Real-World Hereditary Angioedema Attack Rates Before and After Berotralstat Initiation Among Patients With C1 Inhibitor Deficiency (Type I/II) and =8 Attacks Per Month
Author(s)
Raffi Tachdjian, MD, MPH1, Mark Davis-Lorton, MD2, Lorena Lopez-Gonzalez, PhD3, Sean D. MacKnight, MScPH4, François Laliberte, MA4, Ramya Ramasubramanian, PhD5, Patrick Gillard, PharmD, MS3, William R. Lumry, MD, FACP, FAAAAI6;
1University of California Los Angeles, Department of Pediatrics, Los Angeles, CA, USA, 2ENT and Allergy Associates, Tarrytown, NY, USA, 3BioCryst Pharmaceuticals, Inc., Durham, NC, USA, 4Groupe d'Analyse, Ltée, Montreal, QC, Canada, 5Analysis Group, Inc., Los Angeles, CA, USA, 6Allergy and Asthma Research Associates, Dallas, TX, USA
1University of California Los Angeles, Department of Pediatrics, Los Angeles, CA, USA, 2ENT and Allergy Associates, Tarrytown, NY, USA, 3BioCryst Pharmaceuticals, Inc., Durham, NC, USA, 4Groupe d'Analyse, Ltée, Montreal, QC, Canada, 5Analysis Group, Inc., Los Angeles, CA, USA, 6Allergy and Asthma Research Associates, Dallas, TX, USA
Presentation Documents
OBJECTIVES: This study evaluated hereditary angioedema (HAE) attacks before and after initiation of berotralstat, an oral long-term prophylaxis, among patients with C1-inhibitor deficiency (Type I/II; HAE-C1-INH) and ≥8 attacks per month. Frequent HAE attack rates before berotralstat initiation likely reflect a patient population with more severe underlying disease activity.
METHODS: This retrospective pre-post study used Specialty Pharmacy Data (December 15, 2020 - January 8, 2024) from Optime Care, the sole dispenser of berotralstat in the US. Patients were included if they had ≥2 berotralstat dispensings (first dispensing = index), C1-inhibitor deficiency based on laboratory measurements and ≥8 attacks/month (i.e., ≥7.5 due to rounding) at baseline based on the last 90 days before index. Patients were also required to have had ≥90 days of follow-up, which spanned from index to the last berotralstat dispensing (segmented into fixed 90-day intervals), and ≥1 self-assessment of attacks in both baseline and follow-up. 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 56 eligible patients, mean age was 41.3 years and most were female (76.8%). Mean baseline attack rate ranged from 7.78-8.23 attacks/month across 90-day follow-up intervals. Patients experienced significantly lower attack rates after berotralstat initiation during every 90-day follow-up interval relative to baseline, with 6.25 (95% CI: [5.63, 6.87]; p<0.001) fewer attacks per month at 12 months (i.e., 271-360 day interval) and 6.43 (95% CI: [5.78, 7.09]; p<0.001) fewer attacks per month at 18 months (i.e., 451-540 day interval).
CONCLUSIONS: Patients with ≥8 monthly baseline attacks and C1-inhibitor deficiency reported significantly lower HAE attack rates after berotralstat initiation. Treatment effectiveness was consistent and sustained through 18 months of follow-up.
METHODS: This retrospective pre-post study used Specialty Pharmacy Data (December 15, 2020 - January 8, 2024) from Optime Care, the sole dispenser of berotralstat in the US. Patients were included if they had ≥2 berotralstat dispensings (first dispensing = index), C1-inhibitor deficiency based on laboratory measurements and ≥8 attacks/month (i.e., ≥7.5 due to rounding) at baseline based on the last 90 days before index. Patients were also required to have had ≥90 days of follow-up, which spanned from index to the last berotralstat dispensing (segmented into fixed 90-day intervals), and ≥1 self-assessment of attacks in both baseline and follow-up. 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 56 eligible patients, mean age was 41.3 years and most were female (76.8%). Mean baseline attack rate ranged from 7.78-8.23 attacks/month across 90-day follow-up intervals. Patients experienced significantly lower attack rates after berotralstat initiation during every 90-day follow-up interval relative to baseline, with 6.25 (95% CI: [5.63, 6.87]; p<0.001) fewer attacks per month at 12 months (i.e., 271-360 day interval) and 6.43 (95% CI: [5.78, 7.09]; p<0.001) fewer attacks per month at 18 months (i.e., 451-540 day interval).
CONCLUSIONS: Patients with ≥8 monthly baseline attacks and C1-inhibitor deficiency reported significantly lower HAE attack rates after berotralstat initiation. Treatment effectiveness was consistent and sustained 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
PCR182
Topic
Patient-Centered Research
Topic Subcategory
Patient-reported Outcomes & Quality of Life Outcomes
Disease
SDC: Rare & Orphan Diseases