Long-term prophylactic treatment and related real-world outcomes of patients with hereditary angioedema in Germany: A retrospective analysis of German claims and medical chart data

Abstract

Background Hereditary angioedema (HAE) is a rare genetic disorder characterized by recurrent swelling episodes that are potentially life threating and substantially impair quality of life. This study aimed to generate real-world evidence on the utilization of long-term prophylaxis (LTP), residual disease burden, and economic outcomes among patients with HAE in Germany. Methods This retrospective cohort study examined LTP patterns and associated real-world outcomes using 2 anonymized German statutory health insurance datasets (AOK PLUS and GWQ) and a medical chart review (MCR) from 1 specialized HAE center. Claims datasets included patients initiating LTP between 2019 and 2023. The MCR provided complementary clinical information and was used to assess the plausibility of prescription-based estimates for attack frequency. Outcomes included LTP treatment sequences, HAE attacks requiring on-demand treatment (ODT), healthcare resource utilization and direct cost. Results Across the claims datasets, 77 patients initiated first-line LTP: 30 (39.0%) with intravenous C1 esterase inhibitors (C1INH IV), 24 (31.2%) with lanadelumab, 16 (20.8%) with subcutaneous C1INH, and 7 (9.1%) with berotralstat. During follow-up, 27 patients (35.1%) switched therapy, most commonly from C1INH IV to lanadelumab. Despite prophylaxis, 64% of patients experienced at least 1 HAE attack requiring ODT, while 36% remained attack-free during the first year after LTP start. Median time to first treated attack ranged from 114 to 132 days. Prescription-based attack estimates were generally consistent with physician-documented attack frequencies but tended to yield slightly higher attack rates. Annual healthcare costs were driven primarily by outpatient medications, averaging €233,190 and €206,392 per person-year in the AOK PLUS and GWQ cohorts, respectively. Conclusion This study provides real-world evidence on LTP use in Germany and demonstrates that a substantial proportion of patients continues to experience treated attacks despite prophylaxis. These findings highlight the importance of ongoing monitoring, individualized treatment optimization, and continued access to ODT.

The authors' abstract, as published at the source. World Allergy Organization Journal, 2026 · DOI ↗