Dr Sekeres on Outcomes from the ASTREON Trial in Low- to Intermediate-Risk MDS

Mikkael A. Sekeres, MD, professor, medicine, chief, Division of Hematology, Leukemia Section, the University of Miami Sylvester Comprehensive Cancer Center, discusses preliminary safety and efficacy findings with oral azacitidine (Vidaza) in patients with low- to intermediate-risk myelodysplastic syndromes (MDS), as reported in the phase 2/3 ASTREON trial (NCT05469737).

Notably, these data were shared at the 2024 ASCO Annual Meeting. ASTREON trial examined patients with low-to-intermediate-risk MDS who were treated with oral azacitidine at doses of either 200 mg or 300 mg daily, Sekeres begins. Patients were randomly assigned to receive 1 of these dosing regimens, he adds. The study revealed that the 200-mg and 300-mg doses were equally effective, eliciting similar response rates, Sekeres reports. Specifically, between approximately one-quarter and one-third of the patients achieved red blood cell (RBC) transfusion independence. This means that these patients, who started the study dependent on RBC transfusions due to anemia, no longer needed transfusions for at least an 8-week period after being treated with the drug, he emphasizes.

Moreover, the trial found that the adverse effects (AEs) associated with both dosing levels were consistent with expectations and are likely less severe than those observed when azacitidine is administered intravenously (IV) or subcutaneously for 7 days, he notes. This indicates that the oral form of the drug may offer a more favorable AE profile, Sekeres says.

Overall, Sekeres states that the trial demonstrated that oral azacitidine is effective, well tolerated, and more convenient compared with IV or subcutaneous administration. The ability to take the medication in pill form is a significant advantage, simplifying the treatment process for patients and potentially improving adherence to the therapy, he reports. This finding is particularly important for patients who rely on regular RBC transfusions, as achieving transfusion independence can greatly enhance their quality of life, Sekeres concludes.