Salvage Haploidentical HSCT With DLI and Targeted Therapy for R/R AML

This is a prospective, single-center, observational study to evaluate the efficacy and safety of salvage haploidentical allogeneic hematopoietic stem cell transplantation (haplo-HSCT) combined with post-transplant relapse prevention strategies in patients with relapsed/refractory acute myeloid leukemia (R/R AML). Eligible patients are adults aged 18-65 years with active AML (bone marrow blasts \>5% or extramedullary disease) and HCT-CI score ≤5. All patients will receive a uniform conditioning regimen consisting of fludarabine, busulfan, and MECCNU, with addition of targeted agents (such as sorafenib, midostaurin, or venetoclax) according to mutation status. Graft-versus-host disease (GVHD) prophylaxis includes reduced-dose ATG (6 mg/kg), FK506, MMF, and basiliximab. Post-transplant maintenance with targeted therapy or azacitidine and prophylactic donor lymphocyte infusion (DLI) will be administered to reduce relapse risk. The primary endpoints are cumulative incidence of relapse (CIR), overall survival (OS), and progression-free survival (PFS). Secondary endpoints include incidence of acute and chronic GVHD, CMV/EBV reactivation, non-relapse mortality (NRM), and GVHD-free, relapse-free survival. Patients will be followed for 24 months after transplantation. This study aims to explore an optimized transplant strategy to improve long-term survival in this high-risk population.

NCT Number	NCT07572695
Lead Sponsor	Union Hospital, Tongji Medical College, Huazhong University of Science and Technology
Conditions	Acute Myeloid Leukemia, Relapsed/Refractory Acute Myeloid Leukemia, AML, Relapsed/Refractory
Enrollment	40 participants
Start Date	2026-04-30
Primary Completion	2026-06 (estimated)
Study Completion	2028-12 (estimated)
Updated on ClinicalTrials.gov	2026-05-07