Initial pilot studies with thalidomide in MMM were dose escalating, starting with 100 mg of thalidomide per day32-40 (see Table 49.2). The published trials of thalidomide as single-agent therapy in MMM had several interesting findings. First, the activity observed in these trials was mainly manifest in the improvement of cytopenias (anemia and thrombocytopenia), with less frequent improvement in splenomegaly. In addition, patients with MMM did poorly with dose escalation, and in fact did not tolerate the agent well even at doses of 200 mg/day of thalidomide. Finally, a subset of MMM patients can experience unwanted myelopro-liferation with thalidomide therapy.43 Interestingly, myeloproliferation has even been observed in patients without prior identified myeloproliferative disorders.44 In excess of 100 patients with MMM have been reported in the literature to have received thalidomide as single-agent therapy for their disease. These trials (outlined in Table 49.2) demonstrate that thalidomide does appear to be reproducibly active in improving anemia and thrombocytopenia in approximately 30-40% of patients, with more modest relief from symptomatic hepatosplenomegaly. Subsequent trials using low-dose thalidomide (50 mg/day) appear to retain the activity but with less toxicity (see Table 49.2). Additionally, the combination of 50 mg/day of thalidomide with a corticosteroid taper41 has resulted in improvements in the toxicity profile, decreases in toxicity dropout, and equivalent if not superior efficacy. Responses, when obtained, to thalidomide can be durable even after discontinuation of the drug. In a recent report45 of long-term outcomes with thalidomide among initial responders, 35% (7 of 20 patients)

Published clinical trials of thalidomide in CIMF



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