Haslak Fatih, Oner Nimet, Elhani Inès, Hinze Tanja, Mamutova Anna, Bourguiba Rim, Kasap Cuceoglu Muserref, Pateras Konstantinos, Aviel Yonatan Butbul, Delplanque Marion, Caorsi Roberta, Šestan Mario, Bénard Stéphanie Ducharme, Brunner Jürgen, El Moussaoui Majdouline, Kirijas Meri, Constantin Tamas, Arenas Sonia Carriquí, Khellaf Ghalia, Guliyeva Vafa, Assalia Naiera, Backes Stefan, Sozeri Betul, Hofer Michaël, Ayaz Nuray, Lachmann Helen, Wittkowski Helmut, Hentgen Véronique
Department of Pediatric Rheumatology, Istanbul University-Cerrahpasa Cerrahpasa Faculty of Medicine, Fatih, Turkey
Department of Pediatric Rheumatology, Istanbul Medeniyet University Faculty of Medicine, Üsküdar, Turkey.
RMD Open. 2025 Sep 26;11(3):e006097. doi: 10.1136/rmdopen-2025-006097.
Although colchicine is the mainstay of familial Mediterranean fever (FMF) treatment, 5-10% of patients are considered to have colchicine resistance (CR). However, there is no globally agreed CR definition or indications for biological disease-modifying anti-rheumatic drugs (bDMARDs).
A survey on 'Biologics in Monogenic Autoinflammatory Diseases', part of the 'Clinical Practice Strategies' (CLiPS) initiative, was conducted by a JIR cohort-initiated eCOST network among expert participants worldwide. Our primary aim was to provide a flowchart reflecting the different CR definitions and present data regarding bDMARD indications. The secondary aim was to determine how specific biases influence clinical approaches. We analysed the CliPS according to the experience levels of physicians, country-specific FMF prevalence, countries' gross domestic product, bDMARD availability and reimbursement policies of the countries.
A total of 223 responses from 46 countries were included in the study. Almost half of the respondents (73/160, 45.6%) indicated that three to four attacks within the preceding 6 months were necessary for their CR definition. The most frequently used acute-phase reactant was C-reactive protein (157/164, 95.7%). Almost three-fourths of the respondents (74%, n=165) considered that supplementary factors, including complications of FMF, attack severity, elevated activity scores, patient-reported outcome and quality of life scales, influenced their CR definition.
We present a novel flowchart describing physicians' general attitudes and unique findings regarding management strategies for colchicine-resistant FMF and shifting trends influenced by epidemiological and socioeconomic factors.
尽管秋水仙碱是家族性地中海热（FMF）治疗的主要药物，但5%-10%的患者被认为存在秋水仙碱抵抗（CR）。然而，目前尚无全球公认的CR定义或生物性疾病改善抗风湿药物（bDMARDs）的使用指征。
“临床实践策略”（CLiPS）倡议的一部分——“单基因自身炎症性疾病中的生物制剂”调查，由一个JIR队列发起的eCOST网络在全球专家参与者中开展。我们的主要目的是提供一个反映不同CR定义的流程图，并展示有关bDMARDs使用指征的数据。次要目的是确定特定偏差如何影响临床方法。我们根据医生的经验水平、各国FMF的患病率、各国的国内生产总值、bDMARDs的可及性以及各国的报销政策对CLiPS进行了分析。
该研究共纳入了来自46个国家的223份回复。几乎一半的受访者（73/160，45.6%）表示，他们对CR的定义需要在前6个月内发生三到四次发作。最常使用的急性期反应物是C反应蛋白（157/164，95.7%）。近四分之三的受访者（74%，n=165）认为，包括FMF并发症、发作严重程度、活动评分升高、患者报告的结果和生活质量量表在内的补充因素会影响他们对CR的定义。
我们提出了一个新颖的流程图，描述了医生对秋水仙碱抵抗性FMF管理策略的总体态度和独特发现，以及受流行病学和社会经济因素影响的变化趋势。
