OBJECTIVES: Prophylactic therapies are an important tool in HAE management though persistency on therapy may be suboptimal. To determine reasons for discontinuation, we reviewed office visit notes and EMR data generated from community care.

METHODS: Data: PIONEER-HAE, a database containing EMR, continuity of care documents, and extracted visit note data, specific to patients managed by the Consortium of Independent Immunology Clinics (CIIC). Unstructured data were extracted into electronic forms by clinically-trained scribes and included attack details, on demand treatment, symptoms onset and diagnosis, family history, comorbidities, prophylactic treatment initiation, discontinuation, and reasons for discontinuation. Form data were subjected to logical checks, random audit (1-2%), and tertiary data review after combining with CCD and EMR data. Patient selection: Type I or II HAE treated with FDA-approved prophylactic drugs between 2018-2022. Drug episodes, defined as prophylactic drug-specific treatment without drug-free periods of >90 days, occurring between 2018-2022 were examined.

RESULTS: Study population characteristics (n=183): 63% (116) female, mean (median) age 43 (43) years with 15% (27) ≥ 65 and 5% (9) <18 years old, predominantly white (87%, 116/134), commercial insurance coverage (73%, 134), and type I HAE (90%, 164). Drug episodes (n=292): 14% (40) androgens, 2% (5) anti-fibrinolytics, 39% (114) C1-esterase inhibitors, 46% (133) kallikrein inhibitors. As of Dec 2022, 174 drug episodes were active. For the remaining 118 discontinued episodes, 91 had documented discontinuation reasons: 26% (24/91) lack/loss of efficacy, 23% (21/91) comorbids or observed/anticipated adverse events (e.g. weight gain, concern over liver damage), 23% (21/91) patient preferences (e.g. anticipated pregnancy, prefer different drug), and 16% (15/91) payer/payment issues (e.g. denied, can’t afford).

CONCLUSIONS: In this study of prophylactic HAE treatments, 74% of evaluable episodes were discontinued for reasons other than lack of efficacy. These data serve as an important first step towards 1) awareness of real-world factors impacting persistency and 2) optimized disease management.