Phase 2 study of the lenalidomide and azacitidine combination in patients with higher-risk myelodysplastic syndromes
Lenalidomide and azacitidine each have activity in myelodysplastic syndromes (MDS) patients, where both microenvironment and cell-regulatory mechanisms contribute to disease pathogenesis.
The overall response rate (per modiﬁed MDS International Working Group criteria) was 72%: 16 patients (44%) achieved a complete response(CR),and 10 (28%) had hematologic improvement. Median CR duration was 17 months (range, 3-39); median overall survival was 37months (range,7-55)for CR patients, and 13.6months for the entire cohort (range, 3-55). TET2/DNMT3A/IDH1/2 mutational status was associated with response in a limited number of patients. The lenalidomide/azacitidine combination is well-tolerated and highly active in treating greater-risk MDS. This study is registered at http://www.clinicaltrials.gov
as NCT00352001. (Blood. 2012;120(25): 4945-4951)