Pediatric Rheumatology Department, APHP, Bicêtre Hospital, University of Paris Saclay, Kremlin Bicêtre, France.
CEREMAIA (French reference center for auto-inflammatory diseases and inflammatory amyloidosis), RITA network member, Paris, France.
Arthritis Res Ther. 2024 Apr 8;26(1):80. doi: 10.1186/s13075-024-03316-7.
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.
本研究旨在提供有关卡那单抗在法国家族性地中海热(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)中的治疗模式、疗效和安全性的真实世界证据。
本研究使用了 JIR 队列,这是一个于 2013 年创建的多中心国际注册中心,用于收集青少年炎症性风湿病患者的数据。本研究纳入了在法国被诊断为 FMF、MKD 或 TRAPS 并接受卡那单抗治疗的患者。
在研究期间,31 名 FMF 患者、26 名 MKD 患者和 7 名 TRAPS 患者接受了卡那单抗治疗。他们中的大多数人以推荐剂量 2 毫克/公斤或 150 毫克开始使用卡那单抗,但不到一半的 FMF 和 MKD 患者按推荐频率(每 4 周)开始使用。在开始治疗后 2 年,仍有 78.1%的 FMF 患者、73.7%的 MKD 患者和 85.7%的 TRAPS 患者继续接受治疗。虽然在治疗过程中每次注射的剂量保持不变,但观察到剂量间隔的一些调整。有 6 名患者报告发生严重不良事件。其中,3 例可能与卡那单抗有关。
这项中期分析显示,在开始使用卡那单抗治疗 2 年后,治疗的维持情况良好,并证实了其在法国 FMF、MKD 和 TRAPS 患者真实世界实践中的安全性。在接受卡那单抗治疗的患者中观察到剂量和间隔组合的高度多样性,这表明医生根据个体情况调整剂量,而不是采用固定的治疗方案。