Thalidomide has been reported to be an effective agent for treatment o
f chronic graft-versus-host disease (CGVHD), To determine the efficacy
of this agent in patients with refractory CGVHD a total of 80 patient
s who failed to respond to prednisone (PSE) or PSE and cyclosporine (C
SA) were treated with thalidomide, Sixteen patients (20%) had a sustai
ned response, 9 with a complete remission and 7 with a partial respons
e. Twenty-nine patients (36%) had thalidomide discontinued because of
side effects, which included sedation, constipation, neuritis, skin ra
sh, and neutropenia. Side effects were reversible with drug discontinu
ation except for mild residual neuritis in one case, Rashes and neutro
penia have not previously been reported as thalidomide side effects wh
en used for CGVHD treatment. We conclude thalidomide is immunosuppress
ive and active in the treatment of CGVHD. A high incidence of reversib
le side effects limited dose intensity and reduced the number of patie
nts who could benefit from treatment. (C) 1995 by The American Society
of Hematology.