3 Trials Advancing Treatment Options for AML and MDS

In this installment of Clinical Trial Corner, Dr. Dina Khalaf, a clinical associate professor in the University of Florida Division of Hematology and Oncology, walks through three trials shaping the future of care for patients with acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS).

ASCERTAIN-V

This trial tests an all-oral regimen combining Inqovi (decitabine and cedazuridine) with Venclexta (venetoclax) in adults with newly diagnosed AML who are not candidates for intensive chemotherapy, typically those older than 65 or those choosing a less intensive path, including as a bridge to allogeneic stem cell transplant. Khalaf said the oral regimen showed responses comparable with the standard intravenous combination while sparing patients extra infusion visits. The FDA approved the all-oral combination in May 2026.

MyeloMATCH MM1MDS-A01

This trial asks whether adding Idhifa (enasidenib) — already approved for IDH2-mutated AML — to standard oral decitabine and cedazuridine improves outcomes for patients with newly diagnosed, high-risk MDS carrying an IDH2 mutation. Patients are randomly assigned to standard therapy alone or with enasidenib added. Khalaf noted the drug can cause differentiation syndrome, which may present as fever, swelling or shortness of breath and requires close monitoring.

HOVON 181 AML/AMLSG 37-25

This phase 3, randomized, double-blind trial adds the menin inhibitor bleximenib to standard induction chemotherapy for newly diagnosed patients 18 and older with NPM1-mutated or KMT2A-rearranged AML who can tolerate intensive treatment. Earlier-phase data showed an overall response rate of 95.8% and complete remission in up to 87.5% of patients, though menin inhibitors also carry risks of differentiation syndrome and QTc prolongation, which is tracked with EKG monitoring.

Khalaf encouraged caregivers to keep asking questions, lean on their support networks and not hesitate to seek help.

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