Department of Medicine A, Sheba Medical Center, Tel Hashomer, Israel.
Sackler Faculty of Medicine, Tel Aviv University, Israel.
Rheumatology (Oxford). 2021 Jun 18;60(6):2878-2883. doi: 10.1093/rheumatology/keaa728.
FMF is an autoinflammatory disease of genetic origin. Colchicine is the mainstay of treatment for the prevention of attacks and long-term complications but 5-10% of FMF patients are resistant to colchicine therapy. The aim of our study was to investigate the real-life safety and efficacy of anakinra in a cohort of patients with colchicine-resistant FMF.
In this retrospective study, patients treated with anakinra for colchicine-resistant FMF between 2010 and 2018 were identified using the computerized database of Sheba Medical Center and enrolled in the study. Data from structured clinical files were analysed to evaluate the efficacy and safety outcomes. To assess efficacy, we used the Global Assessment Score (GAS), a measure comprised of three different domains: number of attacks per month, duration of attacks and number of sites involved in the attacks. Reported adverse events were compiled.
A total of 44 patients (24 female) were treated with anakinra. Of these patients, 75% were homozygous for the M649V mutation. The mean duration of treatment was 18 months. The GAS decreased significantly from 6.6 (IQR 5.3-7.8) before treatment to 2 (IQR 0-4.2) while on treatment (P < 0.001). During anakinra treatment, six hospitalizations were reported (three due to related adverse effects). In addition, 11 patients suffered from injection site reactions (5 ceased treatment). Twelve patients reported mild side effects.
Treatment with anakinra is beneficial for the majority of colchicine-resistant FMF patients and is relatively safe.
家族性地中海热（FMF）是一种遗传性自身炎症性疾病。秋水仙碱是预防发作和长期并发症的主要治疗药物，但 5-10%的 FMF 患者对秋水仙碱治疗有耐药性。我们研究的目的是研究在一组对秋水仙碱耐药的 FMF 患者中，使用阿那白滞素的真实安全性和疗效。
在这项回顾性研究中，我们使用 Sheba 医疗中心的计算机数据库确定了接受阿那白滞素治疗的对秋水仙碱耐药的 FMF 患者，并将其纳入研究。分析了结构化临床档案中的数据，以评估疗效和安全性结果。为了评估疗效，我们使用了综合评估评分（GAS），这是一个由三个不同的领域组成的衡量标准：每月发作次数、发作持续时间和发作涉及的部位数量。报告了不良事件。
共有 44 名患者（24 名女性）接受了阿那白滞素治疗。其中，75%的患者为 M649V 突变的纯合子。平均治疗时间为 18 个月。GAS 从治疗前的 6.6（IQR 5.3-7.8）显著下降到治疗时的 2（IQR 0-4.2）（P<0.001）。在阿那白滞素治疗期间，报告了 6 例住院治疗（其中 3 例因相关不良反应住院）。此外，11 名患者出现注射部位反应（5 名患者停止治疗）。12 名患者报告有轻度副作用。
阿那白滞素治疗对大多数对秋水仙碱耐药的 FMF 患者是有益的，且相对安全。
