A Randomized, Multi-Center, Double-Blind, Placebo-Controlled Trial Assessing The Safety And Efficacy Of Thalidomide (THALOMID) For The Treatment Of Anemia In Red Blood Cell Transfusion-Dependent Patients With Myelodysplastic Syndromes

OBJECTIVES:

• Determine the efficacy of thalidomide for the treatment of anemia in patients with myelodysplastic syndromes.
• Determine whether this drug reduces the frequency of leukemia transformation and decreases bone marrow blast percentage in these patients.
• Determine the effect of this drug on neutrophil and platelet production and the number of episodes of febrile neutropenia in these patients.
• Determine the safety of this drug in these patients.

OUTLINE: This is a randomized, double-blind, placebo-controlled, multicenter study. Patients are stratified according to International Prognostic Scoring System score (low and intermediate-1 vs intermediate-2 and high) and transfusion dependence (yes vs no). Patients are randomized to one of two treatment arms.

• Arm I: Patients receive oral thalidomide once daily on weeks 1-24.
• Arm II: Patients receive oral placebo once daily on weeks 1-24. In both arms, patients who have not progressed to leukemia after 24 weeks of therapy may receive open-label thalidomide for an additional 24 weeks in the absence of disease progression or unacceptable toxicity.

Patients are followed at 4 weeks.