Division of Immunology and Rheumatology, Stanford School of Medicine, Stanford, CA, USA.
Division of Immunology and Rheumatology, Stanford School of Medicine, 1000 Welch RD #203, Palo Alto, CA, 94304, USA.
Arthritis Res Ther. 2018 Mar 23;20(1):57. doi: 10.1186/s13075-018-1541-z.
The aim was to assess patient-reported outcomes (PROs) in patients with rheumatoid arthritis (RA) treated with filgotinib during two phase 2b, 24-week, randomized, placebo-controlled studies.
Patients with moderate-to-severe active RA and an inadequate response to methotrexate (MTX) were randomized to daily placebo or filgotinib 50 mg, 100 mg, or 200 mg as add-on therapy to MTX (NCT01888874) or as monotherapy (NCT01894516). At week 12, nonresponders receiving filgotinib 50 mg in both studies or placebo in the add-on study, and all patients receiving placebo as monotherapy, were re-assigned to filgotinib 100 mg. PROs were measured using the Health Assessment Questionnaire - Disability Index (HAQ-DI) including Patient Pain assessed by visual analog scale, and the Patient Global Assessment of Disease Activity (Patient Global), the Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue Scale (Version 4), and the 36-Item Short Form Health Survey (SF-36).
At week 12, improvements in all PROs, apart from the SF-36 mental component in the add-on study, were statistically better with filgotinib than placebo; some improvements were noted as early as the first assessment time point (week 1 or week 4). Filgotinib improved HAQ-DI by 0.58-0.84 points, FACIT-Fatigue by 6.9-11.4 points, Patient Global by 25.2-35.6 mm, and Pain by 24.2-37.9 mm; scores were maintained or improved to week 24. Across all PROs, more patients achieved minimal clinically important differences and normative values with filgotinib 200 mg than placebo. Patients re-assigned to filgotinib 100 mg at week 12 experienced improvements in PROs between weeks 12 to 24.
Filgotinib as MTX add-on therapy or as monotherapy demonstrated rapid and sustained (to 24 weeks) improvements in health-related quality of life and functional status in patients with active RA.
MTX add-on study: ClinicalTrials.gov , NCT01888874 . Registered on 28 June 2013. Monotherapy study: ClinicalTrials.gov , NCT01894516 . Registered on 10 July 2013.
本研究旨在评估 filgotinib 治疗中重度活动性类风湿关节炎（RA）患者的患者报告结局（PRO），试验包含两项为期 24 周的 2b 期、随机、安慰剂对照研究。
对接受甲氨蝶呤（MTX）治疗但应答不足的中重度活动性 RA 患者进行随机分组，分别接受每日安慰剂或 filgotinib 50mg、100mg、200mg 作为 MTX 的附加治疗（NCT01888874）或单药治疗（NCT01894516）。在第 12 周，两项研究中接受 filgotinib 50mg 的无应答者和附加研究中接受安慰剂的无应答者，以及所有接受安慰剂单药治疗的患者，均重新分配至 filgotinib 100mg。采用健康评估问卷残疾指数（HAQ-DI）、视觉模拟量表评估的患者疼痛（Patient Pain）、患者总体疾病活动度评估（Patient Global）、慢性病治疗功能评估-疲劳量表（FACIT-Fatigue Scale，第 4 版）和 36 项简明健康调查问卷（SF-36）测量 PRO。
在第 12 周，与安慰剂相比，filgotinib 治疗在所有 PRO 上均有统计学意义的改善，除了附加研究中的 SF-36 精神成分；有些改善早在首次评估时间点（第 1 周或第 4 周）就观察到。Filgotinib 可使 HAQ-DI 改善 0.58-0.84 分、FACIT-Fatigue 改善 6.9-11.4 分、患者总体改善 25.2-35.6mm 和疼痛改善 24.2-37.9mm；在第 24 周时，评分仍保持或改善。在所有 PRO 中，与安慰剂相比，接受 filgotinib 200mg 治疗的患者达到最小临床重要差异和正常值的比例更高。在第 12 周重新分配至 filgotinib 100mg 的患者，在第 12 周至 24 周之间 PRO 得到改善。
作为 MTX 的附加治疗或单药治疗，filgotinib 可快速持续（至 24 周）改善活动性 RA 患者的健康相关生活质量和功能状态。
MTX 附加治疗研究：ClinicalTrials.gov ，NCT01888874。2013 年 6 月 28 日注册。单药治疗研究：ClinicalTrials.gov ，NCT01894516。2013 年 7 月 10 日注册。
