Dr. Patel on Data From the MEDALIST Trial in Low-Risk MDS

Prapti Patel, MD, assistant professor in the Department of Internal Medicine at Harold C. Simmons Comprehensive Cancer Center, UT Southwestern Medical Center, discusses data from the MEDALIST trial on the use of luspatercept-aamt (Reblozyl) in patients with low-risk myelodysplastic syndromes (MDS).

Data from the phase II trial showed that patients with low-risk MDS that were refractory to erythroid-stimulating agents (ESAs) had a response to luspatercept and became transfusion independent within 8 to 12 weeks, says Patel. Patients who had ring sideroblasts did better with the treatment, adds Patel.

Specifically, transfusion independence (TI) for ≥8 weeks was observed in 38% of the patients in the luspatercept group compared with 13% in the placebo group (P <.001). A higher percentage of patients in the luspatercept group than in the placebo group met the key secondary end point of TI for ≥12 weeks (28% vs 8% for weeks 1-24, and 33% vs 12% for weeks 1-48; P <.001).

When looking at the longer-term data of the trial, investigators noted a percentage of patients who needed to undergo transfusions again, says Patel, a finding that was concerning. The next step for this research is to identify new drugs that can provide patients with a longer period of TI, says Patel. One such drug that is being evaluated at UT Southwestern is imetelstat.

Trials are examining imetelstat in patients with MDS who are refractory to ESAs; the hope is that these patients will experience long-term TI for those patients, concludes Patel.