遗传性血管性水肿的长期预防：加拉西单抗和拉那度单抗的初步经验。
Long-term prophylaxis for hereditary angioedema: Initial experiences with garadacimab and lanadelumab.
作者信息
Wong Jane C Y, Chiang Valerie, Lam Dorothy L Y, Lee Elaine, Lam Ki, Au Elaine Y L, Li Philip H
机构信息
Division of Rheumatology and Clinical Immunology, Department of Medicine, The University of Hong Kong, Hong Kong.
Division of Clinical Immunology, Department of Pathology, Queen Mary Hospital, Hong Kong.
出版信息
J Allergy Clin Immunol Glob. 2023 Aug 30;2(4):100166. doi: 10.1016/j.jacig.2023.100166. eCollection 2023 Nov.
BACKGROUND
With no approved long-term prophylaxis (LTP) for the prevention of hereditary angioedema (HAE) attacks in Hong Kong, patients rely on compassionate use programs and drug trials. Moreover, studies regarding the use and efficacy of LTP in Asia are lacking.
OBJECTIVES
Our aim was to assess 2 LTP medications for HAE in Hong Kong: lanadelumab and garadacimab.
METHODS
A prospective study was performed. Adult patients with a diagnosis of type I or type II HAE with 1 or more expert-confirmed attacks per month were consecutively recruited. The patients had been receiving treatment for at least 6 months. Clinical data were obtained, and questionnaires were administered before treatment periodically for at least 6 months following initiation of LTP.
RESULTS
Almost one-third of the patients with HAE experienced frequent attacks and began receiving LTP (8 of the 11 received garadacimab and 3 of the 11 received lanadelumab). At baseline, the time-normalized number of HAE attacks was 2.5 plus or minus 1.3 per month. At month 6, there was an overall reduction of time-normalized number of attacks per month of -2.4 attacks per month (95% CI = -3.3 to -1.5. [01]). The time-normalized number of HAE attacks at month 6 was 0.1 plus or minus 0.1 per month. More than 70% of the patients (8 of 11) were completely attack-free during the 6-month period while receiving LTP, and no patients required hospitalization. LTP improved patients' scores of the Angioedema Quality-of-Life Questionnaire ( .001) and reduced activity impairment due to health ( = .008). Patients experienced significant improvement across all dimensions of the Treatment Satisfaction for Medication Questionnaire (54.5%-76.8% [ = .002]), and no adverse events were reported.
CONCLUSION
The patients receiving LTP with garadacimab and lanadelumab experienced a significant reduction in number of HAE attacks and improvement in quality of life, and they were satisfied with treatment.
背景
在香港，尚无获批用于预防遗传性血管性水肿（HAE）发作的长期预防（LTP）药物，患者依赖于同情用药计划和药物试验。此外，亚洲地区关于LTP的使用和疗效的研究也很缺乏。
目的
我们的目的是评估香港用于HAE的两种LTP药物：lanadelumab和garadacimab。
方法
进行了一项前瞻性研究。连续招募每月有1次或更多次经专家确认发作的I型或II型HAE成年患者。这些患者已接受治疗至少6个月。获取临床数据，并在开始LTP后至少6个月内定期在治疗前进行问卷调查。
结果
几乎三分之一的HAE患者发作频繁并开始接受LTP（11例中有8例接受garadacimab，11例中有3例接受lanadelumab）。基线时，HAE发作的时间标准化次数为每月2.5±1.3次。在第6个月时，每月发作的时间标准化次数总体减少了-2.4次/月（95%CI=-3.3至-1.5[01]）。第6个月时HAE发作的时间标准化次数为每月0.1±0.1次。超过70%的患者（11例中有8例）在接受LTP的6个月期间完全无发作，且无患者需要住院治疗。LTP改善了患者的血管性水肿生活质量问卷得分（P<.001），并减少了因健康问题导致的活动障碍（P=.008）。患者在药物治疗满意度问卷的所有维度上均有显著改善（54.5%-76.8%[P=.002]），且未报告不良事件。
结论
接受garadacimab和lanadelumab进行LTP治疗的患者HAE发作次数显著减少，生活质量得到改善，且对治疗满意。
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