From the Università degli Studi di Genova, and the Istituto Giannina Gaslini, Genoa; Fondazione Institute for Research and Health Care (IRCCS) Ca' Granda, Ospedale Maggiore Policlinico, Milan, Italy; Instituto de Criança - Faculty of Medicine of the University of São Paulo (FMUSP), São Paulo, Brazil.
B. Saccomanno, MD, Research Fellow, Università degli Studi di Genova; J. Tibaldi, MD, PhD Student, Università degli Studi di Genova; F. Minoia, MD, Dirigente Medico, Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico; F. Bagnasco, PhD, Biostatistician, Istituto Giannina Gaslini; A. Pistorio, MD, PhD, Dirigente Medico, Istituto Giannina Gaslini; A. Guariento, MD, Research Fellow, Istituto Giannina Gaslini, and Instituto de Criança - FMUSP; R. Caorsi, MD, Dirigente Medico, Istituto Giannina Gaslini; A. Consolaro, MD, PhD, Assistant Professor, Università degli Studi di Genova and Istituto Giannina Gaslini; M. Gattorno, MD, Dirigente Medico, Istituto Giannina Gaslini; A. Ravelli, MD, Professor of Pediatrics, Università degli Studi di Genova and Istituto Giannina Gaslini.
J Rheumatol. 2019 Apr;46(4):416-421. doi: 10.3899/jrheum.180331. Epub 2019 Jan 15.
To seek predictors of therapeutic response to the interleukin (IL)-1 inhibitor anakinra in children with systemic-onset juvenile idiopathic arthritis (sJIA).
The clinical charts of all patients with sJIA who were newly treated with anakinra at our center between 2004 and 2017 were reviewed retrospectively. Predictors included baseline demographic, clinical, and laboratory variables as well as previous or concomitant therapies. The effectiveness of anakinra was assessed at 1 year after treatment start. Complete clinical response (CCR) was defined as absence of fever, physician's global assessment ≤ 1, count of active joints ≤ 1, negative C-reactive protein, and ≥ 75% reduction of corticosteroid dose. According to the intention-to-treat principle, patients who had anakinra discontinued before 1 year for any reasons other than disease remission were classified as nonresponders. Statistics included univariate and multivariable analyses.
Of the 62 patients included in the study, 24 (39%) met the criteria for CCR at 1 year, whereas 38 (61%) did not. On multivariable analysis, independent correlations with achievement of CCR were identified for shorter disease duration, lower active joint count, higher ferritin level, and greater activity of systemic manifestations. The area under the curve of the model was 0.83.
Our findings help to delineate the clinical profile of patients with sJIA who are more likely to benefit from IL-1 blockade. They also underscore the need for studies aimed at examining the therapeutic role of early IL-1 inhibition and to identify biomarkers predicting response to either IL-1 or IL-6 antagonists.
寻找白细胞介素 (IL)-1 抑制剂阿那白滞素治疗儿童全身型幼年特发性关节炎 (sJIA) 疗效的预测因素。
回顾性分析 2004 年至 2017 年期间在我中心新接受阿那白滞素治疗的所有 sJIA 患者的临床病历。预测因素包括基线人口统计学、临床和实验室变量以及先前或同时进行的治疗。在治疗开始后 1 年评估阿那白滞素的疗效。完全临床缓解 (CCR) 的定义为无发热、医生整体评估≤1、活跃关节数≤1、C 反应蛋白阴性和皮质类固醇剂量减少≥75%。根据意向治疗原则,因疾病缓解以外的任何原因在 1 年前停止使用阿那白滞素的患者被归类为无反应者。统计学分析包括单变量和多变量分析。
在纳入研究的 62 名患者中,24 名 (39%)在 1 年内达到 CCR 标准,而 38 名 (61%)未达到。多变量分析显示,达到 CCR 的独立相关因素为疾病持续时间较短、活跃关节计数较低、铁蛋白水平较高和全身表现活动度较大。该模型的曲线下面积为 0.83。
我们的研究结果有助于描绘更有可能从 IL-1 阻断中获益的 sJIA 患者的临床特征。它们还强调了需要进行研究,以检查早期 IL-1 抑制的治疗作用,并确定预测对 IL-1 或 IL-6 拮抗剂反应的生物标志物。