This is a prospective, multicenter, randomized controlled trial designed to evaluate the efficacy and safety of venetoclax-enhanced BUCY (Ven-BUCY) conditioning compared to the standard BUCY regimen in patients with high-risk acute myeloid leukemia (AML) or myelodysplastic syndromes (MDS) undergoing allogeneic hematopoietic stem cell transplantation (allo-HSCT). Eligible participants aged 12 to 60 years will be randomized 1:1 to receive either Ven-BUCY or standard BUCY conditioning. The primary endpoint is relapse-free survival (RFS) at two years post-transplant. Secondary outcomes include overall survival, relapse rate, non-relapse mortality, measurable residual disease (MRD), and treatment-related adverse events. The study aims to improve post-transplant outcomes by deepening disease remission through the addition of venetoclax, a BCL-2 inhibitor known to target leukemia stem cells and enhance chemotherapy sensitivity.
Diagnosis of acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS) according to 2022 WHO classification
Age between 12 and 60 years
IPSS intermediate-2/high risk or IPSS-R intermediate/high/very high risk
TP53 mutation
Uncontrolled cardiovascular disease or New York Heart Association class III/IV heart failure
Other severe comorbid conditions that may interfere with study participation
Known HIV infection or uncontrolled active hepatitis B or C
Pregnant or breastfeeding women
First Affiliated Hospital of Zhejiang University
Lead sponsor
The First Affiliated Hospital of Zhengzhou University
Collaborator
Union Hospital, Tongji Medical College, Huazhong University of Science and Technology
Collaborator
Tongji Hospital
Collaborator
The Children's Hospital of Zhejiang University School of Medicine
Collaborator
Ruijin Hospital North Shanghai Jiao Tong University School of Medicine
Collaborator