Ross Laura, Nikpour Mandana
Department of Medicine at St Vincent's Hospital, University of Melbourne.
Department of Rheumatology, St Vincent's Hospital Melbourne, Melbourne, Victoria, Australia.
Curr Opin Rheumatol. 2023 Nov 1;35(6):334-340. doi: 10.1097/BOR.0000000000000971. Epub 2023 Aug 29.
The aim of this study was to summarize the composite outcome measures available to assess disease damage, severity and treatment response in systemic sclerosis (SSc).
There have been many recent efforts to rigorously develop and validate composite outcome measures to assess the totality of the impact of SSc on patients. All initiatives have used both consensus and data-driven methods. Recently developed are a SSc-specific damage index, a patient-reported disease impact questionnaire (ScleroID) and a treatment response index (ACR-CRISS).
Multiorgan, composite measures are being increasingly applied to assess treatment efficacy in clinical trials. Although a fully validated, disease-specific composite measure is not yet available, there has been significant recent progress towards developing measures of treatment response, damage and overall impact of SSc for application in randomized controlled trials.
本研究旨在总结可用于评估系统性硬化症（SSc）疾病损害、严重程度及治疗反应的综合结局指标。
近期人们为严格制定和验证综合结局指标付出诸多努力，以评估SSc对患者的整体影响。所有举措均采用了共识法和数据驱动法。最近开发的有SSc特异性损害指数、患者报告的疾病影响问卷（ScleroID）以及治疗反应指数（ACR-CRISS）。
多器官综合指标在临床试验中越来越多地用于评估治疗效果。虽然尚未有经过充分验证的、针对该疾病的综合指标，但近期在开发用于随机对照试验的SSc治疗反应、损害及总体影响指标方面取得了重大进展。
