 2018 were included, according to the researchers. Their findings were published in Pediatrics.
Over the study period, 634 elevated blood lead levels (BLLs) were recorded. Between February 2016 – when the protocol was distributed – and May 2017 – when Epic SmartPhrase was introduced – a mean of 5% of cases received protocol-based care. After introduction of Epic Smartphase, the rate of adherence to protocol increased to 90%, which was maintained for the rest of the study.
“A reliable system for responding to BLLs is critical for optimizing outcomes for individuals, as well as activating public health systems to reduce environmental lead sources. Using tools within the EHR, we increased provider adherence with published guidelines. Our Epic SmartPhrase could be easily reproduced by other practices using EHRs. Similar strategies could be applied for standardizing the response to other laboratory tests,” the investigators wrote. “This type of intervention could ensure that screenings of all kinds trigger meaningful interventions.”
The study was supported by the Cincinnati Children’s Hospital Medical Center through the All Children Thrive community health initiative; the study authors had no relevant financial disclosures.
SOURCE: Brown CM et al. Pediatrics. 2019 May 9. doi: 10.1542/peds.2018-3085.
according to current guidelines, reported Courtney M. Brown, MD, and associates at Cincinnati Children’s Hospital and the University of Cincinnati.
The study protocol, undertaken at Cincinnati Children’s Hospital Medical Center’s pediatric primary care center, consisted of ordering multivitamins with iron and follow-up lead testing, educating families about identifying and reducing sources of lead exposure, and referring to a specialty environmental health clinic when indicated. Quality improvement and a real-time decision support program called Epic SmartPhrase was used to increase provider adherence. Results from patients aged 9-27 months who were seen at the hospital from February 2016 to June 2018 were included, according to the researchers. Their findings were published in Pediatrics.
Over the study period, 634 elevated blood lead levels (BLLs) were recorded. Between February 2016 – when the protocol was distributed – and May 2017 – when Epic SmartPhrase was introduced – a mean of 5% of cases received protocol-based care. After introduction of Epic Smartphase, the rate of adherence to protocol increased to 90%, which was maintained for the rest of the study.
“A reliable system for responding to BLLs is critical for optimizing outcomes for individuals, as well as activating public health systems to reduce environmental lead sources. Using tools within the EHR, we increased provider adherence with published guidelines. Our Epic SmartPhrase could be easily reproduced by other practices using EHRs. Similar strategies could be applied for standardizing the response to other laboratory tests,” the investigators wrote. “This type of intervention could ensure that screenings of all kinds trigger meaningful interventions.”
The study was supported by the Cincinnati Children’s Hospital Medical Center through the All Children Thrive community health initiative; the study authors had no relevant financial disclosures.
SOURCE: Brown CM et al. Pediatrics. 2019 May 9. doi: 10.1542/peds.2018-3085.
according to current guidelines, reported Courtney M. Brown, MD, and associates at Cincinnati Children’s Hospital and the University of Cincinnati.
The study protocol, undertaken at Cincinnati Children’s Hospital Medical Center’s pediatric primary care center, consisted of ordering multivitamins with iron and follow-up lead testing, educating families about identifying and reducing sources of lead exposure, and referring to a specialty environmental health clinic when indicated. Quality improvement and a real-time decision support program called Epic SmartPhrase was used to increase provider adherence. Results from patients aged 9-27 months who were seen at the hospital from February 2016 to June 2018 were included, according to the researchers. Their findings were published in Pediatrics.
Over the study period, 634 elevated blood lead levels (BLLs) were recorded. Between February 2016 – when the protocol was distributed – and May 2017 – when Epic SmartPhrase was introduced – a mean of 5% of cases received protocol-based care. After introduction of Epic Smartphase, the rate of adherence to protocol increased to 90%, which was maintained for the rest of the study.
“A reliable system for responding to BLLs is critical for optimizing outcomes for individuals, as well as activating public health systems to reduce environmental lead sources. Using tools within the EHR, we increased provider adherence with published guidelines. Our Epic SmartPhrase could be easily reproduced by other practices using EHRs. Similar strategies could be applied for standardizing the response to other laboratory tests,” the investigators wrote. “This type of intervention could ensure that screenings of all kinds trigger meaningful interventions.”
The study was supported by the Cincinnati Children’s Hospital Medical Center through the All Children Thrive community health initiative; the study authors had no relevant financial disclosures.
SOURCE: Brown CM et al. Pediatrics. 2019 May 9. doi: 10.1542/peds.2018-3085.
FROM PEDIATRICS
Study finds lower quality of life for patients with hemophilia A
Real‐world data suggests that patients with hemophilia A with inhibitors had lower health-related quality of life (HRQOL) while receiving standard therapy, according to an international study.
“The objective of this analysis was to characterize disease‐specific HRQOL, overall health status and the effect of bleeding on health status,” wrote Johnny Mahlangu, MD, of the University of the Witwatersrand in Johannesburg, South Africa, and colleagues. The study was published in Haemophilia.
The researchers conducted a prospective, noninterventional study of 103 patients aged 12 years and older with hemophilia A who resided in several different countries, including Australia, Japan, South Africa, and the United States, among others.
The majority of participants (n = 75) received episodic treatment at study enrollment, while others (n = 28) received prophylactic-based therapy. Patients were treated with standard therapy, based on local institutional practice.
HRQOL outcome data were collected in adult and adolescent participants using the Haemophilia Quality of Life Questionnaire for Adults and the Haemophilia‐specific Quality of Life Questionnaire for Children Short Form. Other validated instruments were used to measure additional health‐related outcomes.
After analysis, the researchers found that HRQOL scores revealed impaired quality of life in adult and adolescent participants treated with both episodic and prophylactic regimens. The mean scores in the majority of HRQOL domains showed impairments occurring on average “sometimes” to “often,” the researchers reported.
Adults had highest scores, correlated with greatest impairments, in sports and leisure. Similarly, adolescents reported greatest impairment in the sports and school domain.
“These health‐related outcomes may result from a combination of poor bleed control and treatment burden,” the researchers wrote. “Compliance with prophylactic treatment was low, likely reflecting the high burden associated with standard therapies.”
The researchers acknowledged a key limitation of the study was participant dropout. As a result, some time-related data may be incomplete.
“These [data] demonstrate that patients with hemophilia A with inhibitors have impaired HRQOL, despite standard treatment, and that more effective treatment options are needed,” the researchers concluded.
The study was funded by F. Hoffmann-La Roche. The authors reported financial affiliations with Baxalta, Bayer, CSL Behring, Kaketsuken, Novo Nordisk, Pfizer, and several others.
SOURCE: Mahlangu J et al. Haemophilia. 2019 Apr 24. doi: 10.1111/hae.13731.
Real‐world data suggests that patients with hemophilia A with inhibitors had lower health-related quality of life (HRQOL) while receiving standard therapy, according to an international study.
“The objective of this analysis was to characterize disease‐specific HRQOL, overall health status and the effect of bleeding on health status,” wrote Johnny Mahlangu, MD, of the University of the Witwatersrand in Johannesburg, South Africa, and colleagues. The study was published in Haemophilia.
The researchers conducted a prospective, noninterventional study of 103 patients aged 12 years and older with hemophilia A who resided in several different countries, including Australia, Japan, South Africa, and the United States, among others.
The majority of participants (n = 75) received episodic treatment at study enrollment, while others (n = 28) received prophylactic-based therapy. Patients were treated with standard therapy, based on local institutional practice.
HRQOL outcome data were collected in adult and adolescent participants using the Haemophilia Quality of Life Questionnaire for Adults and the Haemophilia‐specific Quality of Life Questionnaire for Children Short Form. Other validated instruments were used to measure additional health‐related outcomes.
After analysis, the researchers found that HRQOL scores revealed impaired quality of life in adult and adolescent participants treated with both episodic and prophylactic regimens. The mean scores in the majority of HRQOL domains showed impairments occurring on average “sometimes” to “often,” the researchers reported.
Adults had highest scores, correlated with greatest impairments, in sports and leisure. Similarly, adolescents reported greatest impairment in the sports and school domain.
“These health‐related outcomes may result from a combination of poor bleed control and treatment burden,” the researchers wrote. “Compliance with prophylactic treatment was low, likely reflecting the high burden associated with standard therapies.”
The researchers acknowledged a key limitation of the study was participant dropout. As a result, some time-related data may be incomplete.
“These [data] demonstrate that patients with hemophilia A with inhibitors have impaired HRQOL, despite standard treatment, and that more effective treatment options are needed,” the researchers concluded.
The study was funded by F. Hoffmann-La Roche. The authors reported financial affiliations with Baxalta, Bayer, CSL Behring, Kaketsuken, Novo Nordisk, Pfizer, and several others.
SOURCE: Mahlangu J et al. Haemophilia. 2019 Apr 24. doi: 10.1111/hae.13731.
Real‐world data suggests that patients with hemophilia A with inhibitors had lower health-related quality of life (HRQOL) while receiving standard therapy, according to an international study.
“The objective of this analysis was to characterize disease‐specific HRQOL, overall health status and the effect of bleeding on health status,” wrote Johnny Mahlangu, MD, of the University of the Witwatersrand in Johannesburg, South Africa, and colleagues. The study was published in Haemophilia.
The researchers conducted a prospective, noninterventional study of 103 patients aged 12 years and older with hemophilia A who resided in several different countries, including Australia, Japan, South Africa, and the United States, among others.
The majority of participants (n = 75) received episodic treatment at study enrollment, while others (n = 28) received prophylactic-based therapy. Patients were treated with standard therapy, based on local institutional practice.
HRQOL outcome data were collected in adult and adolescent participants using the Haemophilia Quality of Life Questionnaire for Adults and the Haemophilia‐specific Quality of Life Questionnaire for Children Short Form. Other validated instruments were used to measure additional health‐related outcomes.
After analysis, the researchers found that HRQOL scores revealed impaired quality of life in adult and adolescent participants treated with both episodic and prophylactic regimens. The mean scores in the majority of HRQOL domains showed impairments occurring on average “sometimes” to “often,” the researchers reported.
Adults had highest scores, correlated with greatest impairments, in sports and leisure. Similarly, adolescents reported greatest impairment in the sports and school domain.
“These health‐related outcomes may result from a combination of poor bleed control and treatment burden,” the researchers wrote. “Compliance with prophylactic treatment was low, likely reflecting the high burden associated with standard therapies.”
The researchers acknowledged a key limitation of the study was participant dropout. As a result, some time-related data may be incomplete.
“These [data] demonstrate that patients with hemophilia A with inhibitors have impaired HRQOL, despite standard treatment, and that more effective treatment options are needed,” the researchers concluded.
The study was funded by F. Hoffmann-La Roche. The authors reported financial affiliations with Baxalta, Bayer, CSL Behring, Kaketsuken, Novo Nordisk, Pfizer, and several others.
SOURCE: Mahlangu J et al. Haemophilia. 2019 Apr 24. doi: 10.1111/hae.13731.
FROM HAEMOPHILIA
Unrecognized focal stenosis after angiographically successful PCI
Moreover, in 82% of DEFINE PCI participants with post-PCI residual ischemia as defined by instantaneous wave-free ratio (iFR) with pullback evaluation of the whole coronary vessel, the impaired physiology was due to an angiographically unrecognized focal stenosis that’s usually potentially treatable, Allen Jeremias, MD, observed in presenting the DEFINE PCI results at the annual meeting of the American College of Cardiology.
“We estimated that if all residual focal lesions could be treated with additional PCI, the rate of significant ischemia could theoretically be reduced from 24% to 5%, said Dr. Jeremias, director of the physiology core laboratory at the Cardiovascular Research Foundation in New York.
Post-PCI ischemia has been associated with recurrent angina and repeat PCI. The 24% prevalence of residual impaired physiology and ischemia despite a successful angiographic result may seem startlingly high to some, but it really shouldn’t be, according to Dr. Jeremias, who is also director of interventional cardiology research and associate director of the cardiac catheterization laboratory at St. Francis Hospital in Roslyn, N.Y.
“There are a lot of physiologic studies looking at FFR [fractional flow reserve] before PCI to determine if we should do it. And we learned how unreliable the angiogram is to make those decisions. So I think obviously we shouldn’t be surprised if the angiogram afterwards is just as unreliable,” he said.
DEFINE PCI was a prospective observational study of 500 patients who underwent PCI for stable or unstable angina at 27 U.S. and European sites. An iFR was done prior to PCI in all vessels with an angiographic lesion severity of 40% or more. Participating interventionalists performed angiographically guided PCI and confirmed their procedural success with post-PCI angiography before the patient left the cath lab. They also performed an iFR manual pullback interrogation of the entire treated vessel. Although the iFR data are linked to the angiographic images via a technology known as co-registration, the operators were blinded to the iFR results, which along with the angiograms were interpreted in a core laboratory. All patients received guideline-directed medical therapy.
The iFR improved on average from 0.69 pre-PCI to 0.93 post treatment. To put that in perspective, an iFR value of 0.89 or less defines hemodynamically significant ischemia.
Residual physiologic impairment post PCI was deemed due to a missed focal stenosis in 82% of cases and to diffuse atherosclerotic disease in the other 18%. Untreated focal lesions were located within the stent in 38% of cases, proximally in 31%, and distal to the stent in the remainder.
Dr. Jeremias said the investigators looked in vain for possible predictors of post-PCI residual impaired physiology. Post-PCI angiographic results were poorly correlated with iFR. For example, 30% of patients with a residual diameter stenosis of 50% or more had a post-PCI iFR of 0.89 or less, as did 21% of those with a residual diameter stenosis of less than 50%, a nonsignificant difference. Moreover, there were no procedural predictors of poor physiologic outcome.
“I don’t think that the answer is more angiograms or procedural changes guided by the angiogram, but rather guiding of the procedure with physiology and also intravascular imaging,” the cardiologist said.
Session cochair J. Dawn Abbott, MD, an interventional cardiologist at Brown University in Providence, R.I., said DEFINE PCI “really brings up the importance of co-registration of iFR, be it with optical coherence tomography or angiography, because we need the information together. If we can’t see these lesions on the angiogram, we need to be doing more complicated combined physiology and anatomy.”
“This is a very interesting study that I think generates a lot of provocative information,” said discussant John J. Warner, MD.
“It certainly challenges, once again, our definition of angiographic success.” The key remaining question is whether additional PCI addressing the residual focal stenoses causing ischemia will result in improved clinical outcomes, added Dr. Warner, an interventional cardiologist and CEO of the University of Texas Southwestern Medical Center Hospitals, Dallas.
Dr. Jeremias noted that DEFINE PCI participants are in the process of being followed through 12 months to see the impact of residual ischemia on recurrent angina, major adverse cardiovascular events, and quality of life. Moreover, a large randomized trial known as DEFINE GPS (Guided Physiologic Stenting) will soon get underway. Participants will be randomized to unblinded iFR-guided therapy with pullback in order to optimize the physiologic result or to conventional angiographically guided PCI. This trial will define the clinical value of PCI with iFR pullback and should answer the question of whether the more important iFR number is the magnitude of the iFR gain achieved via revascularization or the absolute iFR number achieved at the end.
DEFINE PCI and DEFINE GPS are funded by Volcano/Philips. Dr. Jeremias reported serving as a consultant to that company and a handful of others.
SOURCE: Jeremias A. ACC 19 Abstract 408-10.
Moreover, in 82% of DEFINE PCI participants with post-PCI residual ischemia as defined by instantaneous wave-free ratio (iFR) with pullback evaluation of the whole coronary vessel, the impaired physiology was due to an angiographically unrecognized focal stenosis that’s usually potentially treatable, Allen Jeremias, MD, observed in presenting the DEFINE PCI results at the annual meeting of the American College of Cardiology.
“We estimated that if all residual focal lesions could be treated with additional PCI, the rate of significant ischemia could theoretically be reduced from 24% to 5%, said Dr. Jeremias, director of the physiology core laboratory at the Cardiovascular Research Foundation in New York.
Post-PCI ischemia has been associated with recurrent angina and repeat PCI. The 24% prevalence of residual impaired physiology and ischemia despite a successful angiographic result may seem startlingly high to some, but it really shouldn’t be, according to Dr. Jeremias, who is also director of interventional cardiology research and associate director of the cardiac catheterization laboratory at St. Francis Hospital in Roslyn, N.Y.
“There are a lot of physiologic studies looking at FFR [fractional flow reserve] before PCI to determine if we should do it. And we learned how unreliable the angiogram is to make those decisions. So I think obviously we shouldn’t be surprised if the angiogram afterwards is just as unreliable,” he said.
DEFINE PCI was a prospective observational study of 500 patients who underwent PCI for stable or unstable angina at 27 U.S. and European sites. An iFR was done prior to PCI in all vessels with an angiographic lesion severity of 40% or more. Participating interventionalists performed angiographically guided PCI and confirmed their procedural success with post-PCI angiography before the patient left the cath lab. They also performed an iFR manual pullback interrogation of the entire treated vessel. Although the iFR data are linked to the angiographic images via a technology known as co-registration, the operators were blinded to the iFR results, which along with the angiograms were interpreted in a core laboratory. All patients received guideline-directed medical therapy.
The iFR improved on average from 0.69 pre-PCI to 0.93 post treatment. To put that in perspective, an iFR value of 0.89 or less defines hemodynamically significant ischemia.
Residual physiologic impairment post PCI was deemed due to a missed focal stenosis in 82% of cases and to diffuse atherosclerotic disease in the other 18%. Untreated focal lesions were located within the stent in 38% of cases, proximally in 31%, and distal to the stent in the remainder.
Dr. Jeremias said the investigators looked in vain for possible predictors of post-PCI residual impaired physiology. Post-PCI angiographic results were poorly correlated with iFR. For example, 30% of patients with a residual diameter stenosis of 50% or more had a post-PCI iFR of 0.89 or less, as did 21% of those with a residual diameter stenosis of less than 50%, a nonsignificant difference. Moreover, there were no procedural predictors of poor physiologic outcome.
“I don’t think that the answer is more angiograms or procedural changes guided by the angiogram, but rather guiding of the procedure with physiology and also intravascular imaging,” the cardiologist said.
Session cochair J. Dawn Abbott, MD, an interventional cardiologist at Brown University in Providence, R.I., said DEFINE PCI “really brings up the importance of co-registration of iFR, be it with optical coherence tomography or angiography, because we need the information together. If we can’t see these lesions on the angiogram, we need to be doing more complicated combined physiology and anatomy.”
“This is a very interesting study that I think generates a lot of provocative information,” said discussant John J. Warner, MD.
“It certainly challenges, once again, our definition of angiographic success.” The key remaining question is whether additional PCI addressing the residual focal stenoses causing ischemia will result in improved clinical outcomes, added Dr. Warner, an interventional cardiologist and CEO of the University of Texas Southwestern Medical Center Hospitals, Dallas.
Dr. Jeremias noted that DEFINE PCI participants are in the process of being followed through 12 months to see the impact of residual ischemia on recurrent angina, major adverse cardiovascular events, and quality of life. Moreover, a large randomized trial known as DEFINE GPS (Guided Physiologic Stenting) will soon get underway. Participants will be randomized to unblinded iFR-guided therapy with pullback in order to optimize the physiologic result or to conventional angiographically guided PCI. This trial will define the clinical value of PCI with iFR pullback and should answer the question of whether the more important iFR number is the magnitude of the iFR gain achieved via revascularization or the absolute iFR number achieved at the end.
DEFINE PCI and DEFINE GPS are funded by Volcano/Philips. Dr. Jeremias reported serving as a consultant to that company and a handful of others.
SOURCE: Jeremias A. ACC 19 Abstract 408-10.
Moreover, in 82% of DEFINE PCI participants with post-PCI residual ischemia as defined by instantaneous wave-free ratio (iFR) with pullback evaluation of the whole coronary vessel, the impaired physiology was due to an angiographically unrecognized focal stenosis that’s usually potentially treatable, Allen Jeremias, MD, observed in presenting the DEFINE PCI results at the annual meeting of the American College of Cardiology.
“We estimated that if all residual focal lesions could be treated with additional PCI, the rate of significant ischemia could theoretically be reduced from 24% to 5%, said Dr. Jeremias, director of the physiology core laboratory at the Cardiovascular Research Foundation in New York.
Post-PCI ischemia has been associated with recurrent angina and repeat PCI. The 24% prevalence of residual impaired physiology and ischemia despite a successful angiographic result may seem startlingly high to some, but it really shouldn’t be, according to Dr. Jeremias, who is also director of interventional cardiology research and associate director of the cardiac catheterization laboratory at St. Francis Hospital in Roslyn, N.Y.
“There are a lot of physiologic studies looking at FFR [fractional flow reserve] before PCI to determine if we should do it. And we learned how unreliable the angiogram is to make those decisions. So I think obviously we shouldn’t be surprised if the angiogram afterwards is just as unreliable,” he said.
DEFINE PCI was a prospective observational study of 500 patients who underwent PCI for stable or unstable angina at 27 U.S. and European sites. An iFR was done prior to PCI in all vessels with an angiographic lesion severity of 40% or more. Participating interventionalists performed angiographically guided PCI and confirmed their procedural success with post-PCI angiography before the patient left the cath lab. They also performed an iFR manual pullback interrogation of the entire treated vessel. Although the iFR data are linked to the angiographic images via a technology known as co-registration, the operators were blinded to the iFR results, which along with the angiograms were interpreted in a core laboratory. All patients received guideline-directed medical therapy.
The iFR improved on average from 0.69 pre-PCI to 0.93 post treatment. To put that in perspective, an iFR value of 0.89 or less defines hemodynamically significant ischemia.
Residual physiologic impairment post PCI was deemed due to a missed focal stenosis in 82% of cases and to diffuse atherosclerotic disease in the other 18%. Untreated focal lesions were located within the stent in 38% of cases, proximally in 31%, and distal to the stent in the remainder.
Dr. Jeremias said the investigators looked in vain for possible predictors of post-PCI residual impaired physiology. Post-PCI angiographic results were poorly correlated with iFR. For example, 30% of patients with a residual diameter stenosis of 50% or more had a post-PCI iFR of 0.89 or less, as did 21% of those with a residual diameter stenosis of less than 50%, a nonsignificant difference. Moreover, there were no procedural predictors of poor physiologic outcome.
“I don’t think that the answer is more angiograms or procedural changes guided by the angiogram, but rather guiding of the procedure with physiology and also intravascular imaging,” the cardiologist said.
Session cochair J. Dawn Abbott, MD, an interventional cardiologist at Brown University in Providence, R.I., said DEFINE PCI “really brings up the importance of co-registration of iFR, be it with optical coherence tomography or angiography, because we need the information together. If we can’t see these lesions on the angiogram, we need to be doing more complicated combined physiology and anatomy.”
“This is a very interesting study that I think generates a lot of provocative information,” said discussant John J. Warner, MD.
“It certainly challenges, once again, our definition of angiographic success.” The key remaining question is whether additional PCI addressing the residual focal stenoses causing ischemia will result in improved clinical outcomes, added Dr. Warner, an interventional cardiologist and CEO of the University of Texas Southwestern Medical Center Hospitals, Dallas.
Dr. Jeremias noted that DEFINE PCI participants are in the process of being followed through 12 months to see the impact of residual ischemia on recurrent angina, major adverse cardiovascular events, and quality of life. Moreover, a large randomized trial known as DEFINE GPS (Guided Physiologic Stenting) will soon get underway. Participants will be randomized to unblinded iFR-guided therapy with pullback in order to optimize the physiologic result or to conventional angiographically guided PCI. This trial will define the clinical value of PCI with iFR pullback and should answer the question of whether the more important iFR number is the magnitude of the iFR gain achieved via revascularization or the absolute iFR number achieved at the end.
DEFINE PCI and DEFINE GPS are funded by Volcano/Philips. Dr. Jeremias reported serving as a consultant to that company and a handful of others.
SOURCE: Jeremias A. ACC 19 Abstract 408-10.
REPORTING FROM ACC 19
Can an antisense oligonucleotide benefit patients with SOD1-ALS?
PHILADELPHIA – In patients with amyotrophic lateral sclerosis (ALS) caused by gain-of-toxic function mutations in the SOD1 gene, according to phase 1/2 trial results presented at the annual meeting of the American Academy of Neurology. Exploratory analyses suggest that the drug, known as tofersen (also known as BIIB067), may lessen declines in function, respiratory function, and strength. The treatment generally was safe and well tolerated, researchers said.
Most ALS cases are sporadic, but about 10% are genetic, of which approximately 20% are caused by SOD1 mutations. “Although SOD1-ALS disease progression is heterogeneous, the underlying pathophysiology, attributable to mutant SOD1 toxicity, is thought to be consistent across SOD1 mutation types,” said Timothy M. Miller, MD, PhD, of Washington University, St. Louis, and colleagues. “As such, effective reduction of SOD1 protein, irrespective of mutation, has the potential to alter the disease course of people with SOD1-ALS.”
Tofersen is an antisense oligonucleotide ribonuclease H1-mediated inhibitor of SOD1 messenger RNA. To study its safety, tolerability, pharmacokinetics, pharmacodynamics, and exploratory efficacy in patients with SOD1-ALS, investigators conducted a double-blind study. The investigators randomized 50 patients with ALS with a SOD1 mutation to 20 mg (n = 10), 40 mg (n = 9), 60 mg (n = 9), or 100 mg (n = 10) of tofersen or placebo (n = 12).
Patients received tofersen by intrathecal bolus over 1-3 minutes. They received a loading regimen on days 1, 15, and 29 and maintenance dosing on days 57 and 85. After the dosing period, patients completed a 12-week follow-up period.
Adverse events
All patients received at least one dose of the study treatment, and 48 received all treatments. Three patients died during the study. One patient in the 20-mg group died of pulmonary embolism, and one patient in both the 60-mg group and placebo group died of respiratory failure. Investigators considered the deaths secondary to ALS or other conditions and not drug related.
Most adverse events were mild or moderate. The most common adverse events among tofersen-treated patients were headache (n = 16), procedural pain (n = 14), and post–lumbar puncture syndrome (n = 13). Five patients who received tofersen and two who received placebo experienced serious adverse events; no serious adverse events occurred in the 100-mg dose group.
“A reduction from baseline in CSF SOD1 concentrations was observed in the tofersen 40-, 60-, and 100-mg cohorts with the maximal reduction observed in the 100 mg–treated group [37% vs. no reduction in the placebo group; P less than 0.002] at day 85,” the investigators reported.
Possible efficacy
In exploratory analyses, the 100-mg dose slowed decline on the Amyotrophic Lateral Sclerosis Functional Rating Scale–Revised (ASLFRS-R), compared with placebo. Mean change in ASLFRS-R from baseline to day 85 was –1.1 in patients who received 100 mg of tofersen, compared with –5.3 for patients who received placebo. Declines on measures of respiratory function and muscle strength also slowed. “Across clinical measures, separation from placebo was most apparent in participants with fast progressing disease,” the researchers said.
“Lower concentrations of the protein in the spinal fluid suggest that there were also lower concentrations in the brain and spinal cord,” Dr. Miller said in a news release. “Such reductions could lead to preservation of motor neurons and slow progression of the disease, but more study is needed to examine this further.”
The study was sponsored by Biogen, which is developing tofersen. Some of the study authors are Biogen employees. Dr. Miller is on Biogen’s medical advisory board and receives clinical research support from Biogen. In addition, he consults, has licensing agreements with, and is a principal investigator for other companies.
PHILADELPHIA – In patients with amyotrophic lateral sclerosis (ALS) caused by gain-of-toxic function mutations in the SOD1 gene, according to phase 1/2 trial results presented at the annual meeting of the American Academy of Neurology. Exploratory analyses suggest that the drug, known as tofersen (also known as BIIB067), may lessen declines in function, respiratory function, and strength. The treatment generally was safe and well tolerated, researchers said.
Most ALS cases are sporadic, but about 10% are genetic, of which approximately 20% are caused by SOD1 mutations. “Although SOD1-ALS disease progression is heterogeneous, the underlying pathophysiology, attributable to mutant SOD1 toxicity, is thought to be consistent across SOD1 mutation types,” said Timothy M. Miller, MD, PhD, of Washington University, St. Louis, and colleagues. “As such, effective reduction of SOD1 protein, irrespective of mutation, has the potential to alter the disease course of people with SOD1-ALS.”
Tofersen is an antisense oligonucleotide ribonuclease H1-mediated inhibitor of SOD1 messenger RNA. To study its safety, tolerability, pharmacokinetics, pharmacodynamics, and exploratory efficacy in patients with SOD1-ALS, investigators conducted a double-blind study. The investigators randomized 50 patients with ALS with a SOD1 mutation to 20 mg (n = 10), 40 mg (n = 9), 60 mg (n = 9), or 100 mg (n = 10) of tofersen or placebo (n = 12).
Patients received tofersen by intrathecal bolus over 1-3 minutes. They received a loading regimen on days 1, 15, and 29 and maintenance dosing on days 57 and 85. After the dosing period, patients completed a 12-week follow-up period.
Adverse events
All patients received at least one dose of the study treatment, and 48 received all treatments. Three patients died during the study. One patient in the 20-mg group died of pulmonary embolism, and one patient in both the 60-mg group and placebo group died of respiratory failure. Investigators considered the deaths secondary to ALS or other conditions and not drug related.
Most adverse events were mild or moderate. The most common adverse events among tofersen-treated patients were headache (n = 16), procedural pain (n = 14), and post–lumbar puncture syndrome (n = 13). Five patients who received tofersen and two who received placebo experienced serious adverse events; no serious adverse events occurred in the 100-mg dose group.
“A reduction from baseline in CSF SOD1 concentrations was observed in the tofersen 40-, 60-, and 100-mg cohorts with the maximal reduction observed in the 100 mg–treated group [37% vs. no reduction in the placebo group; P less than 0.002] at day 85,” the investigators reported.
Possible efficacy
In exploratory analyses, the 100-mg dose slowed decline on the Amyotrophic Lateral Sclerosis Functional Rating Scale–Revised (ASLFRS-R), compared with placebo. Mean change in ASLFRS-R from baseline to day 85 was –1.1 in patients who received 100 mg of tofersen, compared with –5.3 for patients who received placebo. Declines on measures of respiratory function and muscle strength also slowed. “Across clinical measures, separation from placebo was most apparent in participants with fast progressing disease,” the researchers said.
“Lower concentrations of the protein in the spinal fluid suggest that there were also lower concentrations in the brain and spinal cord,” Dr. Miller said in a news release. “Such reductions could lead to preservation of motor neurons and slow progression of the disease, but more study is needed to examine this further.”
The study was sponsored by Biogen, which is developing tofersen. Some of the study authors are Biogen employees. Dr. Miller is on Biogen’s medical advisory board and receives clinical research support from Biogen. In addition, he consults, has licensing agreements with, and is a principal investigator for other companies.
PHILADELPHIA – In patients with amyotrophic lateral sclerosis (ALS) caused by gain-of-toxic function mutations in the SOD1 gene, according to phase 1/2 trial results presented at the annual meeting of the American Academy of Neurology. Exploratory analyses suggest that the drug, known as tofersen (also known as BIIB067), may lessen declines in function, respiratory function, and strength. The treatment generally was safe and well tolerated, researchers said.
Most ALS cases are sporadic, but about 10% are genetic, of which approximately 20% are caused by SOD1 mutations. “Although SOD1-ALS disease progression is heterogeneous, the underlying pathophysiology, attributable to mutant SOD1 toxicity, is thought to be consistent across SOD1 mutation types,” said Timothy M. Miller, MD, PhD, of Washington University, St. Louis, and colleagues. “As such, effective reduction of SOD1 protein, irrespective of mutation, has the potential to alter the disease course of people with SOD1-ALS.”
Tofersen is an antisense oligonucleotide ribonuclease H1-mediated inhibitor of SOD1 messenger RNA. To study its safety, tolerability, pharmacokinetics, pharmacodynamics, and exploratory efficacy in patients with SOD1-ALS, investigators conducted a double-blind study. The investigators randomized 50 patients with ALS with a SOD1 mutation to 20 mg (n = 10), 40 mg (n = 9), 60 mg (n = 9), or 100 mg (n = 10) of tofersen or placebo (n = 12).
Patients received tofersen by intrathecal bolus over 1-3 minutes. They received a loading regimen on days 1, 15, and 29 and maintenance dosing on days 57 and 85. After the dosing period, patients completed a 12-week follow-up period.
Adverse events
All patients received at least one dose of the study treatment, and 48 received all treatments. Three patients died during the study. One patient in the 20-mg group died of pulmonary embolism, and one patient in both the 60-mg group and placebo group died of respiratory failure. Investigators considered the deaths secondary to ALS or other conditions and not drug related.
Most adverse events were mild or moderate. The most common adverse events among tofersen-treated patients were headache (n = 16), procedural pain (n = 14), and post–lumbar puncture syndrome (n = 13). Five patients who received tofersen and two who received placebo experienced serious adverse events; no serious adverse events occurred in the 100-mg dose group.
“A reduction from baseline in CSF SOD1 concentrations was observed in the tofersen 40-, 60-, and 100-mg cohorts with the maximal reduction observed in the 100 mg–treated group [37% vs. no reduction in the placebo group; P less than 0.002] at day 85,” the investigators reported.
Possible efficacy
In exploratory analyses, the 100-mg dose slowed decline on the Amyotrophic Lateral Sclerosis Functional Rating Scale–Revised (ASLFRS-R), compared with placebo. Mean change in ASLFRS-R from baseline to day 85 was –1.1 in patients who received 100 mg of tofersen, compared with –5.3 for patients who received placebo. Declines on measures of respiratory function and muscle strength also slowed. “Across clinical measures, separation from placebo was most apparent in participants with fast progressing disease,” the researchers said.
“Lower concentrations of the protein in the spinal fluid suggest that there were also lower concentrations in the brain and spinal cord,” Dr. Miller said in a news release. “Such reductions could lead to preservation of motor neurons and slow progression of the disease, but more study is needed to examine this further.”
The study was sponsored by Biogen, which is developing tofersen. Some of the study authors are Biogen employees. Dr. Miller is on Biogen’s medical advisory board and receives clinical research support from Biogen. In addition, he consults, has licensing agreements with, and is a principal investigator for other companies.
REPORTING FROM AAN 2019
Experts discuss what’s new in migraine treatment
PHILADELPHIA – At the annual meeting of the American Academy of Neurology, Alan M. Rapoport, MD, clinical professor of neurology at the University of California, Los Angeles, sat down with Stewart J. Tepper, MD, professor of neurology at the Geisel School of Medicine at Dartmouth, Hanover, N.H., to discuss in a video some of the new data presented at the meeting regarding the CGRP monoclonal antibodies, the small molecule receptor antagonists (gepants), and what Dr. Tepper described as “a real shift in paradigm and a watershed moment in migraine.”
The three gepants that are farthest along in clinical trials are ubrogepant, rimegepant, and atogepant. “Reassuring data” was presented, Dr. Tepper said, regarding liver toxicity, which has been a concern with earlier generations of the gepants. The Food and Drug Administration had mandated a close look at liver function with the use of these drugs, which are metabolized in the liver, and, to date, no safety signals have emerged.
The three CGRP monoclonal antibodies that are currently on the market are erenumab (Aimovig), fremanezumab (Ajovy), and galcanezumab (Emgality). Data from numerous open-label extension studies were presented. In general, it seems that “the monoclonal antibodies accumulate greater efficacy over time,” Dr. Tepper said. No safety concerns have emerged from 5 years of clinical trial data. With 250,000 patients on these drugs worldwide, that is “very reassuring,” Dr. Tepper said.
New data also show that the majority of patients with chronic migraine who are taking monoclonal antibodies convert from chronic migraine to episodic migraine. Additionally, new data show that use of monoclonal antibodies “dramatically reduce all migraine medication use,” Dr. Tepper said.
PHILADELPHIA – At the annual meeting of the American Academy of Neurology, Alan M. Rapoport, MD, clinical professor of neurology at the University of California, Los Angeles, sat down with Stewart J. Tepper, MD, professor of neurology at the Geisel School of Medicine at Dartmouth, Hanover, N.H., to discuss in a video some of the new data presented at the meeting regarding the CGRP monoclonal antibodies, the small molecule receptor antagonists (gepants), and what Dr. Tepper described as “a real shift in paradigm and a watershed moment in migraine.”
The three gepants that are farthest along in clinical trials are ubrogepant, rimegepant, and atogepant. “Reassuring data” was presented, Dr. Tepper said, regarding liver toxicity, which has been a concern with earlier generations of the gepants. The Food and Drug Administration had mandated a close look at liver function with the use of these drugs, which are metabolized in the liver, and, to date, no safety signals have emerged.
The three CGRP monoclonal antibodies that are currently on the market are erenumab (Aimovig), fremanezumab (Ajovy), and galcanezumab (Emgality). Data from numerous open-label extension studies were presented. In general, it seems that “the monoclonal antibodies accumulate greater efficacy over time,” Dr. Tepper said. No safety concerns have emerged from 5 years of clinical trial data. With 250,000 patients on these drugs worldwide, that is “very reassuring,” Dr. Tepper said.
New data also show that the majority of patients with chronic migraine who are taking monoclonal antibodies convert from chronic migraine to episodic migraine. Additionally, new data show that use of monoclonal antibodies “dramatically reduce all migraine medication use,” Dr. Tepper said.
PHILADELPHIA – At the annual meeting of the American Academy of Neurology, Alan M. Rapoport, MD, clinical professor of neurology at the University of California, Los Angeles, sat down with Stewart J. Tepper, MD, professor of neurology at the Geisel School of Medicine at Dartmouth, Hanover, N.H., to discuss in a video some of the new data presented at the meeting regarding the CGRP monoclonal antibodies, the small molecule receptor antagonists (gepants), and what Dr. Tepper described as “a real shift in paradigm and a watershed moment in migraine.”
The three gepants that are farthest along in clinical trials are ubrogepant, rimegepant, and atogepant. “Reassuring data” was presented, Dr. Tepper said, regarding liver toxicity, which has been a concern with earlier generations of the gepants. The Food and Drug Administration had mandated a close look at liver function with the use of these drugs, which are metabolized in the liver, and, to date, no safety signals have emerged.
The three CGRP monoclonal antibodies that are currently on the market are erenumab (Aimovig), fremanezumab (Ajovy), and galcanezumab (Emgality). Data from numerous open-label extension studies were presented. In general, it seems that “the monoclonal antibodies accumulate greater efficacy over time,” Dr. Tepper said. No safety concerns have emerged from 5 years of clinical trial data. With 250,000 patients on these drugs worldwide, that is “very reassuring,” Dr. Tepper said.
New data also show that the majority of patients with chronic migraine who are taking monoclonal antibodies convert from chronic migraine to episodic migraine. Additionally, new data show that use of monoclonal antibodies “dramatically reduce all migraine medication use,” Dr. Tepper said.
EXPERT ANALYSIS FROM AAN 2019
Appendectomy linked to increased risk of subsequent Parkinson’s
.
“One of the factors that’s seen in the brains of patients with Parkinson’s disease is accumulation of an abnormal protein known as alpha-synuclein,” one of the study authors, Gregory S. Cooper, MD, said during a media briefing in advance of the annual Digestive Disease Week. “It’s released by damaged nerve cells in the brain. Not only is alpha-synuclein found in the brain of patients with Parkinson’s disease; it’s also found in the GI tract. It’s thought that its accumulation in the GI tract occurs prior to the development of its accumulation in the brain.”
This has prompted scientists around the world to evaluate the GI tract, including the appendix, for evidence about the pathophysiology and onset of Parkinson’s disease, said Dr. Cooper, professor of medicine, oncology, and population and quantitative health sciences at Case Western Reserve University, Cleveland. “It’s thought that, potentially, in the presence of inflammation, [molecules] of this protein are released from damaged nerves in the gut and then are transported to the brain, where they accumulate,” he said. “Or, it could be that the appendix is a storage place for this protein and gets released at the time of appendectomy.”
To investigate if appendectomy increases the risk of Parkinson’s disease, Dr. Cooper and colleagues drew from the Explorys database, which contains EHRs from 26 integrated U.S. health care systems. They limited their search to patients who underwent appendectomies and those who were diagnosed with Parkinson’s disease based on Systematized Nomenclature of Medicine–Clinical Terms. The researchers chose a washout period of 6 months to the development of Parkinson’s disease after appendectomy, and compared the prevalence of Parkinson’s disease in the general population to those with appendectomies.
Of the 62,218,050 records in the database, Dr. Cooper and colleagues identified 488,190 patients who underwent appendectomies. In all, 4,470 cases of Parkinson’s disease were observed in patients with appendectomies, and 177,230 cases of Parkinson’s disease in patients without appendectomies. The overall relative risk of developing Parkinson’s disease in patients after appendectomies was 3.19 (95% confidence interval, 3.10-3.28; P less than .0001), compared with those who did not undergo the procedure. The relative risk was higher in patients aged 18-64 years (RR, 4.27; 95% CI, 3.99-4.57; P less than .0001), compared with those 65 years and older (RR, 2.20; 95% CI, 2.13-2.27; P less than .0001). “We know that Parkinson’s disease is more common in the elderly,” Dr. Cooper said. “But at virtually all ages, the prevalence of Parkinson’s disease was higher in patients who had an appendectomy, compared to those without an appendectomy.”
The overall relative risk of developing Parkinson’s disease in patients after appendectomies was slightly higher in females (RR, 3.86; 95% CI, 3.71-4.02; P less than .0001), compared with males (RR, 2.67; 95% CI, 2.56-2.79; P less than .0001). The researchers also observed a similar effect of appendectomy by race. The overall relative risk of developing Parkinson’s disease in patients after appendectomy was slightly higher in African Americans (RR, 3.11; 95% CI, 2.69-3.58; P less than .0001), compared with Asians (RR, 2.73; 95% CI, 2.19-3.41; P less than .0001), and whites (RR, 2.55; 95% CI, 2.48-2.63; P less than .0001).
“If these data get borne out, it may question the role of doing a discretionary appendectomy in a patient who’s having surgery for another reason,” Dr. Cooper said. “Our research does show a clear relationship between appendectomy and Parkinson’s disease. However, at this point, it’s only an association. As a next step, we’d like to conduct additional research to confirm this connection and better understand the mechanisms involved.”
He pointed out that, because of the nature of the Explorys database, he and his colleagues were unable to determine the length of time following appendectomy to the development of Parkinson’s disease.
The study’s lead author was Mohammed Z. Sheriff, MD, also of Case Western Reserve University, Cleveland. The researchers reported having no financial disclosures.
SOURCE: Sheriff MZ et al. DDW 2019, Abstract 739.
.
“One of the factors that’s seen in the brains of patients with Parkinson’s disease is accumulation of an abnormal protein known as alpha-synuclein,” one of the study authors, Gregory S. Cooper, MD, said during a media briefing in advance of the annual Digestive Disease Week. “It’s released by damaged nerve cells in the brain. Not only is alpha-synuclein found in the brain of patients with Parkinson’s disease; it’s also found in the GI tract. It’s thought that its accumulation in the GI tract occurs prior to the development of its accumulation in the brain.”
This has prompted scientists around the world to evaluate the GI tract, including the appendix, for evidence about the pathophysiology and onset of Parkinson’s disease, said Dr. Cooper, professor of medicine, oncology, and population and quantitative health sciences at Case Western Reserve University, Cleveland. “It’s thought that, potentially, in the presence of inflammation, [molecules] of this protein are released from damaged nerves in the gut and then are transported to the brain, where they accumulate,” he said. “Or, it could be that the appendix is a storage place for this protein and gets released at the time of appendectomy.”
To investigate if appendectomy increases the risk of Parkinson’s disease, Dr. Cooper and colleagues drew from the Explorys database, which contains EHRs from 26 integrated U.S. health care systems. They limited their search to patients who underwent appendectomies and those who were diagnosed with Parkinson’s disease based on Systematized Nomenclature of Medicine–Clinical Terms. The researchers chose a washout period of 6 months to the development of Parkinson’s disease after appendectomy, and compared the prevalence of Parkinson’s disease in the general population to those with appendectomies.
Of the 62,218,050 records in the database, Dr. Cooper and colleagues identified 488,190 patients who underwent appendectomies. In all, 4,470 cases of Parkinson’s disease were observed in patients with appendectomies, and 177,230 cases of Parkinson’s disease in patients without appendectomies. The overall relative risk of developing Parkinson’s disease in patients after appendectomies was 3.19 (95% confidence interval, 3.10-3.28; P less than .0001), compared with those who did not undergo the procedure. The relative risk was higher in patients aged 18-64 years (RR, 4.27; 95% CI, 3.99-4.57; P less than .0001), compared with those 65 years and older (RR, 2.20; 95% CI, 2.13-2.27; P less than .0001). “We know that Parkinson’s disease is more common in the elderly,” Dr. Cooper said. “But at virtually all ages, the prevalence of Parkinson’s disease was higher in patients who had an appendectomy, compared to those without an appendectomy.”
The overall relative risk of developing Parkinson’s disease in patients after appendectomies was slightly higher in females (RR, 3.86; 95% CI, 3.71-4.02; P less than .0001), compared with males (RR, 2.67; 95% CI, 2.56-2.79; P less than .0001). The researchers also observed a similar effect of appendectomy by race. The overall relative risk of developing Parkinson’s disease in patients after appendectomy was slightly higher in African Americans (RR, 3.11; 95% CI, 2.69-3.58; P less than .0001), compared with Asians (RR, 2.73; 95% CI, 2.19-3.41; P less than .0001), and whites (RR, 2.55; 95% CI, 2.48-2.63; P less than .0001).
“If these data get borne out, it may question the role of doing a discretionary appendectomy in a patient who’s having surgery for another reason,” Dr. Cooper said. “Our research does show a clear relationship between appendectomy and Parkinson’s disease. However, at this point, it’s only an association. As a next step, we’d like to conduct additional research to confirm this connection and better understand the mechanisms involved.”
He pointed out that, because of the nature of the Explorys database, he and his colleagues were unable to determine the length of time following appendectomy to the development of Parkinson’s disease.
The study’s lead author was Mohammed Z. Sheriff, MD, also of Case Western Reserve University, Cleveland. The researchers reported having no financial disclosures.
SOURCE: Sheriff MZ et al. DDW 2019, Abstract 739.
.
“One of the factors that’s seen in the brains of patients with Parkinson’s disease is accumulation of an abnormal protein known as alpha-synuclein,” one of the study authors, Gregory S. Cooper, MD, said during a media briefing in advance of the annual Digestive Disease Week. “It’s released by damaged nerve cells in the brain. Not only is alpha-synuclein found in the brain of patients with Parkinson’s disease; it’s also found in the GI tract. It’s thought that its accumulation in the GI tract occurs prior to the development of its accumulation in the brain.”
This has prompted scientists around the world to evaluate the GI tract, including the appendix, for evidence about the pathophysiology and onset of Parkinson’s disease, said Dr. Cooper, professor of medicine, oncology, and population and quantitative health sciences at Case Western Reserve University, Cleveland. “It’s thought that, potentially, in the presence of inflammation, [molecules] of this protein are released from damaged nerves in the gut and then are transported to the brain, where they accumulate,” he said. “Or, it could be that the appendix is a storage place for this protein and gets released at the time of appendectomy.”
To investigate if appendectomy increases the risk of Parkinson’s disease, Dr. Cooper and colleagues drew from the Explorys database, which contains EHRs from 26 integrated U.S. health care systems. They limited their search to patients who underwent appendectomies and those who were diagnosed with Parkinson’s disease based on Systematized Nomenclature of Medicine–Clinical Terms. The researchers chose a washout period of 6 months to the development of Parkinson’s disease after appendectomy, and compared the prevalence of Parkinson’s disease in the general population to those with appendectomies.
Of the 62,218,050 records in the database, Dr. Cooper and colleagues identified 488,190 patients who underwent appendectomies. In all, 4,470 cases of Parkinson’s disease were observed in patients with appendectomies, and 177,230 cases of Parkinson’s disease in patients without appendectomies. The overall relative risk of developing Parkinson’s disease in patients after appendectomies was 3.19 (95% confidence interval, 3.10-3.28; P less than .0001), compared with those who did not undergo the procedure. The relative risk was higher in patients aged 18-64 years (RR, 4.27; 95% CI, 3.99-4.57; P less than .0001), compared with those 65 years and older (RR, 2.20; 95% CI, 2.13-2.27; P less than .0001). “We know that Parkinson’s disease is more common in the elderly,” Dr. Cooper said. “But at virtually all ages, the prevalence of Parkinson’s disease was higher in patients who had an appendectomy, compared to those without an appendectomy.”
The overall relative risk of developing Parkinson’s disease in patients after appendectomies was slightly higher in females (RR, 3.86; 95% CI, 3.71-4.02; P less than .0001), compared with males (RR, 2.67; 95% CI, 2.56-2.79; P less than .0001). The researchers also observed a similar effect of appendectomy by race. The overall relative risk of developing Parkinson’s disease in patients after appendectomy was slightly higher in African Americans (RR, 3.11; 95% CI, 2.69-3.58; P less than .0001), compared with Asians (RR, 2.73; 95% CI, 2.19-3.41; P less than .0001), and whites (RR, 2.55; 95% CI, 2.48-2.63; P less than .0001).
“If these data get borne out, it may question the role of doing a discretionary appendectomy in a patient who’s having surgery for another reason,” Dr. Cooper said. “Our research does show a clear relationship between appendectomy and Parkinson’s disease. However, at this point, it’s only an association. As a next step, we’d like to conduct additional research to confirm this connection and better understand the mechanisms involved.”
He pointed out that, because of the nature of the Explorys database, he and his colleagues were unable to determine the length of time following appendectomy to the development of Parkinson’s disease.
The study’s lead author was Mohammed Z. Sheriff, MD, also of Case Western Reserve University, Cleveland. The researchers reported having no financial disclosures.
SOURCE: Sheriff MZ et al. DDW 2019, Abstract 739.
REPORTING FROM DDW 2019
Key clinical point: Appendectomy appears to increase the risk of Parkinson’s disease.
Major finding: The overall relative risk of developing Parkinson’s disease in patients after appendectomy was 3.19 (95% CI, 3.10-3.28; P less than .0001), compared with those who did not undergo the procedure.
Study details: A population-based study of more than 62 million medical records from a national database.
Disclosures: The researchers reported having no financial disclosures.
Source: Sheriff MZ et al. DDW 2019, Abstract 739.