Prediction of improvement in skin fibrosis in diffuse cutaneous systemic sclerosis: a EUSTAR analysis
Prediction of improvement in skin fibrosis in diffuse cutaneous systemic sclerosis: a EUSTAR analysis
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DOI：
10.1136/annrheumdis-2015-208024
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发表时间：
2016-10-01
影响因子：
27.4
通讯作者：
Distler, Oliver
中科院分区：
文献类型：
--
作者：
Dobrota, Rucsandra;Maurer, Britta;Distler, Oliver
目的皮肤纤维化的改善是弥漫性皮肤系统性硬化症(DcSSc)自然病程的一部分。认识到那些最有可能好转的患者可能有助于为临床试验量身定做临床管理和队列充实。在这项研究中，我们旨在确定dcSSc患者皮肤纤维化改善的预测因素。方法我们对欧洲硬皮病试验和研究(EUSTAR)登记的dcSSc患者进行了纵向分析，包括符合美国风湿病学会标准的dcSSc患者，基线改良Rodnan皮肤评分(MRSS)7分和122个月的随访MRSS。主要结果是皮肤改善(MRSS分别下降5分和25%)。MRSS的相应增加被认为是进步。通过专家意见选择皮肤改善的候选预测因素，并应用Logistic回归和Bootstrap验证。结果在919例患者中，218例(24%)改善，95例(10%)进展。分析了11个皮肤改善的候选预测因素。最终的模型确定高基线MRSS和无肌腱摩擦摩擦是皮肤改善的独立预测因素。基线MRSS是皮肤改善的最强预测因子，与病程无关。在18和25之间的上限阈值在增加进展者方面比退化者表现得最好。结论：在基线水平上，晚期皮肤纤维化且没有肌腱摩擦摩擦的患者比轻度皮肤纤维化的患者更有可能在未来一年内退化。这些基于证据的数据可以在临床试验设计中实施，以最大限度地减少纳入在护理标准下病情恶化的患者。
Objectives Improvement of skin fibrosis is part of the natural course of diffuse cutaneous systemic sclerosis (dcSSc). Recognising those patients most likely to improve could help tailoring clinical management and cohort enrichment for clinical trials. In this study, we aimed to identify predictors for improvement of skin fibrosis in patients with dcSSc.Methods We performed a longitudinal analysis of the European Scleroderma Trials And Research (EUSTAR) registry including patients with dcSSc, fulfilling American College of Rheumatology criteria, baseline modified Rodnan skin score (mRSS) 7 and follow-up mRSS at 122months. The primary outcome was skin improvement (decrease in mRSS of >5 points and 25%) at 1year follow-up. A respective increase in mRSS was considered progression. Candidate predictors for skin improvement were selected by expert opinion and logistic regression with bootstrap validation was applied.Results From the 919 patients included, 218 (24%) improved and 95 (10%) progressed. Eleven candidate predictors for skin improvement were analysed. The final model identified high baseline mRSS and absence of tendon friction rubs as independent predictors of skin improvement. The baseline mRSS was the strongest predictor of skin improvement, independent of disease duration. An upper threshold between 18 and 25 performed best in enriching for progressors over regressors.Conclusions Patients with advanced skin fibrosis at baseline and absence of tendon friction rubs are more likely to regress in the next year than patients with milder skin fibrosis. These evidence-based data can be implemented in clinical trial design to minimise the inclusion of patients who would regress under standard of care.