OBJECTIVES: To evaluate angioedema-related healthcare resource utilization (HRU) before and after initiation of berotralstat for long-term prophylaxis (LTP) of hereditary angioedema (HAE) in the US.

METHODS: The study was designed as a retrospective pre-post HRU analysis. Claims data from Komodo’s Healthcare Map was used to identify patients with evidence of HAE before berotralstat initiation (December 2020–December 2022), with their first dispensing defining the index date. Other inclusion criteria were ≥6 months of continuous insurance eligibility pre-index and age ≥12 years at index. Patients were followed from index until the end of the study period or enrollment, whichever occurred first. Evidence of HAE included any of the following: diagnosis codes (ICD-10-CM: D84.1, D68.2, T78.3x), HAE medication use (on-demand or LTP), or diagnostic HAE laboratory tests. Medical visits with evidence of HAE were defined as angioedema-related. Rates of angioedema-related visits per person-year (PPY) were compared post- versus pre-index using rate ratios and p-values from generalized estimating equations Poisson regression models with robust standard errors. Results were also evaluated based on prior HAE treatment experience.

RESULTS: Of 260 berotralstat patients in the study population, the average age was 40 years and the majority female (74.2%). Patients were stratified into LTP-experienced (n=126, 48.5%), LTP-naïve and on-demand treatment experienced (n=67, 25.8%), and HAE treatment naïve (n=67, 25.8%). Overall, rates of angioedema-related medical visits decreased post- versus pre-berotralstat initiation, driven by significant reductions in hospitalizations (0.19 vs. 0.39 PPY; 52% reduction, p=0.001) and outpatient/emergency room (ER) visits (2.72 vs. 4.84 PPY; 44% reduction, p<0.001). Results were similar among the LTP-experienced subgroup (hospitalizations: 54% reduction, p=0.050; outpatient/ER visits: 37% reduction, p<0.001) and the LTP-naïve and on-demand treatment experienced subgroup (hospitalizations: 62% reduction, p=0.003; outpatient/ER visits: 45% reduction, p<0.001).

CONCLUSIONS: Results from this real-world study of patients with HAE demonstrate that berotralstat was associated with significant reductions in angioedema-related HRU.