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.
Studieoversikt
Status
Forhold
Studietype
Registrering (Antatt)
Kontakter og plasseringer
Studiekontakt
- Navn: wei MD, PhD
- Telefonnummer: +86 027-85726003
- E-post: shiwei076@hust.edu.cn
Deltakelseskriterier
Kvalifikasjonskriterier
Alder som er kvalifisert for studier
- Voksen
- Eldre voksen
Tar imot friske frivillige
Prøvetakingsmetode
Studiepopulasjon
Beskrivelse
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
Studieplan
Hvordan er studiet utformet?
Designdetaljer
Kohorter og intervensjoner
Gruppe / Kohort |
Intervensjon / Behandling |
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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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Hva måler studien?
Primære resultatmål
Resultatmål |
Tidsramme |
|---|---|
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Cumulative Incidence of Relapse (CIR)
Tidsramme: Up to 24 months post-transplantation
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Up to 24 months post-transplantation
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Overall Survival (OS)
Tidsramme: Up to 24 months post-transplantation
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Up to 24 months post-transplantation
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Sekundære resultatmål
Resultatmål |
Tidsramme |
|---|---|
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Incidence of Acute GVHD (aGVHD)
Tidsramme: Within 100 days post-transplantation
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Within 100 days post-transplantation
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Incidence of Chronic GVHD (cGVHD)
Tidsramme: Up to 24 months post-transplantation
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Up to 24 months post-transplantation
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Samarbeidspartnere og etterforskere
Studierekorddatoer
Studer hoveddatoer
Studiestart (Antatt)
Primær fullføring (Antatt)
Studiet fullført (Antatt)
Datoer for studieregistrering
Først innsendt
Først innsendt som oppfylte QC-kriteriene
Først lagt ut (Faktiske)
Oppdateringer av studieposter
Sist oppdatering lagt ut (Faktiske)
Siste oppdatering sendt inn som oppfylte QC-kriteriene
Sist bekreftet
Mer informasjon
Begreper knyttet til denne studien
Ytterligere relevante MeSH-vilkår
Andre studie-ID-numre
- [2025]num(0970)
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