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

Sponsor
Union Hospital, Tongji Medical College, Huazhong University of Science and Technology
Study ID
NCT07572695
Status
Not Yet Recruiting

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Conditions

Eligibility Criteria

Sex
ALL
Age
18 Years - 65 Years
Healthy Volunteers
Not accepted

Interventions

  • Haploidentical Allogeneic Hematopoietic Stem Cell Transplantation (haplo-HSCT) — PROCEDURE
    Salvage haploidentical allogeneic hematopoietic stem cell transplantation using a conditioning regimen of Fludarabine (120-180 mg/m²), Busulfan (3-4 mg/kg), and MECCNU 250 mg/m² (intensity adjusted based on prognostic index). Targeted agents (sorafenib, midostaurin, or venetoclax) are added according to genetic mutations (e.g., FLT3) until stem cell infusion. GVHD prophylaxis includes ATG 6 mg/kg, tacrolimus (FK506), mycophenolate mofetil (MMF), and basiliximab on day +4. No MTX or post-transplant cyclophosphamide (PTCY) is used. Immunosuppressants are tapered within 100 days if no significant GVHD.
  • Post-transplant Maintenance Therapy — DRUG
    Starting from approximately day +30 after transplantation, patients receive mutation-guided targeted therapy (sorafenib 200 mg daily for FLT3/ITD mutation) or azacitidine 75 mg/m² on days 1-3. Maintenance therapy aims to reduce the risk of relapse.

Study Details

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.

Key Dates

First listed
May 7, 2026
Start date
Apr 30, 2026
Status verified
Apr 2026
Primary completion
Jun 30, 2026
Completion
Dec 31, 2028

Study Design

Enrollment
40 participants (estimated)

Arms

  • Arm: Salvage Haploidentical HSCT Cohort
    Patients with relapsed/refractory acute myeloid leukemia (R/R AML) who have active disease (bone marrow blasts \>5% or extramedullary involvement) before transplantation, aged 18-65 years, and HCT-CI score ≤5. All patients in this single-arm prospective observational cohort will receive salvage haploidentical allogeneic hematopoietic stem cell transplantation (haplo-HSCT) followed by standardized post-transplant relapse prevention strategies. Participants will be followed for 24 months after transplantation.

Primary Outcome Measure

Cumulative Incidence of Relapse (CIR) [ Time Frame: Up to 24 months post-transplantation ]

Central Contacts

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