Teitsma Xavier M, Jacobs Johannes W G, Welsing Paco M J, Pethö-Schramm Attila, Borm Michelle E A, Hendriks Lidy, Denissen Natasja H A M, van Laar Jacob M, Lafeber Floris P J G, Bijlsma Johannes W J
Department of Rheumatology & Clinical Immunology, University Medical Center Utrecht, Utrecht, the Netherlands.
F Hoffmann-La Roche, Basel, Switzerland.
Rheumatology (Oxford). 2017 Dec 1;56(12):2179-2189. doi: 10.1093/rheumatology/kex319.
To evaluate the effect of initiation of tocilizumab, with or without MTX, compared with MTX alone on patient-reported outcomes (PROs), in DMARD-naïve patients with early RA.
In U-Act-Early, patients initiated treat-to-target step-up MTX, tocilizumab or tocilizumab plus MTX therapy. PROs assessed included the Functional Assessment of Chronic Illness Therapy-Fatigue, 36-item Short Form (SF-36), five dimensional EuroQol (EQ-5D) and the Revised Illness Perception Questionnaire. Differences between strategy groups over time and proportions of patients exceeding minimum clinically important differences (MCID) were evaluated.
During the 2-year study period, significant improvements were found in the tocilizumab strategies in the SF-36 physical component score (tocilizumab, P = 0.012; tocilizumab plus MTX, P = 0.044) and EQ-5D score (tocilizumab plus MTX, P = 0.020) when compared with the MTX strategy. No significant differences were noted in other PROs (P ⩾ 0.052, except for the domain 'identity' in the Illness Perception Questionnaire; tocilizumab vs MTX, P = 0.048). The proportions of patients achieving MCID in SF-36 physical component score were significantly higher at 12 and 52 weeks (P ⩽ 0.049) in the tocilizumab arms when compared with the MTX arm. At week 24, the proportion achieving MCID in EQ-5D was significantly higher in the tocilizumab plus MTX arm vs the MTX arm (P = 0.045).
Initiation of treat-to-target tocilizumab therapy resulted in significantly improved PROs, especially within the first 24 weeks, when compared with initiation of MTX therapy. Also on the patients' level, initiating tocilizumab may be considered as a valuable strategy in DMARD-naïve patients with early RA.
ClinicalTrials.gov, http://clinicaltrials.gov, NCT01034137.
评估在初治的早期类风湿关节炎（RA）患者中，托珠单抗单药治疗或联合甲氨蝶呤（MTX）治疗与单纯MTX治疗相比，对患者报告结局（PROs）的影响。
在U-Act-Early研究中，患者开始接受目标导向的逐步增加剂量的MTX、托珠单抗或托珠单抗联合MTX治疗。评估的PROs包括慢性病治疗功能评估-疲劳量表、36项简明健康调查（SF-36）、五维度欧洲生活质量量表（EQ-5D）以及修订的疾病认知问卷。评估了不同治疗策略组随时间的差异以及超过最小临床重要差异（MCID）的患者比例。
在2年的研究期间，与MTX治疗策略相比，托珠单抗治疗策略在SF-36身体成分评分（托珠单抗，P = 0.012；托珠单抗联合MTX，P = 0.044）和EQ-5D评分（托珠单抗联合MTX，P = 0.020）方面有显著改善。在其他PROs方面未观察到显著差异（P⩾0.052，疾病认知问卷中的“身份认同”领域除外；托珠单抗与MTX相比，P = 0.048）。与MTX组相比，托珠单抗治疗组在第12周和第52周时，SF-36身体成分评分达到MCID的患者比例显著更高（P⩽0.049）。在第24周时，托珠单抗联合MTX组EQ-5D达到MCID的比例显著高于MTX组（P = 0.045）。
与MTX治疗相比，开始目标导向的托珠单抗治疗可显著改善PROs，尤其是在最初24周内。在患者层面，对于初治的早期RA患者，开始使用托珠单抗可被视为一种有价值的治疗策略。
ClinicalTrials.gov，http://clinicaltrials.gov，NCT01034137。
