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Limited Utility of Rapamycin in Severe, Refractory Wegener's Granulomatosis CURRY L. KOENING, JOSÉ HERNÁNDEZ-RODRÍGUEZ, EAMONN S. MOLLOY, TIFFANY M. CLARK, and GARY S. HOFFMAN
ABSTRACT. Methods. Eight patients received rapamycin for severe, refractory WG. Clinical outcomes were reviewed retrospectively. Results. Four patients treated with rapamycin sustained remissions of at least 6 months' duration while receiving prednisone ¾ 10 mg/day; 2 relapsed during followup. Five patients discontinued rapamycin due to continued disease activity, cancer, or adverse events. Conclusion. Toxicities due to rapamycin were numerous; consistent proof of efficacy in this pilot experience with WG was not seen. (First Release Nov 15 2008; doi:10.3899/jrheum.080664) Key Indexing Terms:
WEGENER'S GRANULOMATOSIS From the Cleveland Clinic, Center for Vasculitis Care and Research, Department of Rheumatic and Immunologic Diseases, Cleveland, Ohio; and Division of Rheumatology, University of Utah, Salt Lake City, Utah, USA. Dr. Koening was supported by The Cleveland Clinic Center for Vasculitis Care and Research and by the Vasculitis Clinical Research Consortium, Rare Diseases Clinical Research Network, National Institutes of Health (1 U54 RR019497-01). Dr. Hernández-Rodríguez was supported by a research award from Hospital Clínic of Barcelona, Spain, and by the R.J. Fasenmyer Center for Clinical Immunology at the Cleveland Clinic. Dr. Molloy was supported by the R.J. Fasenmyer Center for Clinical Immunology at the Cleveland Clinic and the Vasculitis Foundation. C.L. Koening, MD, MS, Division of Rheumatology, University of Utah; J. Hernández-Rodríguez, MD, Department of Systemic Autoimmune Diseases, Hospital Clínic of Barcelona, Barcelona, Spain; E.S. Molloy, MD, MS, Center for Vasculitis Care and Research, Cleveland Clinic; T.M. Clark, NP, Center for Vasculitis Care and Research, Cleveland Clinic; G.S. Hoffman, MD, MS, Harold C. Schott Professor of Medicine, Cleveland Clinic Lerner College of Medicine, Harold C. Schott Chair of Rheumatic and Immunologic Diseases. Address reprint requests to Dr. C.L. Koening, Division of Rheumatology, University of Utah, 4B200 School of Medicine, 30 North 1900 East, Salt Lake City, UT 84132-2404. E-mail: Curry.Koening@hsc.utah.edu Accepted for publication August 28, 2008.
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