From the Clinical Research, AllerVie Health, Birmingham, Alabama.
Allergy & Immunology, Asthma & Allergy Associates, Colorado Springs, Colorado.
Allergy Asthma Proc. 2024 Nov 1;45(6):426-433. doi: 10.2500/aap.2024.45.240046. Epub 2024 Sep 18.
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. 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. 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. 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). 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.
遗传性血管性水肿 (HAE) 是一种罕见的遗传性疾病,其特征是疼痛、虚弱和潜在致命的肿胀发作。拉那芦单抗已被批准用于 HAE 患者的长期预防 (LTP)。然而,HAE 患者使用 LTP 的真实世界数据有限。本研究旨在描述接受拉那芦单抗或其他 LTP 治疗的 HAE Ⅰ/Ⅱ型患者的临床特征、发作史和生活质量 (QoL)。数据来自 Adelphi HAE 疾病专项计划,这是一项 2021 年 7 月至 11 月在美国开展的 HAE 医生的横断面调查。比较了接受拉那芦单抗或其他 LTP 治疗至少 12 个月的患者的疾病特征、HAE 发作频率和 QoL。医生报告了 144 名患者的数据,其中 29 名接受了拉那芦单抗治疗,115 名接受了其他预防治疗至少 12 个月。在过去 12 个月中,接受拉那芦单抗治疗的患者的平均发作次数(2.3±3.1)低于接受其他 LTP 治疗的患者(3.4±2.8;p=0.075)。尽管两组患者当前疾病活动度和严重程度相似,但接受拉那芦单抗治疗的患者中有更多患者具有高疾病活动度(51.7%比 12.5%;p<0.0001)和严重疾病严重程度(51.7%比 16.1%;p=0.0001)。医生报告称,接受拉那芦单抗治疗的患者中有更多患者(72.4%)的 QoL 良好或非常好,而接受其他 LTP 治疗的患者(36.5%)则较少(p=0.003)。这些发现表明,在真实世界环境中,接受拉那芦单抗治疗的患者发作频率较低,症状影响较小,生活质量较好。