ArticleOrphanet journal of rare diseases2026
Real-world treatment patterns and clinical burden of patients with hereditary angioedema treated with long-term prophylaxis.
Article in Orphanet journal of rare diseases, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.
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Abstract
backgroundLong-term prophylaxis (LTP) may reduce the hereditary angioedema (HAE) burden by reducing attack frequency, but real-world evidence is limited.
objectiveTo describe LTP treatment patterns and the frequency of HAE attacks leading to emergency department or inpatient visits (ED/IP) and healthcare resource use (HRU) before and after LTP initiation.
methodsPatients initiating LTP in the Komodo Research Database (1/1/2021-1/30/2024) were followed until discontinuation or data end. Treatment adherence overall and among patients with > 30 days of treatment was measured as the proportion of days covered ≥ 80%. Treatment persistence, defined as no interruption ≥ 90 days or switch, was evaluated overall and among patients with > 30 days of treatment using Kaplan-Meier (KM) analysis with KM rates and corresponding 95% confidence intervals (CI). Rates of HAE attacks leading to ED/IP and per-patient-per-year all-cause HRU before and after LTP initiation were reported among patients with > 30 days of treatment. All analyses were descriptive and no statistical testing was conducted.
resultsOverall, 499 patients were included (57 Haegarda [11%], 257 Orladeyo [52%], 185 Takhzyro [37%]; mean follow-up = 11.3 months; median age = 39 years; 69% female), among whom 449 had > 30 days of treatment (mean follow-up = 12.5 months). At 12 months after LTP initiation, 52.6% of patients in the overall study sample and 57.0% of patients with > 30 days of treatment were adherent. Based on KM analysis, persistence rates at 12 months after LTP initiation were 58.7% (95% CI = 53.8%; 63.4%) in the overall sample and 65.1% (59.9%; 69.9%) among patients with > 30 days of treatment. Among the patients with > 30 days of treatment, 38% had ≥ 1 HAE attack leading to ED/IP in the year before LTP initiation (mean = 0.82 attacks), decreasing to 23% after (mean = 0.51 attacks). In these 449 patients, annual HRU remained substantial before and after LTP initiation across settings (IP = 2.4 and 1.7 days [0.3 and 0.2 admissions]; ED = 1.5 and 1.2 visits; outpatient = 21.0 and 19.3 visits, respectively).
conclusionAlthough recently available LTP can partly reduce the clinical burden of patients with HAE, novel therapies are needed to further alleviate this burden.
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