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ASH 2024 | Real-world adherence and persistence to emicizumab in patients with hemophilia A in the US
Amy Shapiro, MD, Indiana University Hemophilia Center, Indianapolis, IN, provides insight into a retrospective real-world analysis assessing adherence and persistence to emicizumab prophylaxis in patients with hemophilia A in the United States. Findings from this analysis of claims data suggest that adherence to emicizumab continues to be high, with a low discontinuation rate. The low rate of factor VIII claims indicates that patients treated with emicizumab experience a low number of bleeding episodes requiring factor VIII treatment. These results are positive and consistent with previously published self-reported adherence results. This interview took place at the 66th ASH Annual Meeting and Exposition, held in San Diego, CA.
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Transcript (AI-generated)
This is a poster on the real-world adherence and persistence to emicizumab using a national U.S. claims database. Estimates of adherence to standard of care prophylaxis in people with hemophilia range from 47% to 82%. Approximately 35% of people with hemophilia A receiving factor VIII replacement products are non-adherent, although better adherence is associated with fewer bleeding episodes...
This is a poster on the real-world adherence and persistence to emicizumab using a national U.S. claims database. Estimates of adherence to standard of care prophylaxis in people with hemophilia range from 47% to 82%. Approximately 35% of people with hemophilia A receiving factor VIII replacement products are non-adherent, although better adherence is associated with fewer bleeding episodes.
Emicizumab is a bispecific factor IXa, factor X directed antibody approved in the U.S. for routine prophylaxis in adult and pediatric patients with hemophilia A with or without an inhibitor. It is administered subcutaneously on a flexible dosing schedule, either once per week, once every two weeks, or once every four weeks.
This poster described the real-world adherence to and persistence with emicizumab prophylaxis in the U.S. population of persons with hemophilia A using a retrospective observational cohort study through an adjudicated healthcare claims data from IQVIA PharMetrics.
Included patients were those with an ICD-10 code for hemophilia A between 5-1-2017 and 9-30-2023; they had to have greater than or equal to two emicizumab prescription fills greater than or equal to two days apart; the first fill being the index date, and continuous medical and pharmacy insurance benefit coverage for greater than or equal to six months before and greater than or equal to 12 months after that index fill. The patients were excluded if they had any ICD-10 code for hemophilia B or another rare bleeding disorder, for example, von Willebrand disease or acquired hemophilia A. Days supply was imputed from the claims with this missing field.
Adherence to emicizumab was defined as having greater than or equal to 80% proportion of days covered, which is PDC, during the 12-month period of time after the index case. PDC was determined by calculation of days covered by ME divided by the days in the period. Persistence with emicizumab was defined as a proportion of patients with hemophilia A who did not discontinue emicizumab within the 12 months of the index date. Discontinuation was defined as a treatment gap of greater than or equal to 60 days per expert clinical input.
Originally, there were 19,454 persons identified with an ICD-10 code for hemophilia A. Many of these were excluded in subsequent data cuts due to either not having greater than or equal to two emicizumab prescription fills greater than or equal to two days apart. They may not have had continuous medical and pharmacy benefits for greater than or equal to six months, or they may have had another bleeding disorder. In the end, there were 280 individuals in the group to be analyzed, which represent 1.4% of the original cohort.
Overall, the rates of adherence and persistence were very high across different age groups ranging from 61% to 97%. Although adherence was lower in the 19 to 30 year old age group, rates were not different from the greater than or equal to 51 year old age group. In the 213 patients with hemophilia who were adherent to emicizumab, most had no claims for factor VIII during the post-index period. Persons with hemophilia A had a mean of 1.14 factor VIII claims in the 12 months post-index. The mean number of days with factor VIII claims during the post-index period was 0.79.
Claims data only capture medications dispensed during a period of time and do not reflect a home supply of medications or if the medications were actually utilized by the patient, and these may represent weaknesses within the data inherent in the type of analysis that was performed. There may be misclassifications based upon the ICD-10 code, and this data represents commercial health insurance and may not be applicable to other forms of insurance for patients in the United States. Despite these limitations, long follow-up times and the sample size may provide strength to the analysis.
Real-world adherence to emicizumab as measured by claims data continues to be high with a low discontinuation rate. High adherence and few factor VIII claims suggest that emicizumab was well tolerated and efficacious. The low frequency of factor VIII claims filled during the 12-month post-index period can be correlated with low number of treated bleeding episodes. These emicizumab adherence results are consistent with previously published self-reported adherence results in persons with hemophilia A and caregivers.
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Disclosures
Plasminogen Deficiency Foundation: Membership on an entity’s Board of Directors or advisory committees; Regeneron: Research Funding; Sanofi-Genzyme/Bioverativ: Honoraria, Membership on an entity’s Board of Directors or advisory committees, Research Funding, Speakers Bureau; Hema Biologics: Membership on an entity’s Board of Directors or advisory committees; Novo Nordisk: Consultancy, Honoraria, Membership on an entity’s Board of Directors or advisory committees, Research Funding, Speakers Bureau; Pfizer: Membership on an entity’s Board of Directors or advisory committees, Research Funding; Kedrion Biopharma: Consultancy, Honoraria, Research Funding, Speakers Bureau; Takeda Pharmaceuticals: Research Funding; Pharmacosmos A/S: Research Funding; Cantessa Pharmaceuticals/ApcinteX Ltd.: Research Funding; Novo Nordisk Haemophilia Foundation: Membership on an entity’s Board of Directors or advisory committees; Be Biopharma: Membership on an entity’s Board of Directors or advisory committees; Genentech/Roche: Honoraria, Membership on an entity’s Board of Directors or advisory committees, Research Funding, Speakers Bureau; Indiana Hemophilia & Thrombosis Center, Inc.: Current Employment; BioMarin: Membership on an entity’s Board of Directors or advisory committees; NHPCC: Other: Medical Director.
