Metroplex Clinical Research Center , Dallas , Texas , USA.
Biopharmaceutical Consultant , Portola Valley, California , USA.
RMD Open. 2016 Apr 26;2(1):e000232. doi: 10.1136/rmdopen-2015-000232. eCollection 2016.
To compare the relationship between clinical measures and patient-reported outcomes (PROs) in patients with rheumatoid arthritis (RA) treated with tofacitinib or methotrexate (MTX).
In a phase 3 randomised controlled trial, patients (N=956) who were MTX-naïve or had received ≤3 doses were randomised and received tofacitinib 5 or 10 mg twice daily or MTX titrated to 20 mg/week. Outcomes included: per cent of patients achieving American College of Rheumatology 70% responses (ACR70), ACR50, low disease activity (LDA) by Simplified Disease Activity Index (SDAI ≤11) and Clinical Disease Activity Index (CDAI ≤10), remission by SDAI (≤3.3) and CDAI (≤2.8), patient-reported Health Assessment Questionnaire-Disability Index (HAQ-DI scores <0.5), pain and global assessment of disease activity.
At month 6, most patients who achieved LDA/remission by one definition achieved LDA/remission with others; however, discordance between measures was greater with MTX than with tofacitinib. As expected, concordance between CDAI and SDAI responses was high. Overall, patients achieving LDA or ACR50 responses reported less improvement in PROs (HAQ-DI, pain and patient global assessment) compared with clinical measures (tender and swollen joint counts).
Variability in levels of responses between clinical outcomes and PROs should be considered when setting treat-to-target goals in patients with RA.
NCT01039688; Post-results.
比较类风湿关节炎（RA）患者接受托法替尼或甲氨蝶呤（MTX）治疗时临床指标与患者报告结局（PRO）的关系。
在一项 3 期随机对照试验中，MTX 初治或接受 ≤3 剂 MTX 的患者被随机分组，接受托法替尼 5 或 10mg，每日 2 次，或 MTX 滴定至 20mg/周。主要结局包括：达到美国风湿病学会 70%反应（ACR70）、50%反应（ACR50）、简化疾病活动指数（SDAI≤11）和临床疾病活动指数（CDAI≤10）的患者比例、SDAI 缓解（≤3.3）和 CDAI 缓解（≤2.8）的患者比例、健康评估问卷残疾指数（HAQ-DI 评分<0.5）、疼痛和疾病活动度的患者报告。
在第 6 个月，通过一种定义达到低疾病活动/缓解的大多数患者，通过其他定义也达到低疾病活动/缓解；然而，MTX 组与托法替尼组相比，各指标之间的不一致性更大。与 SDAI 相比，CDAI 与 SDAI 反应的一致性更高。总体而言，与临床指标（压痛和肿胀关节计数）相比，达到低疾病活动或 ACR50 反应的患者报告的 PRO（HAQ-DI、疼痛和患者整体评估）改善较小。
在设定 RA 患者的治疗目标时，应考虑临床结局和 PRO 之间反应水平的差异。
NCT01039688；研究结果注册后。
