A Prospective, Randomized Controlled Clinical Study on the Efficacy and Safety of the TmBU Regimen Versus mBUCY Regimen for Conditioning Before Allo-HSCT in High-risk or Relapsed/Refractory Acute Leukemia
This project is a prospective, single-center, randomized controlled clinical study. The subjects were high-risk or relapsed/refractory AML or ALL patients aged ≤ 65 years diagnosed by bone marrow cell morphology, immunology, genetics and therapeutic efficacy evaluation. The TmBU scheme or modified Bu/Cy (mBuCy) scheme was used for pretreatment in allo-HSCT. The primary endpoint of the study was the 2-year cumulative incidence of relapse (CIR) after allo-HSCT, and the secondary endpoints were 2-year overall survival rate (OS), progressing-free survival rate (PFS), non-relapse mortality rate (NRM), graft-versus-host disease (GVHD)-free relapse-free survival (GRFS) rate.
• Confirmed diagnosis of AML or ALL according to WHO 2022 guideline criteria, with indications for allo-HSCT list below:
‣ Relapsed/primary refractory (definitions refer to NCCN 2025) or genetic high-risk group AML at diagnosis (risk stratification refers to ELN 2022)
⁃ High-risk at diagnosis (risk stratification refers to ELN 2022) or MRD positive before transplantation B-ALL
⁃ Confirmed diagnosis of T-ALL
⁃ History of central nervous system leukemia (CNSL) or histopathologically confirmed extramedullary manifestation (EMD) during the course of the AML or ALL
• Age 15-65 years old (≤ 65 years old)
• HCT-CI score \< 2 points ECOG 0-2 points
• Adequate organ function:
‣ Cardiac NYHA grade ≤ 2, left ventricular ejection fraction ≥55%
⁃ Creatinine clearance ≥ 50ml/min
⁃ ALT and AST ≤ 2.5 times the upper limit of the normal range, and total bilirubin ≤ 1.5 times the upper limit of the normal range
⁃ Oxygen saturation \> 92% without oxygen
• Expected survival time ≥ 3 months
• Ability to understand and voluntarily sign the informed consent form