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.
Ann Allergy Asthma Immunol. 2023 Jul;131(1):101-108.e3. doi: 10.1016/j.anai.2023.03.028. Epub 2023 Apr 5.
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).
To measure the impact of long-term lanadelumab treatment on patient-reported outcomes (PROs).
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.
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.
Clinically meaningful improvement in HRQoL was exhibited with long-term lanadelumab treatment, supporting the benefit of lanadelumab therapy associated with attack prevention.
ClinicalTrials.gov Identifiers: NCT02586805 (HELP Study) and NCT02741596 (HELP open-label extension).
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。
衡量长期拉那芦单抗治疗对患者报告结局 (PRO) 的影响。
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。
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。
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。
ClinicalTrials.gov 标识符：NCT02586805（HELP 研究）和 NCT02741596（HELP 开放性延伸）。
