Reductions in medical visits and hospitalizations following berotralstat initiation in patients with hereditary angioedema in the United States.

Background: Hereditary angioedema (HAE) is a rare disease characterized by unpredictable recurrent, debilitating, and potentially fatal attacks of subcutaneous and submucosal tissue swelling.

Objective: To evaluate all-cause, angioedema-related, and HAE attack-related medical visits and hospitalizations before and after initiation of berotralstat long-term prophylaxis (LTP) for patients with HAE in the United States.

Methods: This retrospective pre-post analysis used Komodo's Healthcare Map claims data to identify patients who initiated berotralstat (December 2020 to December 2022). The first entry for berotralstat dispensing was defined as the index date. Inclusion criteria comprised patients aged at least 12 years at index with at least 6 months of continuous insurance eligibility pre-index and evidence consistent with HAE pre-index (International Classification of Diseases, Tenth Revision, Clinical Modification diagnosis codes D84.1, D68.2, or T78.3x; medication use [on-demand or LTP]; or presence of diagnostic HAE laboratory tests). Rates of all-cause, angioedema-related, and HAE attack-related medical visits per person-year were compared post-index vs pre-index using rate ratios with 95% CIs and P values from generalized estimating equation Poisson regression models with robust SEs. Study limitations included the inability to distinguish HAE types and the uncertainty of whether a dispensed medication was consumed or taken as prescribed.

Results: The study population included 260 patients treated with berotralstat (mean age = 39.7 years; 74.2% female). After berotralstat initiation, there were significant decreases in the rates of all-cause health care resource utilization (HRU): all-cause inpatient (IP) visits decreased by 34% (P = 0.037) and all-cause outpatient/emergency department (OP/ED) visits decreased by 14% (P = 0.005). There were also significant decreases in rates of angioedema-related HRU (IP visits: 52%, P = 0.001; OP/ED visits: 44%, P < 0.001) as well as HAE attack-related HRU (IP visits: 60%, P < 0.001; OP/ED visits: 50%, P < 0.001). Use of on-demand medications decreased significantly after berotralstat initiation (32%, P = 0.002). Results were similar among subgroups of patients defined by HAE treatment history, including patients who were LTP-experienced (n = 126) and LTP-naive but on-demand treatment-experienced (n = 67).

Conclusions: Prophylactic treatment of HAE with berotralstat was associated with significant reductions in all-cause HRU, including decreases to angioedema-related and HAE attack-related medical visits, hospitalizations, and administration of on-demand treatment.