Division of Pediatric Rheumatology, Department of Pediatrics and Autoinflammation Reference Center, Tuebingen, University Hospital Tuebingen, Germany.
Patient Access Services, Novartis Healthcare Pvt. Ltd, Hyderabad, India.
Rheumatology (Oxford). 2020 Oct 1;59(10):2711-2724. doi: 10.1093/rheumatology/keaa205.
To identify and summarize the existing evidence on the efficacy, effectiveness and safety of biologic therapies used, either as indicated or off-label, in the treatment of FMF.
A systematic literature review was conducted using Embase®, MEDLINE®, MEDLINE®-In Process, and Cochrane databases to identify randomized/non-randomized controlled trials (RCTs/non-RCTs) and real-world observational studies of FMF published as full-text articles (2000-September 2017) or conference abstracts (2014-September 2017). Studies with data for ≥1 biologic were included. Studies with <5 patients were excluded.
Of the 3342 retrieved records, 67 publications, yielding 38 unique studies, were included. All studies were published after the year 2010, and the majority (21) were full-text articles. Most studies (33/38) were prospective/retrospective observational; three were double-blind, placebo-controlled RCTs (one each of anakinra, canakinumab and rilonacept); and two were non-RCTs (both canakinumab). Anakinra (26), canakinumab (21) and etanercept (6) were the most frequently used biologics across studies, whereas use of adalimumab, tocilizumab, rilonacept and infliximab was limited (1-2 studies). The available evidence suggested benefits of anakinra and canakinumab in FMF.
Anti-IL-1 therapies (i.e. anakinra and canakinumab) appear to be effective and safe options in the treatment of overall FMF, including patients with colchicine resistance and FMF-related amyloidosis. There is a need for properly designed prospective or controlled studies to conclude the superiority of one anti-IL-1 therapy over another. Evidence on the use of TNF-α and IL-6 inhibitors is limited, and further research is suggested.
确定并总结生物疗法在治疗纤维肌痛综合征（FMF）中的疗效、有效性和安全性的现有证据，包括适应证内和适应证外使用的生物疗法。
系统检索 Embase、MEDLINE、MEDLINE 在线和 Cochrane 数据库，以获取 2000 年 9 月至 2017 年发表的全文文章或 2014 年 9 月至 2017 年会议摘要中关于 FMF 的随机/非随机对照试验（RCT/非 RCT）和真实世界观察性研究。纳入至少有 1 种生物制剂数据的研究。排除患者数<5 的研究。
在检索到的 3342 条记录中，有 67 篇出版物，共 38 项研究纳入分析。所有研究均发表于 2010 年以后，其中 21 篇为全文文章。大多数研究（33/38）为前瞻性/回顾性观察性研究；3 项为双盲、安慰剂对照 RCT（分别为阿那白滞素、卡那奴单抗和瑞立奈普特）；2 项为非 RCT（均为卡那奴单抗）。阿那白滞素（26 项研究）、卡那奴单抗（21 项研究）和依那西普（6 项研究）是各研究中最常使用的生物制剂，而阿达木单抗、托珠单抗、瑞立奈普特和英夫利昔单抗的使用则较为局限（各 1-2 项研究）。现有证据表明，阿那白滞素和卡那奴单抗治疗 FMF 有效且安全。
抗白细胞介素-1 治疗（即阿那白滞素和卡那奴单抗）似乎是治疗整体 FMF 的有效且安全的选择，包括对秋水仙碱耐药和 FMF 相关淀粉样变性的患者。需要设计适当的前瞻性或对照研究来确定一种抗白细胞介素-1 疗法是否优于另一种。抗肿瘤坏死因子-α 和白细胞介素-6 抑制剂的证据有限，建议进一步研究。
