Phase III, Randomized, Placebo-Controlled Trial of CC-486 (Oral Azacitidine) in Patients With Lower-Risk Myelodysplastic Syndromes.

ABSTRACT:

Purpose

Treatment options are limited for patients with lower-risk myelodysplastic syndromes (LR-MDS). This phase III, placebo-controlled trial evaluated CC-486 (oral azacitidine), a hypomethylating agent, in patients with International Prognostic Scoring System LR-MDS and RBC transfusion-dependent anemia and thrombocytopenia.

Methods

Patients were randomly assigned 1:1 to CC-486 300-mg or placebo for 21 days/28-day cycle. The primary end point was RBC transfusion independence (TI).

Results

Two hundred sixteen patients received CC-486 (n = 107) or placebo (n = 109). The median age was 74 years, median platelet count was 25 × 10⁹/L, and absolute neutrophil count was 1.3 × 10⁹/L. In the CC-486 and placebo arms, 31% and 11% of patients, respectively

SUBMITTER: Garcia-Manero G 

PROVIDER: S-EPMC8099416 | biostudies-literature | 2021 May

REPOSITORIES: biostudies-literature