Intensive chemotherapy (IC) may be more efficacious than hypomethylating agents (HMA) as first-line treatment for younger patients with myelodysplastic syndrome (MDS) and elevated bone marrow blasts, according to a study published in the American Journal of Hematology.
The retrospective study included data from 106 patients with MDS or myelodysplastic/myeloproliferative neoplasms (MDS/MPN) who were younger than 60 years and had received either HMA or IC, as a combination of anthracyclines and cytarabine, for first-line therapy.
HMAs were administered to 57 patients and IC was administered to the remaining 49. Median patient age was 55 years (range, 18-59) in the HMA group and 45 years (range, 21-59) in the IC group (P =.03); men and women were equally represented.
Patients treated with IC had a higher overall response rate (82% vs 60%; P =.02), a significantly higher complete remission rate (63% vs 30%; P <.001), and significantly shorter median time to response (1 month vs 2 months; P <.001) compared with HMA. A multivariate analysis including age, NPM1 mutation status, and treatment type showed that IC treatment was the only variable associated with higher overall response rate (odds ratio, 3.3; 95% CI: 1.4-7.9; P =.009).
After median follow-up of 15 months, 51% (38/74) of patients who had achieved a response relapsed or progressed. Median response duration was 19 months (range, 1-166). Transformation to AML occurred in 7 patients receiving HMA and 1 patient receiving IC (P =.07).
The authors noted that future studies exploring whether IC retains increased efficacy when HMA or IC is combined with targeted agents are warranted.
1. Strati P, Garcia-Manero G, Zhao C, et al. Intensive chemotherapy (IC) is more effective than hypomethylating agents (HMA) for the treatment of younger patients with myelodysplastic syndrome (MDS) and elevated bone marrow blasts [published online April 11, 2019]. Am J Hematol. doi:10.1002/ajh.25490