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Blood, 1 December 2000, Vol. 96, No. 12, pp. 3995-3996
BRIEF REPORT
Thalidomide for treatment of patients with chronic
graft-versus-host disease
Sibel Koc,
Wendy Leisenring,
Mary E. D. Flowers,
Claudio Anasetti,
H. Joachim Deeg,
Richard A. Nash,
Jean E. Sanders,
Robert P. Witherspoon,
Frederick R. Appelbaum,
Rainer Storb, and
Paul J. Martin
From the Division of Clinical Research, Fred Hutchinson
Cancer Research Center, Seattle, WA, and Department of Medicine,
University of Washington, Seattle, WA.
In a randomized, placebo-controlled, double-blind trial,
thalidomide or placebo together with glucocorticoids and either
cyclosporine or tacrolimus was administered as initial therapy for
clinical extensive chronic graft-versus-host disease (cGVHD). All
patients had thrombocytopenia or cGVHD that evolved directly from acute GVHD as an indicator of a poor prognosis. The study drug (thalidomide or placebo) was administered initially at a dose of 200 mg orally per
day, followed by a gradual increase to 800 mg/d if side effects 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). Neutropenia and neurologic symptoms were the most frequent reasons for early discontinuation of treatment with thalidomide. The duration of treatment with thalidomide was too short
to assess its efficacy in controlling cGVHD.

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