Patient-reported outcomes (PROs) are important when evaluating treatment benefits in rheumatoid arthritis (RA). We compared upadacitinib, an oral, selective JAK-1 inhibitor, with placebo to assess clinically meaningful improvements in PROs in patients with RA who have had inadequate responses to biologic disease-modifying antirheumatic drugs (bDMARD-IR). PRO responses between upadacitinib 15 mg or 30 mg and placebo were evaluated at week 12 from the SELECT-BEYOND trial. Improvement was determined by measuring Patient Global Assessment of Disease Activity (PtGA), pain, Health Assessment Questionnaire Disability Index (HAQ-DI), Short Form-36 Health Survey (SF-36), duration and severity of morning (AM) stiffness, and Insomnia Severity Index (ISI). Least squares mean changes and percentage of patients reporting improvements ≥ minimum clinically important differences (MCID) and scores greater than or equal to normative values were determined. The number needed to treat (NNT) to achieve clinically meaningful improvements was calculated. In 498 patients, both upadacitinib doses resulted in statistically significant changes from baseline versus placebo in PtGA, pain, HAQ-DI, SF-36 Physical Component Summary (PCS), 7 of 8 SF-36 domains (15 mg), 6 of 8 SF-36 domains (30 mg), and AM stiffness duration and severity. Compared with placebo, more upadacitinib-treated patients reported improvements ≥ MCID in PtGA, pain, HAQ-DI, SF-36 PCS, 7 of 8 SF-36 domains (15 mg), 5 of 8 SF-36 domains (30 mg), AM stiffness duration and severity, and ISI (30 mg) and scores ≥ normative values in HAQ-DI and SF-36 domains. Across most PROs, NNTs to achieve MCID with upadacitinib ranged from 4 to 7 patients. In bDMARD-IR RA patients, upadacitinib (15 mg or 30 mg) improved multiple aspects of quality of life, and more patients reached clinically meaningful improvements approaching normative values compared with placebo. The trial is registered with ClinicalTrials.gov (NCT02706847), registered 6 March 2016.

中文翻译：

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Upadacitinib对患者报告的结局的影响：SELECT-BEYOND的结果，这是类风湿性关节炎患者的3期随机试验，对改变生物性疾病的抗风湿药反应不足。

在评估类风湿关节炎（RA）的治疗益处时，患者报告的结局（PROs）很重要。我们将口服选择性JAK-1抑制剂upadacitinib与安慰剂进行了比较，以评估对改善生物疾病的抗风湿药（bDMARD-IR）反应不足的RA患者PROs的临床意义。SELECT-BEYOND试验在第12周评估了15 mg或30 mg upadacitinib与安慰剂之间的PRO反应。通过测量患者对疾病活动的总体评估（PtGA），疼痛，健康评估问卷残疾指数（HAQ-DI），36型健康调查简表（SF-36），早晨僵硬的持续时间和严重程度，和失眠严重程度指数（ISI）。最小二乘均值表示报告改善的患者的变化和百分比，≥最小临床重要差异（MCID），且得分大于或等于规范值。计算出实现临床上有意义的改善所需的治疗次数（NNT）。在498例患者中，两种upadacitinib剂量均导致PtGA，疼痛，HAQ-DI，SF-36物理成分摘要（PCS）相对于安慰剂在基线水平上有统计学上的显着变化，在8个SF-36域（15毫克）中占7个SF-36域（30 mg），以及AM僵硬持续时间和严重程度。与安慰剂相比，更多接受upadacitinib治疗的患者报告PtGA，疼痛，HAQ-DI，SF-36 PCS，8个SF-36域（15毫克）中的7个改善，≥8个SF-36域（30毫克）中≥MCID的改善，AM僵硬持续时间和严重程度，和ISI（30 mg），并且在HAQ-DI和SF-36域中的得分≥规范值。在大多数PRO中，使用upadacitinib实现MCID的NNT范围为4至7位患者。在bDMARD-IR RA患者中，upadacitinib（15 mg或30 mg）改善了生活质量的多个方面，并且与安慰剂相比，更多的患者达到了接近标准值的具有临床意义的改善。该试验已于2016年3月6日在ClinicalTrials.gov（NCT02706847）上进行了注册。