Furst Daniel, Khanna Dinesh, Matucci-Cerinic Marco, Clements Philip, Steen Virginia, Pope Janet, Merkel Peter, Foeldvari Ivan, Seibold James, Pittrow David, Polisson Richard, Strand Vibeke
University of California at Los Angeles, Los Angeles, California 90025, USA.
J Rheumatol. 2007 May;34(5):1194-200.
Few randomized controlled trials (RCT) have shown a demonstrable treatment effect in systemic sclerosis (SSc), making it difficult to evaluate outcome measures in this disease indication. Results from recent RCT, including those evaluating cyclophosphamide for SSc interstitial lung disease and endothelin receptor antagonists for pulmonary hypertension, have allowed analysis of certain organ-specific endpoints using the OMERACT filter. An earlier metaanalysis established that skin score, measures of Raynaud's, pulmonary function tests, blood pressure, pain, Health Assessment Questionnaire, and Medical Outcomes Survey Short-Form 36 are validated outcome measures in SSc. At OMERACT 8, data regarding validation of high-resolution computed tomography of the lungs, 6-minute walk test, and patient reported outcomes in SSc were presented. A Delphi exercise to develop consensus regarding a combined set of noninvasive measures for pulmonary arterial hypertension (PAH) is under way. Given the protean nature of this illness and its multiorgan system involvement, a composite responder index may be preferable. Another Delphi exercise is designed to develop consensus regarding a combined SSc response index to be validated in future RCT.
很少有随机对照试验（RCT）在系统性硬化症（SSc）中显示出可证实的治疗效果，这使得评估该疾病适应症的疗效指标变得困难。近期RCT的结果，包括那些评估环磷酰胺治疗SSc间质性肺病以及内皮素受体拮抗剂治疗肺动脉高压的试验，使得能够使用OMERACT标准对某些器官特异性终点进行分析。一项较早的荟萃分析确定，皮肤评分、雷诺现象测量、肺功能测试、血压、疼痛、健康评估问卷以及医学结局调查简表36是SSc中经过验证的疗效指标。在OMERACT 8会议上，展示了关于肺部高分辨率计算机断层扫描、6分钟步行试验以及SSc患者报告结局验证的数据。一项关于就一套用于肺动脉高压（PAH）的联合非侵入性测量方法达成共识的德尔菲法正在进行。鉴于这种疾病的多变性质及其多器官系统受累情况，综合反应指数可能更为可取。另一项德尔菲法旨在就一套将在未来RCT中进行验证的联合SSc反应指数达成共识。
