Roth Michael D, Tseng Chi-Hong, Clements Philip J, Furst Daniel E, Tashkin Donald P, Goldin Jonathan G, Khanna Dinesh, Kleerup Eric C, Li Ning, Elashoff David, Elashoff Robert M
Department of Medicine, David Geffen School of Medicine, University of California, Los Angeles, CA 90095-1690, USA.
Arthritis Rheum. 2011 Sep;63(9):2797-808. doi: 10.1002/art.30438.
To identify baseline characteristics of patients with scleroderma-related interstitial lung disease (SSc-ILD) that could serve as predictors of the most favorable response to 12-month treatment with oral cyclophosphamide (CYC).
Regression analyses were retrospectively applied to the Scleroderma Lung Study data in order to identify baseline characteristics that correlated with the absolute change in forced vital capacity (FVC) (% predicted values) and the placebo-adjusted change in % predicted FVC over time (the CYC treatment effect).
Completion of the CYC arm of the Scleroderma Lung Study was associated with a placebo-adjusted improvement in the % predicted FVC of 2.11% at 12 months, which increased to 4.16% when patients were followed up for another 6 months (P=0.014). Multivariate regression analyses identified the maximal severity of reticular infiltrates (assessed as maximum fibrosis scores) on high-resolution computed tomography (HRCT) at baseline, the modified Rodnan skin thickness score (MRSS) at baseline, and the Mahler baseline dyspnea index as independent correlates of treatment response. When patients were stratified on the basis of whether 50% or more of any lung zone was involved by reticular infiltrates on HRCT and/or whether patients exhibited an MRSS of at least 23, a subgroup of patients emerged in whom there was an average CYC treatment effect of 9.81% at 18 months (P<0.001). Conversely, there was no treatment effect (a -0.58% difference) in patients with less severe HRCT findings and a lower MRSS at baseline.
A retrospective analysis of the Scleroderma Lung Study data identified the severity of reticular infiltrates on baseline HRCT and the baseline MRSS as patient features that might be predictive of responsiveness to CYC therapy.
确定硬皮病相关间质性肺病（SSc-ILD）患者的基线特征，这些特征可作为口服环磷酰胺（CYC）治疗12个月时最有利反应的预测指标。
对硬皮病肺部研究数据进行回顾性回归分析，以确定与用力肺活量（FVC）的绝对变化（预测值百分比）以及随时间推移预测FVC百分比的安慰剂调整变化（CYC治疗效果）相关的基线特征。
硬皮病肺部研究中CYC组的完成与12个月时预测FVC百分比的安慰剂调整改善2.11%相关，当患者再随访6个月时，该改善增加至4.16%（P=0.014）。多变量回归分析确定基线高分辨率计算机断层扫描（HRCT）上网状浸润的最大严重程度（评估为最大纤维化评分）、基线改良Rodnan皮肤厚度评分（MRSS）和Mahler基线呼吸困难指数为治疗反应的独立相关因素。当根据HRCT上任何肺区是否有50%或更多被网状浸润累及和/或患者是否表现出至少23的MRSS对患者进行分层时，出现了一个亚组患者，其在18个月时CYC平均治疗效果为9.81%（P<0.001）。相反，基线HRCT表现较轻且MRSS较低的患者没有治疗效果（差异为-0.58%）。
对硬皮病肺部研究数据的回顾性分析确定基线HRCT上网状浸润的严重程度和基线MRSS为可能预测对CYC治疗反应性的患者特征。
