HMA Therapy Associated with Favorable Long-Term Clinical Outcomes in Lower-Risk MDS

A long-term analysis of a previously reported randomized, phase II study of low-dose treatment with a hypomethylating agent (HMA) (specifically, azacitidine versus decitabine) in lower-risk myelodysplastic syndromes (MDS) and MDS/myeloproliferative neoplasms found that the treatment was associated with favorable long-term clinical outcomes.

The results were published in NEJM Evidence in a paper by Koji Sasaki, MD, PhD, of the Department of Leukemia at the University of Texas MD Anderson Cancer Center, and colleagues.

The researchers analyzed data from previously untreated adult patients with low- or intermediate-risk MDS or chronic myelomonocytic leukemia, as measured by the International Prognostic Scoring System, who had been enrolled in a randomized, phase II study of low-dose decitabine versus low-dose azacitidine.

Patients were randomly assigned to receive either decitabine 20 mg/m² daily or azacitidine 75 mg/m² daily on days one to three in every 28-day cycle. Under the Bayesian response-adaptive design of the original trial, effective therapy was assigned more frequently. A total of 113 patients were treated: 73 (65%) with decitabine and 40 (35%) with azacitidine.

The overall response rate was 67% and 48% in the decitabine and azacitidine groups, respectively (P=.042). Among 59 patients with baseline transfusion dependency, 19 (32%) reached transfusion independence (decitabine, n=16/39 [41%]; azacitidine, n=3/20 [15%]; P=.039). With a median follow-up of 68 months, the median overall event-free survival and overall survival were 17 months and 33 months, respectively.

“In summary, therapy with low-dose HMAs in patients with lower-risk MDS can induce durable response of transfusion independency,” the authors wrote. “The response to HMA therapy was associated with favorable clinical outcomes in lower-risk MDS.”

Sasaki K, Jabbour E, Montalban-Bravo G. Low-dose decitabine versus low-dose azacitidine in lower-risk MDS. 2022. doi:10.1056/EVIDoa2200034