Hou Ling, Zhou Peng, Zhao Chengguang, Wang Xiuli, Du Yue
Department of Pediatrics, Shengjing Hospital of China Medical University, Shenyang, China.
Front Pediatr. 2025 Aug 20;13:1650675. doi: 10.3389/fped.2025.1650675. eCollection 2025.
To describe the efficacy and safety of tofacitinib for pediatric Still's disease, also referred to as systemic-onset juvenile idiopathic arthritis (sJIA). Traditional non-biological drugs and drugs targeting the interleukin-1 and interleukin-6 pathways benefit some patients, but others show inadequate responses.
We retrospectively analyzed ten patients with pediatric Still's disease who were treated with tofacitinib and had at least one follow-up visit. Data on patient history, laboratory findings, and treatments were collected at disease onset, at the initiation of tofacitinib, and during follow-up.
Tofacitinib led to complete remission in six patients, partial remission in three patients, and loss of efficacy in one patient. Among the nine patients with remission, two discontinued corticosteroids entirely and seven used lower dosages of corticosteroids; these patients also used fewer concurrent medications (1 or 2) after tofacitinib initiation. The one patient who experienced loss of efficacy continued to require a higher dosage of corticosteroids and received five different additional medications. The other nine patients received tocilizumab and had a decreased frequency of these injections after tofacitinib initiation. Tofacitinib was well-tolerated, with only one reported instance of an upper respiratory tract infection.
Tofacitinib appears to be an effective adjunct therapy for management of pediatric Still's disease, particularly for patients with unstable clinical conditions and adverse reactions to corticosteroids.
描述托法替布治疗儿童斯蒂尔病(也称为全身型幼年特发性关节炎,sJIA)的疗效和安全性。传统的非生物药物以及靶向白细胞介素 -1 和白细胞介素 -6 通路的药物使部分患者获益,但其他患者反应欠佳。
我们回顾性分析了 10 例接受托法替布治疗且至少有一次随访的儿童斯蒂尔病患者。在疾病发作时、开始使用托法替布时以及随访期间收集患者病史、实验室检查结果和治疗数据。
托法替布使 6 例患者完全缓解,3 例部分缓解,1 例出现疗效丧失。在 9 例缓解的患者中,2 例完全停用了皮质类固醇,7 例使用较低剂量的皮质类固醇;这些患者在开始使用托法替布后同时使用的药物也较少(1 种或 2 种)。1 例出现疗效丧失的患者继续需要较高剂量的皮质类固醇,并额外接受了 5 种不同药物治疗。其他 9 例患者接受了托珠单抗治疗,在开始使用托法替布后这些注射的频率降低。托法替布耐受性良好,仅报告了 1 例上呼吸道感染病例。
托法替布似乎是治疗儿童斯蒂尔病的一种有效辅助疗法,特别是对于临床病情不稳定且对皮质类固醇有不良反应的患者。