General Pediatrics, Versailles Hospital, Versailles, France.
CEREMAIA (French reference center for auto-inflammatory diseases and inflammatory amyloidosis), Kremlin-Bicêtre, France.
Front Immunol. 2021 Nov 11;12:744780. doi: 10.3389/fimmu.2021.744780. eCollection 2021.
Interleukin (IL)-1 inhibitors represent the main treatment in patients with colchicine-resistant/intolerant familial Mediterranean fever (crFMF), mevalonate kinase deficiency (MKD), and tumor necrosis factor receptor-associated periodic syndrome (TRAPS). However, the reasons for the use of IL-1 inhibitors in these diseases are still not completely clarified.
Identify real-life situations that led to initiating anakinra or canakinumab treatment in hereditary recurrent fevers (HRFs), combining data from an international registry and an up-to-date literature review.
Data were extracted from the JIRcohort, in which clinical information (demographic data, treatment, disease activity, and quality of life) on patients with FMF, MKD, and TRAPS was retrospectively collected. A literature search was conducted using Medline, EMBASE, and Cochrane databases.
Complete data of 93 patients with HRF (53.8% FMF, 31.2% MKD, and 15.1% TRAPS) were analyzed. Data from both the registry and the literature review confirmed that the main reasons for use of IL-1 blockers were the following: failure of previous treatment (n = 57, 61.3% and n = 964, 75.3%, respectively), persistence of disease activity with frequent attacks (n = 44, 47.3% and n = 1,023, 79.9%) and/or uncontrolled inflammatory syndrome (n = 46, 49.5% and n = 398, 31.1%), severe disease complication or associated comorbidities (n = 38, 40.9% and n = 390, 30.4%), and worsening of patients' quality of life (n = 36, 38.7% and n = 100, 7,8%). No reasons were specified for 12 (16.4%) JIRcohort patients and 154 (12%) patients in the literature.
In the absence of standardized indications for IL-1 inhibitors in crFMF, MKD, and TRAPS, these results could serve as a basis for developing a treat-to-target strategy that would help clinicians codify the therapeutic escalation with IL-1 inhibitors.
白细胞介素（IL）-1 抑制剂是治疗秋水仙碱耐药/不耐受家族性地中海热（crFMF）、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）患者的主要药物。然而，这些疾病中使用 IL-1 抑制剂的原因仍未完全阐明。
结合国际注册中心的数据和最新的文献综述，确定导致遗传性复发性发热（HRF）患者开始使用阿那白滞素或卡那单抗治疗的真实情况。
从 JIRcohort 中提取数据，该队列中回顾性收集了 FMF、MKD 和 TRAPS 患者的临床信息（人口统计学数据、治疗、疾病活动度和生活质量）。使用 Medline、EMBASE 和 Cochrane 数据库进行文献检索。
分析了 93 例 HRF 患者（53.8% FMF、31.2% MKD 和 15.1% TRAPS）的完整数据。来自注册中心和文献综述的数据均证实，使用 IL-1 阻滞剂的主要原因如下：先前治疗失败（n=57，61.3%和 n=964，75.3%）、疾病活动度持续存在且频繁发作（n=44，47.3%和 n=1023，79.9%）和/或炎症综合征未得到控制（n=46，49.5%和 n=398，31.1%）、严重疾病并发症或合并症（n=38，40.9%和 n=390，30.4%）以及患者生活质量恶化（n=36，38.7%和 n=100，7.8%）。JIRcohort 中有 12 名（16.4%）患者和文献中有 154 名（12%）患者未指明原因。
在 crFMF、MKD 和 TRAPS 中没有 IL-1 抑制剂的标准化适应证的情况下，这些结果可以为制定靶向治疗策略提供依据，帮助临床医生规范使用 IL-1 抑制剂进行治疗升级。
