Leukemia, Myeloid, Acute(AML)

This study evaluates the effectiveness and safety of a new conditioning regimen called VABu before stem cell transplantation in patients with acute myeloid leukemia (AML) who are in their first complete remission (CR1), classified as non-high-risk per ELN 2022 guidelines, but have poor physical condition (ECOG performance status ≥ 2) or high comorbidity burden (HCT-CI ≥ 2). VABu combines venetoclax, azacitidine (or decitabine as an alternative), and busulfan. This is a single-arm, prospective study with 42 participants. All participants will receive the VABu regimen followed by stem cell transplantation. The primary outcome measure is 2-year overall survival (OS). Secondary outcomes include 2-year relapse-free survival (RFS), transplant-related mortality (TRM), engraftment failure rate, bloodstream infection (BSI), and 2-year all-cause mortality and non-relapse mortality (NRM). The study is conducted at the First Affiliated Hospital of Soochow University.

Adult patients with refractory or relapsed CD123+ hematologic malignancies, including acute myeloid leukemia, myelodysplastic syndrome, acute lymphoblastic leukemia, or blastic plasmocytoid dentritic cell neoplasm will be recruited in the trial. CART123 cells will be manufatured from blood of each patient. During the production of CAR123 cells, patients may receive appropriate bridging therapy. After cells are produced, participants will undergo a single course of lymphodepleting chemotherapy and receive a single dose of CAR123 T cells. The trial will establish the recommended dose for further studies, either the Maximum Tolerated Dose (MTD) or Maximum Feasible Dose (MFD). Patients must be eligible for hematopoietic stem cell transplantation in order to participate in the trial.