Khanna Dinesh, Furst Daniel E, Clements Philip J, Park Grace S, Hays Ron D, Yoon Jeonglim, Korn Joseph H, Merkel Peter A, Rothfield Naomi, Wigley Fredrick M, Moreland Larry W, Silver Richard, Steen Virginia D, Weisman Michael, Mayes Maureen D, Collier David H, Medsger Thomas A, Seibold James R
Division of Rheumatology, Department of Medicine, David Geffen School of Medicine, Los Angeles, California, USA.
J Rheumatol. 2005 May;32(5):832-40.
This study compares the responsiveness to change of the Medical Outcomes Study Short Form Health Survey (SF-36), a measure of health related quality of life (HRQOL), and the Health Assessment Questionnaire Disability Index (HAQ-DI), a function instrument, in a randomized clinical trial for treatment of systemic sclerosis (SSc).
A phase 2/3, multicenter, prospective, placebo controlled trial was conducted to evaluate human recombinant relaxin treatment in patients with diffuse SSc over 24 weeks. At baseline, subjects had stable, moderately severe, diffuse SSc of disease duration < or = 5 years, modified Rodnan skin score > or = 20, serum creatinine < 2.0 mg/dl, percentage forced vital capacity (% FVC) predicted > or = 50%, and % DLCO predicted > or = 40% and were not receiving concomitant disease modifying therapies. Internal consistency reliability of multi-item scales was estimated using Cronbach's alpha. Responsiveness to change of the SF-36 and HAQ-DI was computed between Weeks 0 and 24. Subjects were classified as unchanged or having a meaningful change in 4 different external measures: Change in (1) skin score > or = 30%; (2) % FVC predicted of > or = 15%; (3) self-reported patient global assessment by visual analog scale (VAS) > or = 20%; and (4) physician global assessment by VAS of > or = 20%. Responsiveness indices were computed and Cohen's effect size criteria were used to assess the magnitude of change.
A total of 239 patients participated in this trial, with 196 completing the 24 week trial. Cronbach's alpha for the SF-36 scales ranged from 0.76 to 0.93 and for the HAQ-DI ranged from 0.69 to 0.91 (good to excellent). The SF-36 had a larger magnitude of responsiveness in overall disease (patient and physician global assessment) compared to the HAQ-DI, while the HAQ-DI had a larger magnitude of responsiveness in clinical measures (i.e., change in skin score and % FVC predicted) than the SF-36.
These data support inclusion of both the SF-36 and HAQ-DI as outcome measures in future clinical trials of diffuse SSc.
本研究在一项治疗系统性硬化症（SSc）的随机临床试验中，比较健康相关生活质量（HRQOL）测量工具医学结局研究简明健康调查（SF - 36）和功能评估工具健康评估问卷残疾指数（HAQ - DI）对变化的反应性。
开展一项2/3期、多中心、前瞻性、安慰剂对照试验，以评估人重组松弛素对弥漫性SSc患者超过24周的治疗效果。基线时，受试者患有病程＜或 = 5年的稳定、中度严重弥漫性SSc，改良Rodnan皮肤评分＞或 = 20，血清肌酐＜2.0 mg/dl，预计用力肺活量百分比（%FVC）＞或 = 50%，预计一氧化碳弥散量百分比（%DLCO）＞或 = 40%，且未接受伴随的疾病改善治疗。使用Cronbach's α估计多项目量表的内部一致性信度。计算第0周和第24周之间SF - 36和HAQ - DI对变化的反应性。受试者被分类为无变化或在4种不同外部测量中有有意义的变化：（1）皮肤评分变化＞或 = 30%；（2）预计%FVC变化＞或 = 15%；（3）通过视觉模拟量表（VAS）自我报告的患者整体评估变化＞或 = 20%；（4）通过VAS的医生整体评估变化＞或 = 20%。计算反应性指数，并使用Cohen效应量标准评估变化程度。
共有239名患者参与本试验，196名完成了24周试验。SF - 36量表的Cronbach's α范围为0.76至0.93，HAQ - DI的范围为0.69至0.91（良好至优秀）。与HAQ - DI相比，SF - 36在整体疾病（患者和医生整体评估）中的反应性幅度更大，而HAQ - DI在临床测量（即皮肤评分变化和预计%FVC变化）中的反应性幅度比SF - 36更大。
这些数据支持在未来弥漫性SSc的临床试验中纳入SF - 36和HAQ - DI作为结局指标。
