Our study aimed to provide real-world evidence on the treatment patterns, effectiveness and safety of canakinumab in France in Familial Mediterranean Fever (FMF), Mevalonate Kinase Deficiency (MKD), and Tumor necrosis factor Receptor Associated Periodic Syndrome (TRAPS). This study used the JIR cohort, a multicentre international registry created in 2013 to collect data on patients with juvenile inflammatory rheumatic diseases. French patients diagnosed with FMF, MKD or TRAPS and treated with canakinumab were included in this study. 31 FMF, 26 MKD and 7 TRAPS patients received canakinumab during the study period. Most of them initiated canakinumab at the recommended dose of 2 mg/kg or 150 mg, but less than half of FMF and MKD patients initiated it at the recommended frequency (every 4 weeks). Two years after initiation, the rate of patients still on treatment was 78.1% in FMF, 73.7% in MKD, and 85.7% in TRAPS patients. While the dose per injection remained globally the same over the course of the treatment, some adjustments of the dose intervals were observed. Six patients had a severe adverse event reported. Of those, three were possibly related to canakinumab. This interim analysis showed a good maintenance of canakinumab treatment 2 years after initiation and confirmed its safety profile in real-life practice in France in patients diagnosed with FMF, MKD and TRAPS. The high variety of dose and interval combinations observed in canakinumab treated patients let suppose that physicians adapt the posology to individual situations rather than a fixed treatment plan.

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2009-2022 年卡那奴单抗治疗 3 种单基因周期性发热综合征的真实世界证据：使用法国 JIR 队列数据库的中期分析

我们的研究旨在提供关于法国卡那奴单抗治疗家族性地中海热 (FMF)、甲羟戊酸激酶缺乏症 (MKD) 和肿瘤坏死因子受体相关周期性综合征 (TRAPS) 的治疗模式、有效性和安全性的真实世界证据。这项研究使用了 JIR 队列，这是一个成立于 2013 年的多中心国际注册中心，旨在收集青少年炎症性风湿病患者的数据。本研究纳入了诊断为 FMF、MKD 或 TRAPS 并接受卡那奴单抗治疗的法国患者。 31 名 FMF、26 名 MKD 和 7 名 TRAPS 患者在研究期间接受了卡那奴单抗治疗。他们中的大多数人以 2 mg/kg 或 150 mg 的推荐剂量开始使用卡那单抗，但不到一半的 FMF 和 MKD 患者以推荐频率（每 4 周）开始使用。开始两年后，FMF 患者仍在接受治疗的患者比例为 78.1%，MKD 患者为 73.7%，TRAPS 患者为 85.7%。虽然每次注射的剂量在治疗过程中总体保持相同，但观察到剂量间隔的一些调整。六名患者报告了严重的不良事件。其中，三个可能与卡那单抗有关。这项中期分析显示，卡那奴单抗治疗在开始 2 年后得到了良好的维持，并证实了其在法国诊断为 FMF、MKD 和 TRAPS 的患者的现实实践中的安全性。在卡那单抗治疗的患者中观察到的多种剂量和间隔组合让我们假设医生会根据个人情况调整剂量，而不是固定的治疗计划。