López-Medina Clementina, Capelusnik Dafne, Webers Casper, Van den Bosch Filip, Boonen Annelies, Carron Philippe, Molto Anna, Ramiro Sofia
Rheumatology, Reina Sofia University Hospital, Cordoba, Spain
GC05, Maimonides Biomedical Research Institute of Cordoba (IMIBIC), Cordoba, Spain.
RMD Open. 2025 Jul 15;11(3):e005525. doi: 10.1136/rmdopen-2025-005525.
Unravelling the performance of disease activity measures in peripheral spondyloarthritis (pSpA) is crucial for the development of clinical studies. We aimed to evaluate the construct validity and discriminatory capacity of various instruments assessing disease activity and response criteria in patients with pSpA.
Post-hoc analysis of the CRESPA randomised controlled trial including patients with early active pSpA. Patients were randomised to golimumab (GOL) or placebo (PBO). Data of the placebo-controlled part until week 12 were used. Construct validity (known group discrimination assessed with standardised mean difference, SMD) in the 12-week data, longitudinal construct validity (ie, standardised response mean and effect size) and trial discrimination (SMD) of several disease activity instruments were assessed. As part of trial discrimination, a χ test was performed for binary outcomes.
A total of 60 patients (40 GOL, 20 PBO) were included. Construct validity was better for composite outcomes (ie, Disease Activity in Psoriatic Arthritis (DAPSA), Bath Ankylosing Spondylitis Disease Activity Index (BASDAI) and Axial Spondyloarthritis Disease Activity Score (ASDAS)-higher SMD between active and inactive patients). Longitudinal construct validity was consistently good for composite outcomes (eg, ASDAS, DAPSA) and global assessments (Patient Global Assessment (PGA), Physician Global Assessment (PhGA)). Clinical trial discrimination was good for composites (BASDAI, ASDAS, DAPSA), global assessments (PGA, PhGA) and joint counts (swollen joint count (SJC66) and tender joint count (TJC68)).Among binary outcomes, trial discrimination was strongest for clinical remission (ie, absence of arthritis, enthesitis and dactylitis), BASDAI50, DAPSA-Low Disease Activity (LDA) and ASDAS-LDA.
While both composite and global outcome measurement instruments performed well in pSpA, composite scores like DAPSA, ASDAS and BASDAI showed better construct validity. The clinical remission definition was the most discriminatory response criterion.
了解外周型脊柱关节炎（pSpA）疾病活动度测量指标的性能对于临床研究的开展至关重要。我们旨在评估评估pSpA患者疾病活动度和反应标准的各种工具的结构效度和区分能力。
对包括早期活动性pSpA患者的CRESPA随机对照试验进行事后分析。患者被随机分为戈利木单抗（GOL）组或安慰剂（PBO）组。使用安慰剂对照部分至第12周的数据。评估了12周数据中的结构效度（用标准化均值差，SMD评估已知组区分度）、纵向结构效度（即标准化反应均值和效应量）以及几种疾病活动度工具的试验区分度。作为试验区分度的一部分，对二元结局进行了χ检验。
共纳入60例患者（40例GOL组，20例PBO组）。复合结局（即银屑病关节炎疾病活动度（DAPSA）、巴斯强直性脊柱炎疾病活动指数（BASDAI）和轴性脊柱关节炎疾病活动评分（ASDAS）——活动和非活动患者之间的SMD更高）的结构效度更好。复合结局（如ASDAS、DAPSA）和整体评估（患者整体评估（PGA）、医生整体评估（PhGA））的纵向结构效度一直良好。临床试验区分度在复合指标（BASDAI、ASDAS、DAPSA）、整体评估（PGA、PhGA）和关节计数（肿胀关节计数（SJC66）和压痛关节计数（TJC68））方面表现良好。在二元结局中，试验区分度在临床缓解（即无关节炎、附着点炎和指（趾）炎）、BASDAI50、DAPSA-低疾病活动度（LDA）和ASDAS-LDA方面最强。
虽然复合指标和整体结局测量工具在pSpA中均表现良好，但DAPSA、ASDAS和BASDAI等复合评分显示出更好的结构效度。临床缓解定义是最具区分性的反应标准。
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