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  • Title: Limited utility of rapamycin in severe, refractory Wegener's granulomatosis.
    Author: Koening CL, Hernández-Rodríguez J, Molloy ES, Clark TM, Hoffman GS.
    Journal: J Rheumatol; 2009 Jan; 36(1):116-9. PubMed ID: 19012360.
    Abstract:
    OBJECTIVE: We report our experience using rapamycin in patients with Wegener's granulomatosis (WG) who failed to achieve remission with conventional treatment. 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<or=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.
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