Tofacitinib for the Treatment of Juvenile Idiopathic Arthritis: Patient-Reported Outcomes in a Phase 3, Randomized, Double-Blind, Placebo-Controlled Withdrawal Trial.

We evaluated the impact of tofacitinib on patient-reported outcomes (PROs) in patients with JIA.

我们评估了托法替尼对幼年特发性关节炎（JIA）患者自我报告结果（PROs）的影响。

Methods

This was a post hoc analysis of a phase 3, randomized, double-blind, placebo-controlledwithdrawal trial (NCT02592434) in patients with JIA.

这是一项事后分析，针对的是针对JIA患者的3期、随机、双盲、安慰剂对照的撤药试验（NCT02592434）。

In the open-label phase (part 1; weeks 0-18), patients received body weight-based doses of tofacitinib.

在开放标签阶段（第1部分；第0-18周），患者接受了基于体重的托法替尼剂量。

During the double-blindphase (part 2; weeks 18-44), responders (per JIA-American College of Rheumatology 30 response criteria) were randomized 1:1 to continue tofacitinib or switch to placebo for up to 26 weeks.

在双盲阶段（第2部分；第18-44周），根据JIA-美国风湿病学会30%的反应标准，反应者被随机分为1:1，继续接受托法替尼治疗或改用安慰剂，最长可达26周。

Assessed PROs included the validated parent and/or legal guardian versions of the Childhood Health Assessment Questionnaire for evaluation of disability, arthritis pain, overall well-being, and the Child Health Questionnaire (CHQ).

评估的患者报告结果（PROs）包括经过验证的父母和/或法定监护人的儿童健康评估问卷版本，用于评估残疾、关节炎疼痛、整体健康状况以及儿童健康问卷（CHQ）。

Results

Overall, 225 patients were enrolled and received open-label tofacitinib in part 1, and 173 patients were randomized in part 2.

总体而言，共有225名患者在第一部分接受了公开标签的托法替尼治疗，并且在第二部分有173名患者被随机分配。

During part 1, least-squares (LS) mean (SE) disability, arthritis pain, and overall well-being scores numerically improved from mean 1.04 (SE 0.05), mean 5.53 (SE 0.20), and mean 5.07 (SE 0.20) at baseline to mean 0.57 (SE 0.05), mean 2.46 (SE 0.18), and mean 2.47 (SE 0.18) at week 18, respectively.

在第一部分期间，从基线时的平均值1.04（标准误0.05）、平均值5.53（标准误0.20）和平均值5.07（标准误0.20）分别改善到第18周的平均值0.57（标准误0.05）、平均值2.46（标准误0.18）和平均值2.47（标准误0.18），这些是最小二乘（LS）平均值（标准误）的残疾、关节痛和整体健康状况评分的数值改善。

LS mean (SE) CHQ physical summary and psychological summary scores numerically improved from mean 29.51 (SE 1.15) and mean 47.24 (SE 0.85) at baseline to mean 42.70 (SE 0.98) and mean 51.53 (SE 0.80) at week 18, respectively.

LS平均值（SE）CHQ身体总结和心理总结评分从基线的平均值29.51（SE 1.15）和平均值47.24（SE 0.85）分别提高到第18周的平均值42.70（SE 0.98）和平均值51.53（SE 0.80）。

Improvements were generally maintained to week 44 in part 2.

在第二部分，改善通常维持到第44周。

Conclusions

Tofacitinib improved a range of PROs in patients with JIA, suggesting potential HRQoL benefits.

托法替尼改善了JIA患者的多种患者报告结果(PROs)，表明可能对健康相关生活质量(HRQoL)有益。