Real-World Hereditary Angioedema Attack Rates Before and After Berotralstat Initiation Among Adolescents
Author(s)
William R. Lumry, MD, FACP, FAAAAI1, Mark Davis-Lorton, MD2, Lorena Lopez-Gonzalez, PhD3, Sean D. MacKnight, MScPH4, François Laliberte, MA4, Cristina Martinez, MA4, Patrick Gillard, PharmD, MS3, Raffi Tachdjian, MD, MPH5;
1Allergy and Asthma Research Associates, Dallas, TX, USA, 2ENT and Allergy Associates, Tarrytown, NY, USA, 3BioCryst Pharmaceuticals, Inc., Durham, NC, USA, 4Groupe d'Analyse, Ltée, Montreal, QC, Canada, 5University of California Los Angeles, Department of Pediatrics, Los Angeles, CA, USA
1Allergy and Asthma Research Associates, Dallas, TX, USA, 2ENT and Allergy Associates, Tarrytown, NY, USA, 3BioCryst Pharmaceuticals, Inc., Durham, NC, USA, 4Groupe d'Analyse, Ltée, Montreal, QC, Canada, 5University of California Los Angeles, Department of Pediatrics, Los Angeles, CA, USA
Presentation Documents
OBJECTIVES: This study evaluated hereditary angioedema (HAE) attacks before and after initiation of berotralstat among adolescent patients with HAE. Berotralstat, a once-daily, oral long-term prophylaxis for HAE, may be preferred by younger patients over parenteral treatment options; however, limited real-world evidence is available regarding its impact on HAE attack rates among adolescents.
METHODS: This retrospective pre-post study used Specialty Pharmacy Data from Optime Care (December 15, 2020 - January 8, 2024), which includes patient-reported HAE attacks at each berotralstat dispensing. Optime Care is the sole dispenser of berotralstat in the US. Patients with ≥2 berotralstat dispensings (first dispensing = index) and aged 12-17 at index were included. Patients were also required to have had ≥90 days of follow-up and ≥1 self-assessment of attacks in both baseline and follow-up. Baseline was 90 days pre-index and follow-up spanned from index to the last berotralstat dispensing (segmented into fixed 90-day intervals). Each 90-day follow-up interval was analyzed separately. Monthly HAE attack rates in each follow-up interval were compared with baseline using mean differences, 95% confidence intervals (CIs), and p-values from generalized estimating equations linear regression models with robust standard errors.
RESULTS: Of 99 adolescents treated with berotralstat, mean age was 15.1 years and most were female (68.7%). Compared to baseline (mean baseline rate ranging from 2.07-2.30 attacks/month across follow-up intervals), adolescents experienced significantly lower attack rates after berotralstat initiation during each 90-day follow-up interval. Patients experienced 1.56 (95% CI: [0.89, 2.23]; p<0.001) fewer attacks per month at 12 months (i.e., 271-360 day interval) and 1.85 (95% CI: [1.12, 2.58]; p<0.001) fewer attacks per month at 18 months (i.e., 451-540 day interval).
CONCLUSIONS: Adolescents initiating berotralstat reported statistically significant and sustained reductions in HAE attack rates through 18 months of follow-up.
METHODS: This retrospective pre-post study used Specialty Pharmacy Data from Optime Care (December 15, 2020 - January 8, 2024), which includes patient-reported HAE attacks at each berotralstat dispensing. Optime Care is the sole dispenser of berotralstat in the US. Patients with ≥2 berotralstat dispensings (first dispensing = index) and aged 12-17 at index were included. Patients were also required to have had ≥90 days of follow-up and ≥1 self-assessment of attacks in both baseline and follow-up. Baseline was 90 days pre-index and follow-up spanned from index to the last berotralstat dispensing (segmented into fixed 90-day intervals). Each 90-day follow-up interval was analyzed separately. Monthly HAE attack rates in each follow-up interval were compared with baseline using mean differences, 95% confidence intervals (CIs), and p-values from generalized estimating equations linear regression models with robust standard errors.
RESULTS: Of 99 adolescents treated with berotralstat, mean age was 15.1 years and most were female (68.7%). Compared to baseline (mean baseline rate ranging from 2.07-2.30 attacks/month across follow-up intervals), adolescents experienced significantly lower attack rates after berotralstat initiation during each 90-day follow-up interval. Patients experienced 1.56 (95% CI: [0.89, 2.23]; p<0.001) fewer attacks per month at 12 months (i.e., 271-360 day interval) and 1.85 (95% CI: [1.12, 2.58]; p<0.001) fewer attacks per month at 18 months (i.e., 451-540 day interval).
CONCLUSIONS: Adolescents initiating berotralstat reported statistically significant and sustained reductions in HAE attack rates through 18 months of follow-up.
Conference/Value in Health Info
2025-05, ISPOR 2025, Montréal, Quebec, CA
Value in Health, Volume 28, Issue S1
Code
PCR132
Topic
Patient-Centered Research
Topic Subcategory
Patient-reported Outcomes & Quality of Life Outcomes
Disease
SDC: Rare & Orphan Diseases