REAL-WORLD TREATMENT PATTERNS AND ATTACK BURDEN IN HEREDITARY ANGIOEDEMA: FINDINGS FROM GERMAN CLAIMS DATA AND MEDICAL CHARTS
Author(s)
Nils Picker, MSc1, Thomas Wilke, Prof. Dr. rer. oec.2, Fabian Juelich, Dipl.-Kfm.3, Ulf Maywald, PharmD, MPH4, Jens Greve, Prof. Dr. med.5.
1GIPAM GmbH, Wismar, Germany, 2IPAM e.V., Wismar, Germany, 3CSL Behring GmbH, Hattersheim am Main, Germany, 4payers GmbH, Hamburg, Germany, 5Department of Otorhinolaryngology - Head and Neck Surgery, Ulm University Medical Center, Ulm, Germany.
1GIPAM GmbH, Wismar, Germany, 2IPAM e.V., Wismar, Germany, 3CSL Behring GmbH, Hattersheim am Main, Germany, 4payers GmbH, Hamburg, Germany, 5Department of Otorhinolaryngology - Head and Neck Surgery, Ulm University Medical Center, Ulm, Germany.
OBJECTIVES: Hereditary angioedema (HAE) due to C1 esterase inhibitor deficiency (C1-INH) is a rare genetic disorder characterized by recurrent swelling episodes. Long-term prophylaxis (LTP) is key to attack prevention. This study evaluated real-world LTP utilization and residual attack burden among patients with HAE in Germany.
METHODS: This cohort study examined LTP patterns and real-world outcomes in HAE in Germany using two anonymized claims databases (AOK PLUS and GWQ) and a single-center medical chart review (MCR). Claims data included patients initiating LTP between 2019 and 2023. Outcomes included first-line LTP utilization, treatment switching, and post-LTP attack occurrence and timing. HAE attacks in claims data were estimated using prescriptions for on-demand treatment (ODT). The MCR provided complementary clinical information to assess the agreement between ODT prescription-based estimates of treated attacks and clinically documented attack frequencies.
RESULTS: In the claims cohort, 77 patients initiated first-line LTP: 30 (39.0%) received intravenous C1-INH, 24 (31.2%) lanadelumab, 16 (20.8%) subcutaneous C1-INH, and 7 (9.1%) berotralstat. During follow-up, 27 patients (35.1%) switched LTP, mainly from intravenous C1-INH to lanadelumab. Among claims cohort patients, 64% had evidence of ≥1 HAE attack based on ODT prescriptions, while 36% remained attack-free during the first year after LTP initiation. Median time to first treated attack ranged from 114 to 132 days, although 24 patients received ODT on day 1. Comparison with medical records showed that attack frequencies derived from ODT prescriptions were slightly higher than those documented in clinical records (0.80 vs. 0.65 attacks per patient-year).
CONCLUSIONS: In this real-world study, LTP use evolved toward newer therapies, with over one-third of patients switching treatment, most commonly from intravenous C1-INH to lanadelumab. Despite prophylaxis, a substantial proportion of patients experienced HAE attacks, indicating residual disease burden. ODT prescription data provided a reasonable approximation of attack frequency, supporting its use for assessing HAE outcomes in claims-based research.
METHODS: This cohort study examined LTP patterns and real-world outcomes in HAE in Germany using two anonymized claims databases (AOK PLUS and GWQ) and a single-center medical chart review (MCR). Claims data included patients initiating LTP between 2019 and 2023. Outcomes included first-line LTP utilization, treatment switching, and post-LTP attack occurrence and timing. HAE attacks in claims data were estimated using prescriptions for on-demand treatment (ODT). The MCR provided complementary clinical information to assess the agreement between ODT prescription-based estimates of treated attacks and clinically documented attack frequencies.
RESULTS: In the claims cohort, 77 patients initiated first-line LTP: 30 (39.0%) received intravenous C1-INH, 24 (31.2%) lanadelumab, 16 (20.8%) subcutaneous C1-INH, and 7 (9.1%) berotralstat. During follow-up, 27 patients (35.1%) switched LTP, mainly from intravenous C1-INH to lanadelumab. Among claims cohort patients, 64% had evidence of ≥1 HAE attack based on ODT prescriptions, while 36% remained attack-free during the first year after LTP initiation. Median time to first treated attack ranged from 114 to 132 days, although 24 patients received ODT on day 1. Comparison with medical records showed that attack frequencies derived from ODT prescriptions were slightly higher than those documented in clinical records (0.80 vs. 0.65 attacks per patient-year).
CONCLUSIONS: In this real-world study, LTP use evolved toward newer therapies, with over one-third of patients switching treatment, most commonly from intravenous C1-INH to lanadelumab. Despite prophylaxis, a substantial proportion of patients experienced HAE attacks, indicating residual disease burden. ODT prescription data provided a reasonable approximation of attack frequency, supporting its use for assessing HAE outcomes in claims-based research.
Conference/Value in Health Info
2026-11, ISPOR Europe 2026, Vienna, Austria
Value in Health, Volume 29, Issue 12S
Code
EPH88
Topic
Epidemiology & Public Health
Topic Subcategory
Disease Classification & Coding
Disease
Rare & Orphan Diseases, Systemic Disorders/Conditions (Anesthesia, Auto-Immune Disorders (n.e.c.), Hematological Disorders (non-oncologic), Pain)