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

NCT07572695 | Not Yet Recruiting DMC

• 1 other identifier: [2025]num(0970)
• Study Type: observational
• Target: 40
• Locations: 0 countries
• Sites: N/A

Brief Summary

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.

Conditions

Acute Myeloid Leukemia; Relapsed/Refractory Acute Myeloid Leukemia; AML, Relapsed/Refractory

Outcome Measures

Primary Outcomes (2)

• Cumulative Incidence of Relapse (CIR)

  Up to 24 months post-transplantation

• Overall Survival (OS)

  Up to 24 months post-transplantation

Secondary Outcomes (2)

• Incidence of Acute GVHD (aGVHD)

  Within 100 days post-transplantation

• Incidence of Chronic GVHD (cGVHD)

  Up to 24 months post-transplantation

Study Arms (1)

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.

Procedure: Haploidentical Allogeneic Hematopoietic Stem Cell Transplantation (haplo-HSCT); Drug: Post-transplant Maintenance Therapy

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.

Salvage Haploidentical HSCT Cohort

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.

Salvage Haploidentical HSCT Cohort

Eligibility Criteria

• Age: 18 Years - 65 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

This is a single-center prospective observational study enrolling adult patients (18-65 years) with relapsed/refractory acute myeloid leukemia (R/R AML) who have active disease (bone marrow blasts \>5% or extramedullary involvement) and are scheduled to undergo salvage haploidentical allogeneic hematopoietic stem cell transplantation (haplo-HSCT) at Union Hospital, Tongji Medical College, Huazhong University of Science and Technology. The study population consists of high-risk AML patients with HCT-CI ≤5 who are eligible for intensive transplant therapy according to institutional practice.

You may qualify if:

• Signed and dated informed consent Willing and able to comply with all study procedures and follow-up Adults aged 18 to 65 years Diagnosis of acute myeloid leukemia (AML) Active disease before transplantation, defined as bone marrow blasts \>5% or presence of extramedullary disease HCT-CI (Hematopoietic Cell Transplantation-Comorbidity Index) score ≤5

You may not qualify if:

• Bone marrow blasts ≤5% without extramedullary disease before transplantation Age \<18 years or \>65 years HCT-CI score \>5 Patients with other diagnoses besides AML

Sponsors & Collaborators

• Union Hospital, Tongji Medical College, Huazhong University of Science and Technology: lead

MeSH Terms

Conditions

Leukemia, Myeloid, Acute; Recurrence

Condition Hierarchy (Ancestors)

Leukemia, Myeloid; Leukemia; Neoplasms by Histologic Type; Neoplasms; Hematologic Diseases; Hemic and Lymphatic Diseases; Disease Attributes; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Central Study Contacts

wei MD, PhD

CONTACT

+86 027-85726003; shiwei076@hust.edu.cn

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Target Duration: 24 Months
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 3, 2026
• First Posted: May 7, 2026
• Study Start: April 30, 2026
• Primary Completion (Estimated): June 1, 2026
• Study Completion (Estimated): December 1, 2028
• Last Updated: May 7, 2026

Record last verified: 2026-04

Data Sharing

• IPD Sharing: Will not share