Garadacimab improves long-term health-related quality of life in patients with hereditary angioedema.

IF 2.6 3区 医学 Q2 ALLERGY
Mar Guilarte, William R Lumry, Markus Magerl, Inmaculada Martinez Saguer, Avner Reshef, Marta Sobotkova, Julia Braverman, John-Philip Lawo, Lolis Wieman, Chiara Nenci, Constance H Katelaris
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引用次数: 0

Abstract

Background: Hereditary angioedema (HAE) attacks substantially impair health-related quality of life (HRQoL). Current World Allergy Organization and the European Academy of Allergy and Clinical Immunology guidelines goals include complete control and normalization of patients' lives. Garadacimab (anti-activated factor XII monoclonal antibody) reduced the mean attack rate after first administration in the pivotal phase III (VANGUARD; NCT04656418) and ongoing long-term phase III open-label extension (OLE) (NCT04739059) studies. Objective: To report exploratory HRQoL data from the interim analysis of the phase III OLE study (data cutoff February 13, 2023). Methods: Patients ages ≥12 years and with HAE received garadacimab 200 mg subcutaneously once monthly in the OLE study. The patient population comprised patients who were garadacimab naive (received placebo in the previous phase III study and newly enrolled patients) and patients who received garadacimab in previous phase II/III studies. The Angioedema Quality of Life (AE-QoL) questionnaire, Treatment Satisfaction Questionnaire for Medication version II (TSQM II), and Work Productivity and Activity Impairment: General Health (WPAI:GH) questionnaire were administered at baseline and every 3 months during the OLE study. AE-QoL and TSQM II scores were evaluated in comparison with minimal clinically important differences (MCID). Results: Overall, 90 patients who were garadacimab naive and 71 patients with previous garadacimab exposure received garadacimab in the phase III OLE study. The mean ± standard deviation AE-QoL total score improved by 34.2 ± 18.8 points in patients who were garadacimab naive and by 2.3 ± 13.1 points further to the reduction experienced in patients with previous garadacimab exposure. The AE-QoL MCID was met by 92.1% of patients who were garadacimab naive; 81.6% of patients with previous garadacimab exposure experienced stable AE-QoL scores or further improvements per MCID. TSQM II scores were improved from day 1 with garadacimab and sustained to month 12. Improvements in WPAI:GH scores were consistent with AE-QoL and TSQM II. Conclusion: Garadacimab elicited clinically meaningful long-term improvements in HRQoL, work productivity, and treatment satisfaction in patients with HAE, which brought them closer to complete disease control and normalization of life.Clinical trial NCT04739059, clinicaltrials.gov.

Garadacimab改善遗传性血管性水肿患者的长期健康相关生活质量
背景:遗传性血管性水肿(HAE)发作严重损害健康相关生活质量(HRQoL)。目前世界过敏组织和欧洲过敏和临床免疫学学会的指导方针目标包括完全控制和正常化患者的生活。Garadacimab(抗活化因子XII单克隆抗体)降低了关键III期(VANGUARD;NCT04656418)和正在进行的长期III期开放标签扩展(OLE) (NCT04739059)研究。目的:报告III期OLE研究中期分析的探索性HRQoL数据(数据截止于2023年2月13日)。方法:在OLE研究中,年龄≥12岁且患有HAE的患者每月一次皮下注射加达西单抗200mg。患者群体包括未使用garadacimab的患者(在之前的III期研究中接受安慰剂和新入组的患者)和在之前的II/III期研究中接受garadacimab的患者。在OLE研究期间,在基线和每3个月进行一次血管性水肿生活质量(AE-QoL)问卷、药物治疗满意度问卷II (TSQM II)和工作效率和活动障碍:一般健康(WPAI:GH)问卷。AE-QoL和TSQM II评分比较最小临床重要差异(MCID)。结果:总体而言,在III期OLE研究中,90例首次使用加达西单抗的患者和71例既往使用过加达西单抗的患者接受了加达西单抗治疗。未经加达西单抗治疗的患者AE-QoL总分平均±标准差提高了34.2±18.8分,比先前接触过加达西单抗的患者进一步降低了2.3±13.1分。加达西单抗初始患者的AE-QoL MCID达到92.1%;81.6%的既往加达西单抗暴露患者的AE-QoL评分稳定或每MCID进一步改善。使用加达西单抗后,TSQM II评分从第1天开始改善,并持续到第12个月。WPAI:GH评分的改善与AE-QoL和TSQM II一致。结论:Garadacimab对HAE患者的HRQoL、工作效率和治疗满意度有临床意义的长期改善,使患者更接近疾病的完全控制和生活的正常化。临床试验NCT04739059, clinicaltrials.gov。
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来源期刊
CiteScore
5.70
自引率
35.70%
发文量
106
审稿时长
6-12 weeks
期刊介绍: Allergy & Asthma Proceedings is a peer reviewed publication dedicated to distributing timely scientific research regarding advancements in the knowledge and practice of allergy, asthma and immunology. Its primary readership consists of allergists and pulmonologists. The goal of the Proceedings is to publish articles with a predominantly clinical focus which directly impact quality of care for patients with allergic disease and asthma. Featured topics include asthma, rhinitis, sinusitis, food allergies, allergic skin diseases, diagnostic techniques, allergens, and treatment modalities. Published material includes peer-reviewed original research, clinical trials and review articles.
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