Department of Immunology and Rheumatology, Division of Advanced Preventive Medical Sciences, Nagasaki University Graduate School of Biomedical Sciences, Nagasaki, Japan.
Center for Bioinformatics and Molecular Medicine, Nagasaki University Graduate School of Biomedical Sciences, Nagasaki, Japan.
Mod Rheumatol. 2022 Jul 1;32(4):797-802. doi: 10.1093/mr/roab048.
To evaluate the efficacy and safety of canakinumab in Japanese patients with colchicine-resistant or colchicine-intolerant familial Mediterranean fever (FMF) in a real-world clinical setting.
We reviewed 13 Japanese FMF patients to whom canakinumab was introduced during the period of October 2017 to December 2020. All patients were diagnosed as FMF according to Tel-Hashomer criteria. We performed genetic analyses for Mediterranean fever or MEFV by targeted next-generation sequencing. Efficacy was assessed by attack frequency and the percentage of patients who achieved attack improvement at 24 weeks. Safety was assessed by adverse events observed during canakinumab treatment.
The median duration and follow-up of canakinumab treatment were 13 and 16 months, respectively. The median attack frequency was 0.50 [0.30-1.00] at 24 weeks, which was a significant decrease from 2.00 [0.85-2.88] at the time of induction (p = .019). There were three patients (23%) with complete resolution of attacks at 24 weeks. No serious adverse events were observed. However, one patient had small intestinal ulceration which led to the discontinuation of canakinumab.
Although the number of cases is small, this study suggests that canakinumab is efficacious and safe for use in Japanese patients with colchicine-resistant or colchicine-intolerant FMF in a real-world clinical setting in Japan.
评估卡那奴单抗在日本秋水仙碱耐药或不耐受家族性地中海热（FMF）患者中的疗效和安全性。
我们回顾了 2017 年 10 月至 2020 年 12 月期间接受卡那奴单抗治疗的 13 例日本 FMF 患者。所有患者均根据 Tel-Hashomer 标准诊断为 FMF。我们通过靶向下一代测序对地中海热或 MEFV 进行基因分析。通过 24 周时的发作频率和实现发作改善的患者百分比评估疗效。通过卡那奴单抗治疗期间观察到的不良事件评估安全性。
卡那奴单抗治疗的中位持续时间和随访时间分别为 13 个月和 16 个月。24 周时的中位发作频率为 0.50 [0.30-1.00]，与诱导时的 2.00 [0.85-2.88]相比显著降低（p=0.019）。24 周时有 3 例患者（23%）发作完全缓解。未观察到严重不良事件。然而，有 1 例患者发生小肠溃疡，导致卡那奴单抗停药。
尽管病例数较少，但本研究表明，卡那奴单抗在日本真实临床环境中对于秋水仙碱耐药或不耐受 FMF 的日本患者是有效且安全的。
