DOI 10.2500/aap.2024.45.240046
Real-world outcomes in patients with hereditary angioedema prescribed lanadelumab versus other prophylaxis.
Аннотация
Background
Hereditary angioedema (HAE) is a rare genetic disorder characterized by painful, debilitating, and potentially fatal swelling attacks. Lanadelumab is approved as long-term prophylaxis (LTP) in patients with HAE. However, real-world data on LTP use in patients with HAE are limited.
Objective
To describe clinical characteristics, attack history, and quality of life (QoL) of patients with HAE type I/II who were receiving lanadelumab or other LTPs.
Methods
Data were drawn from the Adelphi HAE Disease Specific Program, a cross-sectional survey of HAE physicians conducted in the United States from July to November 2021. Physician-reported disease characteristics, HAE attack frequency, and QoL were compared among patients receiving lanadelumab or other LTPs for at least 12 months.
Results
Physicians reported data for 144 patients, of whom 29 had received lanadelumab and 115 had received another prophylaxis for at least 12 months. The mean ± standard deviation number of attacks in the previous 12 months was lower among patients receiving lanadelumab than other LTPs (2.3 ± 3.1 versus 3.4 ± 2.8, respectively; p = 0.075). Although both groups had similar current disease activity and severity, more patients receiving lanadelumab versus other LTPs had high disease activity (51.7% versus 12.5%, respectively; p < 0.0001) and disease severity rated as severe (51.7% versus 16.1%, respectively; p = 0.0001) at diagnosis. Physicians reported that more patients who received lanadelumab had good or very good QoL (72.4%) than those receiving other LTPs (36.5%) (p = 0.003).
Conclusion
Analysis of these findings suggests lower attack frequency, lower symptomatic impact, and better QoL in patients treated with lanadelumab than another prophylaxis in a real-world setting.
Предметные рубрики MeSH
Подготовка и перевод публикации
Обработка статьи · этапы, ошибки и управление
Ошибки и ограничения
История · было → стало
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