Rheumatology & Clinical Immunology, University Medical Center Utrecht, G02.230, P.O. Box 85500, 3508GA, Utrecht, The Netherlands.
Rheumatology, Máxima Medical Center, Eindhoven, The Netherlands.
Trials. 2019 Apr 17;20(1):226. doi: 10.1186/s13063-019-3285-8.
Assessment of disease activity is a critical component of tight-control, treat-to-target treatment strategies of rheumatoid arthritis (RA). Recently, the HandScan has been validated as a novel method for objectively assessing RA disease activity in only 1.5 min, using optical spectral transmission (OST) in hands and wrists. We describe the protocol of a randomized controlled clinical trial (RCT) to investigate whether HandScan-guided treatment aimed at 'HandScan remission' (HandScan arm) is at least as effective as and more cost-effective than clinically guided treatment aimed at ACR/EULAR 2011 Boolean remission (DAS arm).
METHODS/DESIGN: The study is a multi-center, double-blind, non-inferiority RCT of 18 months duration. Patients ≥ 18 years with newly diagnosed, disease-modifying antirheumatic drug (DMARD)-naïve RA according to the ACR 2010 classification criteria, will be randomized to the DAS arm or the HandScan arm. The efficacy of the arms will be compared by evaluating Health Assessment Questionnaire (HAQ) scores (primary outcome) after 18 months of DMARD therapy, aimed at remission. The equivalence margin in HAQ scores between study arms is 0.2. Secondary outcomes are differences in cost-effectiveness and radiographic joint damage between treatment arms. The non-inferiority sample size calculation to obtain a power of 80% at a one-sided p value of 0.05, with 10% dropouts, resulted in 61 patients per arm. In both arms, DMARD strategy will be intensified monthly according to predefined steps until remission is achieved; in both arms DMARDs and treatment steps are identical. If sustained remission, defined as remission that persists consistently over three consecutive months, is achieved, DMARD therapy will be tapered.
The study protocol and the specifically designed decision-making software application allow for implementation of this RCT. To test a novel method of assessing disease activity and comparing (cost-)effectiveness with the contemporary method in treat-to-target DMARD strategies in early RA patients.
Dutch Trial Register, NTR6388. Registered on 6 April 2017 ( NL50026.041.14 ). Protocol version 3.0, 19-01-2017.
疾病活动评估是类风湿关节炎（RA）紧密控制、达标治疗策略的关键组成部分。最近，HandScan 已被验证为一种新方法，可在 1.5 分钟内通过手部和腕部的光学光谱透射（OST）客观评估 RA 疾病活动。我们描述了一项随机对照临床试验（RCT）的方案，以研究 HandScan 指导的治疗是否至少与临床指导的治疗一样有效，并且更具成本效益，治疗目标是达到“HandScan 缓解”（HandScan 组），而临床指导的治疗目标是 ACR/EULAR 2011 年的布尔缓解（DAS 组）。
方法/设计：该研究是一项为期 18 个月的多中心、双盲、非劣效 RCT。根据 ACR 2010 分类标准，将≥18 岁的新诊断、疾病修饰抗风湿药物（DMARD）初治 RA 患者随机分配至 DAS 组或 HandScan 组。通过 18 个月的 DMARD 治疗评估健康评估问卷（HAQ）评分（主要结局）来比较手臂的疗效，旨在达到缓解。研究臂之间 HAQ 评分的等效性边界为 0.2。次要结局为治疗臂之间成本效益和放射学关节损伤的差异。为了在单侧 p 值为 0.05 时获得 80%的效能，计算了非劣效性样本量，每组有 61 名患者。在两组中，根据预定步骤每月强化 DMARD 策略，直至达到缓解；两组中 DMARD 和治疗步骤相同。如果达到持续缓解，定义为连续三个月持续缓解，DMARD 治疗将逐渐减少。
该研究方案和专门设计的决策软件应用程序允许实施这项 RCT。测试一种评估疾病活动的新方法，并比较（成本）效益与早期 RA 患者达标 DMARD 策略中的当代方法。
荷兰试验登记处，NTR6388。于 2017 年 4 月 6 日注册（NL50026.041.14）。方案版本 3.0，2017 年 1 月 19 日。
