Stem cell transplant from two donor sources for leukemia and myelodysplastic syndromes

Trial details

Who runs it

Fujian Medical University Union Hospital

Trial code

NCT07588360

Record updated

10 June 2026

Original description

This study aims to investigate the clinical efficacy of haploidentical-cord blood hematopoietic stem cell transplantation in patients with acute myeloid leukemia (AML) and high-risk myelodysplastic syndromes (MDS), and to analyze the impact of different engraftment patterns (haploidentical engraftment versus cord blood engraftment) on clinical outcomes. By comparing the efficacy of haploidentical-cord blood transplantation in different subtypes of AML and MDS, this research will explore its unique advantages and comparative effectiveness relative to conventional transplantation strategies, so as to provide new evidence for clinical practice.Read full original descriptionShow less

Haploidentical Combined With Cord Blood Hematopoietic Stem Cell Transplantation for Acute Myeloid Leukemia and Myelodysplastic Syndromes: A Prospective, Multicenter Clinical Study

This study aims to investigate the clinical efficacy of haploidentical-cord blood hematopoietic stem cell transplantation in patients with acute myeloid leukemia (AML) and high-risk myelodysplastic syndromes (MDS), and to analyze the impact of different engraftment patterns (haploidentical engraftment versus cord blood engraftment) on clinical outcomes. By comparing the efficacy of haploidentical-cord blood transplantation in different subtypes of AML and MDS, this research will explore its unique advantages and comparative effectiveness relative to conventional transplantation strategies, so as to provide new evidence for clinical practice.

Specific research objectives I. To evaluate the efficacy of haploidentical-cord blood hematopoietic stem cell transplantation for AML and high-risk MDS, including the speed of hematopoietic recovery, immune tolerance, and long-term survival rates.

II. To compare the effects of different engraftment patterns (haploidentical engraftment vs. cord blood engraftment) on quality of life, immune tolerance, early complications, and long-term prognosis.

III. To identify the clinical advantages and indications of haploidentical-cord blood transplantation through data analysis, and to provide a theoretical basis for clinical decision-making.

Novelty of the Study I. Innovation in Hematopoietic Stem Cell Infusion Schedule The present study employs a sequential infusion strategy: haploidentical stem cells are infused on Day 0, and umbilical cord blood cells are infused on Day +6 after transplantation.In contrast to the conventional approach used at most domestic and international centers (including the uzhou Protocol), in which both stem cell sources are infused simultaneously on Day 0, the current protocol delays cord blood infusion. This design confers potential advantages for immune reconstitution and long-term cord blood engraftment.

II. Unique Myeloablative Conditioning Regimen

The conditioning regimen used in this study is as follows:

Fludarabine 25 mg/m² for 5 days, Cytarabine 2 mg/m² for 5 days, intravenous Busulfan 3.2 mg/kg for 3 days, ATG 5 mg/m² for 2 days, Melphalan 60 mg/m² for 2 days, and CTX 50.0 mg/kg daily for 2 days.

(For patients in complete remission (CR) with negative MRD before transplantation, Fludarabine and Cytarabine are administered for 3 days instead of 5 days.) Distinct from regimens at other centers, our team administers cyclophosphamide within the critical window after haploidentical stem cell infusion but before cord blood infusion, establishing a novel sequential conditioning model. This approach balances myeloablative intensity and immunomodulation, creating a favorable environment for subsequent long-term cord blood engraftment.

III. Engraftment Outcomes and Clinical Value Preliminary clinical experience demonstrates that haplo-cord sequential transplantation following the FA5Cy2Bu3 conditioning regimen combined with low-dose ATG/PTCY can achieve long-term cord blood engraftment in approximately 50% of patients.

By comparison, other domestic protocols (e.g., the Suzhou Protocol) rarely result in sustained cord blood engraftment.

Achievement of long-term cord blood engraftment is clinically meaningful for reducing relapse rates, lowering the incidence and severity of graft-versus-host disease (GVHD), and improving patient prognosis. These outcomes represent a key advantage of the present protocol.

Inclusion: Age between 14 and 60 years, with no gender restriction; Intermediate- or high-risk AML in first complete remission (CR1); AML in second or subsequent complete remission (≥ CR2); Relapsed or refractory AML; Low-risk AML meeting any of the following: Failure to achieve a ≥3-log reduction in RUNX1::RUNX1T1 transcript level compared with baseline after 2 consolidation cycles, or loss of major molecular remission (MMR) within 6 months; CBFB::MYH11/ABL ratio > 0.1% at any time point after 2 consolidation cycles in patients with CBFB::MYH11-rearranged AML; Presence of D816 KIT mutation in patients with CBFB::MYH11-rearranged AML; Flow cytometry-positive MRD at any time point after 2 consolidation cycles in patients with CEBPA double-mutant AML; Persistently positive MRD after chemotherapy in patients with NPM1-mutated AML; Intermediate-2 or high-risk MDS according to the IPSS scoring system; Adequate general health status and ability to tolerate hematopoietic stem cell transplantation; Provision of signed informed consent and willingness to comply with study-required follow-up and examinations.

Exclusion: Prior history of other hematopoietic stem cell transplantation; Survival duration of less than 1 month after transplantation; Concurrent malignancy of other organ system; History of ex vivo T-cell-depleted stem cell transplantation; Severe organ dysfunction, including significant impairment of hepatic, renal, cardiac, or pulmonary function; Active severe infection, such as uncontrolled pneumonia, sepsis, or other systemic infections; History of severe hypersensitivity reactions to study medications, including cyclophosphamide or anti-thymocyte globulin (ATG); Presence of severe psychiatric disorders or cognitive impairment that precludes compliance with study treatment and follow-up; Any other medical conditions deemed inappropriate for study participation by the treating investigators; Pregnant or lactating women.

ClinicalTrials.gov NCT07588360