Salvage Haploidentical HSCT With DLI and Targeted Therapy for R/R AML
Salvage Haploidentical Allogeneic Hematopoietic Stem Cell Transplantation Combined With Post-transplant Relapse Prevention Strategies for Relapsed/Refractory Acute Myeloid Leukemia (AML): A Prospective Observational Study
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.
Studie Overzicht
Toestand
Conditie
Studietype
Inschrijving (Geschat)
Contacten en locaties
Studiecontact
- Naam: wei MD, PhD
- Telefoonnummer: +86 027-85726003
- E-mail: shiwei076@hust.edu.cn
Deelname Criteria
Geschiktheidscriteria
Leeftijden die in aanmerking komen voor studie
- Volwassen
- Oudere volwassene
Accepteert gezonde vrijwilligers
Bemonsteringsmethode
Studie Bevolking
Beschrijving
Inclusion Criteria:
- 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
Exclusion Criteria:
- 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
Studie plan
Hoe is de studie opgezet?
Ontwerpdetails
Cohorten en interventies
Groep / Cohort |
Interventie / Behandeling |
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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.
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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.
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.
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Wat meet het onderzoek?
Primaire uitkomstmaten
Uitkomstmaat |
Tijdsspanne |
|---|---|
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Cumulative Incidence of Relapse (CIR)
Tijdsspanne: Up to 24 months post-transplantation
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Up to 24 months post-transplantation
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Overall Survival (OS)
Tijdsspanne: Up to 24 months post-transplantation
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Up to 24 months post-transplantation
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Secundaire uitkomstmaten
Uitkomstmaat |
Tijdsspanne |
|---|---|
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Incidence of Acute GVHD (aGVHD)
Tijdsspanne: Within 100 days post-transplantation
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Within 100 days post-transplantation
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Incidence of Chronic GVHD (cGVHD)
Tijdsspanne: Up to 24 months post-transplantation
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Up to 24 months post-transplantation
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Medewerkers en onderzoekers
Studie record data
Bestudeer belangrijke data
Studie start (Geschat)
Primaire voltooiing (Geschat)
Studie voltooiing (Geschat)
Studieregistratiedata
Eerst ingediend
Eerst ingediend dat voldeed aan de QC-criteria
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Updates van studierecords
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Meer informatie
Termen gerelateerd aan deze studie
Aanvullende relevante MeSH-voorwaarden
Andere studie-ID-nummers
- [2025]num(0970)
Plan Individuele Deelnemersgegevens (IPD)
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Informatie over medicijnen en apparaten, studiedocumenten
Bestudeert een door de Amerikaanse FDA gereguleerd geneesmiddel
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