Immunosuppressive agents such as cyclophosphamide have long been used to treat autoimmune disease, but the dose is often limited by bone marrow suppression. In a randomized placebo-controlled trial, those with scleroderma-related alveolitis and interstitial lung disease who received Cytoxan for one year had better forced vital capacity than patients on placebo, a difference still seen at two-year follow-up. In addition, patients who had taken Cytoxan had modest improvement of dyspnea, skin thickening, functional ability, and some health-related quality-of-life measures. In an accompanying NEJM editorial, Fernando J. Martinez, M.D., and W. Joseph McCune, M.D., of the University of Michigan Health System in Ann Arbor, commented that “this well-designed trial will be regarded as a sentinel study confirming a beneficial response to cyclophosphamide in highly selected patients with scleroderma-related interstitial lung disease.”
Tashkin DP et al. “Cyclophosphamide versus Placebo in Scleroderma Lung Disease.” N Engl J Med 2006;354:2655-66.
Martinez FJ and McCune WJ. “Cyclophosphamide for Scleroderma Lung Disease.” N Engl J Med 2006;354:2707-09.
Kowal-Bielecka, O, Landewe, R, Avouac, J, Chwiesko, S, Miniati, I, Czirjak, L, Clements, P, Denton, C, Farge, D, Fligelstone, K, Foldvari, I, Furst, D E, Muller-Ladner, U, Seibold, J, Silver, R M, Takehara, K, Garay Toth, B, Tyndall, A, Valentini, G, van den Hoogen, F, Wigley, F, Zulian, F, Matucci-Cerinic, M
EULAR recommendations for the treatment of systemic sclerosis: a report from the EULAR Scleroderma Trials and Research group (EUSTAR)
Ann Rheum Dis 2009 0: ard.2008.096677