• Arthritis and rheumatism · Sep 2011

    Randomized Controlled Trial Multicenter Study

    Predicting treatment outcomes and responder subsets in scleroderma-related interstitial lung disease.

    • Michael D Roth, Chi-Hong Tseng, Philip J Clements, Daniel E Furst, Donald P Tashkin, Jonathan G Goldin, Dinesh Khanna, Eric C Kleerup, Ning Li, David Elashoff, Robert M Elashoff, and Scleroderma Lung Study Research Group.
    • Department of Medicine, David Geffen School of Medicine, University of California, Los Angeles, CA 90095-1690, USA. mroth@mednet.ucla.edu
    • Arthritis Rheum. 2011 Sep 1; 63 (9): 2797-808.

    ObjectiveTo 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).MethodsRegression 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).ResultsCompletion 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.ConclusionA 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.Copyright © 2011 by the American College of Rheumatology.

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