Division of Rheumatology and Department of Pediatrics, Cincinnati Children's Hospital Medical Center and University of Cincinnati College of Medicine, Cincinnati, Ohio, USA
Paediatric Rheumatology Service, The Royal Children's Hospital, Parkville, Victoria, Australia.
Ann Rheum Dis. 2024 Oct 21;83(11):1561-1571. doi: 10.1136/ard-2023-225094.
We report the safety, tolerability and efficacy of tofacitinib in patients with juvenile idiopathic arthritis (JIA) in an ongoing long-term extension (LTE) study.
Patients (2-<18 years) with JIA who completed phase 1/3 index studies or discontinued for reasons excluding treatment-related serious adverse events (AEs) entered the LTE study and received tofacitinib 5 mg two times per day or equivalent weight-based doses. Safety outcomes included AEs, serious AEs and AEs of special interest. Efficacy outcomes included improvement since tofacitinib initiation per the JIA-American College of Rheumatology (ACR)70/90 criteria, JIA flare rate and disease activity measured by Juvenile Arthritis Disease Activity Score (JADAS)27, with inactive disease corresponding to JADAS ≤1.0.
Of 225 patients with JIA (median (range) duration of treatment, 41.6 (1-103) months), 201 (89.3%) had AEs; 34 (15.1%) had serious AEs. 10 patients developed serious infections; three had herpes zoster. Two patients newly developed uveitis. Among patients with polyarticular course JIA, JIA-ACR70/90 response rates were 60.0% (78 of 130) and 33.6% (47 of 140), respectively, at month 1, and generally improved over time. JIA flare events generally occurred in <5% of patients through to month 48. Observed mean (SE) JADAS27 was 22.0 (0.6) at baseline, 6.2 (0.7) at month 1 and 2.8 (0.5) at month 48, with inactive disease in 28.8% (36 of 125) of patients at month 1 and 46.8% (29 of 82) at month 48.
In this interim analysis of LTE study data in patients with JIA, safety findings were consistent with the known profile of tofacitinib, and efficacy was maintained up to month 48.
NCT01500551.
我们报告托法替布在正在进行的长期扩展（LTE）研究中治疗幼年特发性关节炎（JIA）患者的安全性、耐受性和疗效。
完成了 I 期/3 期指数研究或因除治疗相关严重不良事件（AE）以外的原因停药的 JIA 患者（2-<18 岁）进入 LTE 研究并接受托法替布 5mg，每日两次或等效的基于体重的剂量。安全性结果包括 AE、严重 AE 和特别关注的 AE。疗效结果包括根据 JIA-美国风湿病学会（ACR）70/90 标准、JIA 发作率和用幼年特发性关节炎疾病活动度评分（JADAS）27 测量的疾病活动度评估的自托法替布起始的改善，无疾病活动对应 JADAS≤1.0。
225 例 JIA 患者（治疗中位（范围）时间，41.6（1-103）个月）中，201 例（89.3%）发生 AE；34 例（15.1%）发生严重 AE。10 例患者发生严重感染；3 例发生带状疱疹。2 例患者新发生葡萄膜炎。多关节炎病程 JIA 患者中，JIA-ACR70/90 应答率分别为 60.0%（78/130）和 33.6%（47/140），分别在第 1 个月和随后的时间有所改善。JIA 发作事件通常在第 48 个月前＜5%的患者中发生。观察到的平均（SE）JADAS27 在基线时为 22.0（0.6），第 1 个月时为 6.2（0.7），第 48 个月时为 2.8（0.5），第 1 个月时有 28.8%（36/125）的患者无疾病活动，第 48 个月时为 46.8%（29/82）。
在 JIA 的 LTE 研究数据的中期分析中，安全性发现与托法替布的已知特征一致，疗效在第 48 个月仍保持。
NCT01500551。
