D2.361 - Reductions in Healthcare Resource Utilization in Adolescents with Hereditary Angioedema on Berotralstat

Hereditary angioedema (HAE) is a rare disease characterized by painful and potentially life-threatening swelling attacks. Berotralstat, a once-daily, oral long-term prophylaxis for HAE, reduces the frequency and severity of attacks. This real-world study evaluated the impact of berotralstat initiation on healthcare resource utilization among adolescents with HAE.

Optime Care Specialty Pharmacy data (Dec. 3, 2020 – Jan. 31, 2024) were linked to Komodo Healthcare Map claims data (Oct. 1, 2015 – Jan. 31, 2024) using de-identified patient tokens. Optime Care is the sole dispenser of berotralstat in the United States. The study population had ≥2 berotralstat dispensings (first=index) recorded in Optime data, ≥6 months of continuous insurance enrollment pre-index in Komodo data, evidence of HAE during continuous eligibility any time pre-index in Komodo data, and were adolescents aged 12–17 years at index. Rates of healthcare resource utilization were defined as angioedema-related outpatient (OP)/emergency department (ED) visits and hospitalizations per patient-year (PPY) up to 24 months post-index, which were compared to pre-index rates using rate ratios, 95% confidence intervals (CIs), and p-values from generalized estimating equations Poisson regression models with robust standard errors.

Of 52 eligible adolescents, the mean age was 15.1 years and 65.4% were female. Most adolescents were HAE type 1 or 2 (61.5%), with 9.6% having HAE with normal C1 esterase inhibitor and 28.9% having undetermined HAE type. Before berotralstat initiation, the rate of angioedema-related OP/ED visits was 4.7 PPY, which decreased significantly by 58% after berotralstat initiation (rate ratio [95% CI]: 0.42 [0.31–0.56], p<0.001). The rate of angioedema-related hospitalizations was 0.5 PPY pre-index and 0.4 PPY post-index (rate ratio [95% CI]: 0.84 [0.33–2.11], non-significant).

This real-world study of linked specialty pharmacy and healthcare claims data found significant reductions in healthcare resource utilization after berotralstat initiation among adolescents with HAE.