Lainka Elke, Baehr Melanie, Raszka Bernadette, Haas Johannes-Peter, Hügle Boris, Fischer Nadine, Foell Dirk, Hinze Claas, Weissbarth-Riedel Elisabeth, Kallinich Tilmann, Horneff Gerd, Windschall Daniel, Lilienthal Eggert, Niehues Tim, Neudorf Ulrich, Berendes Rainer, Küster Rolf-Michael, Oommen Prasad Thomas, Rietschel Christoph, Lutz Thomas, Weller-Heinemann Frank, Tenbrock Klaus, Heubner Georg Leonhard, Klotsche Jens, Wittkowski Helmut
Department of Pediatric Rheumatology, University Children's Hospital Essen, Essen, Germany.
German Center for Pediatric and Adolescent Rheumatology, Garmisch-Partenkirchen, Germany.
Pediatr Rheumatol Online J. 2021 Mar 22;19(1):38. doi: 10.1186/s12969-021-00510-8.
Systemic juvenile idiopathic arthritis (sJIA) is a complex disease with dysregulation of the innate immune system driven by cytokines. A major role is ascribed to interleukin-1β (IL-1β), supporting the autoinflammatory character of the disease and offering an effective blocking mechanism for treatment. Here we present clinical practice data from the German AID-registry for patients treated with IL-1 inhibition (IL-1i).
In 2009 a clinical and research consortium (AID-Net) was established, including an online AID-registry. Patients with documented sJIA diagnosis were identified. Data for this retrospective IL-1i study were recorded by 17 centers. Response to treatment was evaluated according to Wallace criteria and additionally by an own classifying clinical response system.
In 6 years, 202 patients with confirmed sJIA were recorded in the AID-registry. Out of these, 111 children received therapy with Anakinra (ANA) (n = 84, 39 f) and/or Canakinumab (CANA) (n = 27, 15 f) at a median age of 8.7 y (range 0.6-19.1). During the first 12 months 75/111 (ANA 55, CANA 20) patients were evaluated according to Wallace criteria (achievement of inactive disease 28/55 and 17/20, remission over 6 months under medication 13/55 and 7/20 cases). Over the whole period of time, clinical response was preserved in the majority of patients (ANA 54/80, CANA 20/27). Arthritis mostly persisted in polyarticular (PA) courses. During treatment with IL-1i concomitant medication could be tapered in about 15%. IL-1i was discontinued in 59/111 patients. 45 (15) adverse events (AE)s in ANA (CANA) treated patients (19.7 (26.6) AE/100 ANA (CANA) exposure years, 95%CI: 14.4-26.4 (14.9-43.9)) were reported.
In a large cohort of sJIA patients from Germany, we can confirm an overall favorable clinical response to both available IL-1 blocking agents. IL-1i was well tolerated with acceptable safety and effectiveness in a real-life clinical setting.
全身型幼年特发性关节炎（sJIA）是一种复杂疾病，细胞因子驱动先天性免疫系统失调。白细胞介素-1β（IL-1β）起主要作用，这支持了该疾病的自身炎症特征，并为治疗提供了一种有效的阻断机制。在此，我们展示了来自德国AID注册中心接受IL-1抑制（IL-1i）治疗患者的临床实践数据。
2009年成立了一个临床和研究联盟（AID-Net），包括一个在线AID注册中心。确定有记录的sJIA诊断患者。这项回顾性IL-1i研究的数据由17个中心记录。根据华莱士标准评估治疗反应，并另外通过一个自行分类的临床反应系统进行评估。
6年间，AID注册中心记录了202例确诊的sJIA患者。其中，111名儿童接受了阿那白滞素（ANA）（n = 84，39名女性）和/或卡那单抗（CANA）（n = 27，15名女性）治疗，中位年龄为8.7岁（范围0.6 - 19.1岁）。在最初12个月期间，75/111（ANA 55例，CANA 20例）患者根据华莱士标准进行评估（达到疾病非活动状态28/55例和17/20例，用药6个月以上缓解13/55例和7/20例）。在整个时间段内，大多数患者（ANA 54/80例，CANA 20/27例）保持临床反应。关节炎大多在多关节型（PA）病程中持续存在。在IL-1i治疗期间，约15%的患者可逐渐减少联合用药。111例患者中有59例停用IL-1i。报告了ANA（CANA）治疗患者中的45（15）例不良事件（AE）（19.7（26.6）例AE/100 ANA（CANA）暴露年，95%置信区间：14.4 - 26.4（14.9 - 43.9））。
在来自德国的一大群sJIA患者中，我们可以证实两种可用的IL-1阻断剂总体临床反应良好。在现实临床环境中，IL-1i耐受性良好，安全性和有效性可接受。
