Thalidomide for treatment of patients with chronic graft-versus-host disease

In a randomized, placebo-controlled, double-blind trial, thalidomide or pla cebo together with glucocorticoids and either cyclosporine or tacrolimus wa s administered as initial therapy for clinical extensive chronic graft-vers us-host disease (cGVHD). All patients had thrombocytopenia or cGVHD that ev olved directly from acute GVHD as an indicator of a poor prognosis. The stu dy drug (thalidomide or placebo) was administered initially at a dose of 20 0 mg orally per day, followed by a gradual increase to 800 mg/d if side eff ects were tolerable. Treatment with the study drug was discontinued before resolution of cGVHD in 23 (92%) of the 25 patients who received thalidomide and in 17 (65%) of the 26 patients who received placebo (P = .02). Neutrop enia and neurologic symptoms were the most frequent reasons for early disco ntinuation of treatment with thalidomide. The duration of treatment with th alidomide was too short to assess its efficacy in controlling cGVHD. (C) 20 00 by The American Society of Hematology.