长期拉那芦单抗治疗可改善遗传性血管性水肿患者的健康相关生活质量。
Long-term lanadelumab treatment improves health-related quality of life in patients with hereditary angioedema.
机构信息
Allergy and Asthma Research Associates, Dallas, Texas.
Institute of Allergology, Charité - Universitätsmedizin Berlin, corporate member of Freie Universität Berlin and Humboldt-Universität zu Berlin, Berlin, Germany; Fraunhofer Institute for Translational Medicine and Pharmacology (ITMP), Immunology, and Allergology, Berlin, Germany.
出版信息
Ann Allergy Asthma Immunol. 2023 Jul;131(1):101-108.e3. doi: 10.1016/j.anai.2023.03.028. Epub 2023 Apr 5.
BACKGROUND
Hereditary angioedema (HAE) is associated with a substantial disease burden. Lanadelumab reduced the HAE attack rate during 132 weeks of follow-up in the HELP open-label extension (OLE) Study (NCT02741596).
OBJECTIVE
To measure the impact of long-term lanadelumab treatment on patient-reported outcomes (PROs).
METHODS
Rollover patients (completed the 26-week HELP study [NCT02586805]) and nonrollovers (newly enrolled) received lanadelumab 300 mg every 2 weeks. PROs (Angioedema Quality of Life Questionnaire [AE-QoL], Short Form Health Survey 12-item version 2, Hospital Anxiety and Depression Scale, Work Productivity and Activity Impairment-General Health Questionnaire, and EQ-5D-5L questionnaire) were assessed at baseline (day 0 of HELP OLE) and various time points until the end-of-study visit. The Angioedema Control Test, Treatment Satisfaction Questionnaire for Medication, and Global Impression of Treatment Response were administered starting at week 52.
RESULTS
The mean (SD) change in AE-QoL total score from baseline to end-of-study for rollovers (n = 90) was -10.2 (17.9), exhibiting further improvement from HELP in health-related quality of life (HRQoL); 48.9% of rollovers achieved the previously defined 6-point minimal clinically important difference. Nonrollovers (n = 81) reported a change of -19.5 (21.3). Controlled disease (Angioedema Control Test total score ≥10) was reported by 90.2% of rollovers and 95.9% of nonrollovers at the end of the study. Excellent treatment response was reported by 78.7% of patients and 82.4% of investigators. Results from other PROs indicated a slight improvement in anxiety, a high level of satisfaction with treatment, and increased work productivityor activity.
CONCLUSION
Clinically meaningful improvement in HRQoL was exhibited with long-term lanadelumab treatment, supporting the benefit of lanadelumab therapy associated with attack prevention.
TRIAL REGISTRATION
ClinicalTrials.gov Identifiers: NCT02586805 (HELP Study) and NCT02741596 (HELP open-label extension).
背景
遗传性血管性水肿 (HAE) 与大量疾病负担相关。在 HELP 开放性延伸 (OLE) 研究（NCT02741596）中，拉那芦单抗在 132 周的随访期间降低了 HAE 发作率。
目的
衡量长期拉那芦单抗治疗对患者报告结局 (PRO) 的影响。
方法
滚动入组患者（完成 26 周 HELP 研究 [NCT02586805]）和非滚动入组患者（新入组）接受拉那芦单抗 300mg，每两周一次。在基线（HELP OLE 研究的第 0 天）和研究结束访视之前的各种时间点评估 PROs（血管性水肿生活质量问卷 [AE-QoL]、健康调查简表 12 项版本 2、医院焦虑和抑郁量表、工作效率和活动障碍-一般健康问卷以及 EQ-5D-5L 问卷）。从第 52 周开始，评估血管性水肿控制测试、药物治疗满意度问卷和整体治疗反应印象。
结果
滚动入组患者（n=90）从基线到研究结束时 AE-QoL 总分的平均（SD）变化为-10.2（17.9），表明在健康相关生活质量（HRQoL）方面较 HELP 进一步改善；48.9%的滚动入组患者达到了先前定义的 6 分最小临床重要差异。非滚动入组患者（n=81）报告的变化为-19.5（21.3）。研究结束时，90.2%的滚动入组患者和 95.9%的非滚动入组患者报告疾病得到控制（血管性水肿控制测试总分为≥10）。78.7%的患者和 82.4%的研究者报告了极好的治疗反应。其他 PROs 的结果表明焦虑略有改善，对治疗的满意度很高，工作效率或活动能力提高。
结论
长期拉那芦单抗治疗表现出 HRQoL 的临床意义上的改善，支持与预防发作相关的拉那芦单抗治疗的益处。
试验注册
ClinicalTrials.gov 标识符：NCT02586805（HELP 研究）和 NCT02741596（HELP 开放性延伸）。
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