Umbilical Cord Blood Consolidation for Older Adults With Intermediate- and High-Risk AML

Acute myeloid leukemia (AML) is a serious blood cancer that commonly affects older adults. Although patients may achieve complete remission after initial treatment, relapse remains common, especially in patients with intermediate- or adverse-risk disease.

This prospective, multicenter, randomized, open-label study will evaluate whether umbilical cord blood infusion used as consolidation therapy can improve outcomes in older patients with AML who have achieved complete remission after induction therapy. The study will enroll approximately 132 patients aged 60 to 80 years with newly diagnosed AML classified as intermediate or adverse risk according to the 2022 European LeukemiaNet criteria. Patients with acute promyelocytic leukemia, TP53 mutations, complex karyotypes, relapsed or refractory AML, or other conditions specified in the eligibility criteria will be excluded.

Participants will be randomly assigned in a 2:1 ratio to an experimental group or a control group. Participants in the experimental group will receive two cycles of consolidation treatment with decitabine, intermediate-dose cytarabine, and unrelated umbilical cord blood infusion. After completion of the two cord blood infusions, maintenance treatment with azacitidine will be recommended for up to 12 months. Participants in the control group will receive two cycles of standard consolidation chemotherapy with intermediate-dose cytarabine, followed by the same recommended azacitidine maintenance treatment.

Participants will be followed during maintenance treatment and for up to 2 years after consolidation therapy, or until disease progression, relapse, death, or another study endpoint occurs. The primary outcome is the proportion of participants who remain alive without leukemia relapse or additional anti-leukemia treatment at 2 years. Secondary outcomes include overall survival, conversion of measurable residual disease to negative status and duration of negativity, recovery of neutrophil and platelet counts, treatment-related mortality, and blood-related and non-blood-related toxicities. Exploratory outcomes include donor cell chimerism, immune status, and, where available, single-cell RNA sequencing or RNA sequencing results.