Chlorambucil

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Abstract
To investigate the effectiveness of oral chlorambucil treatment in patients with dermatomyositis (DM) that is recalcitrant to treatment with prednisone and/or immunosuppressive agents. Five patients with recalcitrant DM were treated with oral chlorambucil, 4 mg/day, after discontinuation of the other immunosuppressive agent (azathioprine or methotrexate). Three patients were treated with a combination of prednisone and chlorambucil, and 2 with chlorambucil alone. Beneficial effects were noted within 4-6 weeks in all 5 patients, and corticosteroids were eventually discontinued in 4. Currently, only 1 patient is still receiving therapy; the other 4 have stopped chlorambucil after 13-30 months of treatment, and their disease remains in remission. Minimal chlorambucil toxicity was noted, consisting of leukopenia in 2 patients. Our findings suggest that chlorambucil is an effective, corticosteroid-sparing agent for the control of DM.

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