遗传性血管性水肿患者服用拉那珠单抗与其他预防药物的实际效果。

John Anderson,Daniel Soteres,Raffi Tachdjian,Jennifer Mellor,Lucy Earl,Hannah Connolly,Kieran Wynne-Cattanach,Kellyn Moran,Krystal Sing,Bob G Schultz,Salome Juethner
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引用次数: 0

摘要

背景遗传性血管性水肿(HAE)是一种罕见的遗传性疾病,其特点是肿胀发作时疼痛难忍、使人衰弱并可能致命。拉那珠单抗被批准作为 HAE 患者的长期预防用药 (LTP)。目的描述接受拉那珠单抗或其他 LTPs 的 I/II 型 HAE 患者的临床特征、发作史和生活质量(QoL)。方法数据来自 Adelphi HAE 特定疾病项目,该项目是 2021 年 7 月至 11 月在美国对 HAE 医生进行的横断面调查。结果医生报告了144名患者的数据,其中29名患者接受了拉那珠单抗治疗,115名患者接受了至少12个月的其他预防治疗。接受拉那珠单抗治疗的患者在过去12个月中的平均+/-标准差发作次数低于接受其他LTP治疗的患者(分别为2.3 +/- 3.1对3.4 +/- 2.8;P = 0.075)。虽然两组患者目前的疾病活动度和严重程度相似,但接受拉那珠单抗治疗的患者比接受其他 LTP 治疗的患者在诊断时疾病活动度高(分别为 51.7% 对 12.5%;p < 0.0001),疾病严重程度被评为重度(分别为 51.7% 对 16.1%;p = 0.0001)。医生报告称,与接受其他 LTPs 治疗的患者(36.5%)相比,接受 lanadelumab 治疗的患者(72.4%)的 QoL 更好或非常好(p = 0.003)。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Real-world outcomes in patients with hereditary angioedema prescribed lanadelumab versus other prophylaxis.
BACKGROUND Hereditary angioedema (HAE) is a rare genetic disorder characterized by painful, debilitating, and potentiallyfatal swelling attacks. Lanadelumab is approved as long-term prophylaxis (LTP) in patients with HAE. However, realworld 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 whowere 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 loweramong patients receiving lanadelumab than other LTPs (2.3 +/- 3.1 versus 3.4 +/- 2.8, respectively; p = 0.075). Although bothgroups 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 inpatients treated with lanadelumab than another prophylaxis in a real-world setting.
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