Yao Wo, Diao Ran, Yang Boyun, Wang Yongfang, Li Bohui, Li Ting, Ge Liuya, Yu Yongmei, Zhu Rongfei, Wang Huiying
Department of Allergy, Second Affiliated Hospital of Zhejiang University School of Medicine, Hangzhou, China,
Department of Allergy, Second Affiliated Hospital of Zhejiang University School of Medicine, Hangzhou, China.
Int Arch Allergy Immunol. 2025;186(3):221-231. doi: 10.1159/000541242. Epub 2024 Oct 3.
Hereditary angioedema (HAE) is a rare genetic disorder caused by deficiency or dysfunction of C1-esterase inhibitor that is characterized by recurrent episodes of bradykinin-mediated edema. Lanadelumab has been the only available first-line therapy for long-term prophylaxis (LTP) of HAE in China since its approval in 2020. The present study aimed to investigate the clinical efficacy and safety of lanadelumab for LTP in Chinese patients.
A retrospective clinical data were collected for the 6 patients and used to examine the frequency of attack symptoms, disease-related loss of work days, and quality of life before and after LTP with lanadelumab. Health-related quality of life was assessed using the Dermatology Life Quality Index (DLQI) and the Angioedema Quality of Life Questionnaire (AE-QoL).
Lanadelumab led to reductions of 97.8% and 98.5% in the attack rate and treated attack rate, respectively. All patients exhibited significant improvements in AE-QoL and DLQI scores (100% reduction rates) during the early treatment period (4 weeks and 2 weeks, respectively) and in missed work days/year (98.9% reduction rate). The efficacy of lanadelumab remained stable during COVID-19 vaccination and infection. No serious/severe treatment-emergent adverse events occurred during lanadelumab treatment.
This study is the first report that demonstrates the clinical efficacy of lanadelumab and safety of LTP in HAE patients from Chinese mainland. A reasonable dosage plan can ensure a quick and long-lasting protective role of lanadelumab against HAE attacks, during COVID-19 pandemic period.
遗传性血管性水肿（HAE）是一种罕见的遗传性疾病，由C1酯酶抑制剂缺乏或功能障碍引起，其特征为缓激肽介导的水肿反复发作。自2020年获批以来，拉那度单抗一直是中国唯一可用于HAE长期预防（LTP）的一线治疗药物。本研究旨在探讨拉那度单抗对中国大陆HAE患者LTP的临床疗效和安全性。
收集6例患者的回顾性临床数据，用于检查使用拉那度单抗进行LTP前后的发作症状频率、与疾病相关的工作日损失以及生活质量。使用皮肤病生活质量指数（DLQI）和血管性水肿生活质量问卷（AE-QoL）评估与健康相关的生活质量。
拉那度单抗使发作率和治疗发作率分别降低了97.8%和98.5%。所有患者在治疗早期（分别为4周和2周）的AE-QoL和DLQI评分（降低率均为100%）以及每年旷工天数（降低率为98.9%）均有显著改善。在新冠病毒疫苗接种和感染期间，拉那度单抗的疗效保持稳定。在拉那度单抗治疗期间未发生严重/重度治疗中出现的不良事件。
本研究是首份证明拉那度单抗对中国大陆HAE患者LTP的临床疗效和安全性的报告。合理的给药方案可确保拉那度单抗在新冠疫情期间对HAE发作起到快速且持久的保护作用。
