Haplo-Cord HSCT for AML/MDS

NCT07588360 | Recruiting DMC

• 1 other identifier: 2025XHYG0034-01
• Study Type: interventional
• Target: 180
• Locations: 1 country
• Sites: 4

Brief Summary

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.

Conditions

MDS (Myelodysplastic Syndrome); AML (Acute Myeloid Leukemia)

Keywords

Acute Myeloid Leukemia(AML); Myelodysplastic Syndrome(MDS); haplo-cord HSCT

Outcome Measures

Primary Outcomes (1)

• Overall Survival (OS)

  3 years after transplantation

Secondary Outcomes (4)

• Progression-Free Survival(PFS)

  3 years after transplantation

• Disease-Free Survival(DFS)

  3 years after transplantation

• GVHD and Relapse-Free Survival(GRFS)

  3 years after transplantation

• Non-Relapse Mortality(NRM)

  3 years after transplantation

Study Arms (1)

Haplo-Cord HSCT

EXPERIMENTAL

Procedure: haplo-cord HSCT; Drug: Fludarabine, Cytarabine, Busulfan, Antithymocyte Globulin (ATG), Melphalan, Cyclophosphamide (CTX)

Interventions

haplo-cord HSCT PROCEDURE

Patients with disease status in CR and MRD-negative before transplantation received the FA3Cy2Bu3 regimen: Fludarabine 25 mg/m² for 3 days, Cytarabine 2 mg/m² for 3 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. All other patients received the FA5Cy2Bu3 regimen: 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. Following the conditioning regimen, patients underwent haploidentical-cord blood hematopoietic stem cell transplantation. Haploidentical hematopoietic stem cells were infused on day 0, and umbilical cord blood hematopoietic stem cells were infused on day 6.

Haplo-Cord HSCT

Fludarabine, Cytarabine, Busulfan, Antithymocyte Globulin (ATG), Melphalan, Cyclophosphamide (CTX) DRUG

Patients with disease status in CR and MRD-negative before transplantation received the FA3Cy2Bu3 regimen: Fludarabine 25 mg/m² for 3 days, Cytarabine 2 mg/m² for 3 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. All other patients received the FA5Cy2Bu3 regimen: 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. Following the conditioning regimen, patients underwent haploidentical-cord blood hematopoietic stem cell transplantation. Haploidentical hematopoietic stem cells were infused on day 0, and umbilical cord blood hematopoietic stem cells were infused on day 6.

Haplo-Cord HSCT

Eligibility Criteria

• Age: 14 Years - 60 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• 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.

You may not qualify if:

• Prior history of other hematopoietic stem cell transplantation.
• History of ex vivo T-cell-depleted stem cell transplantation.
• Survival duration of less than 1 month after 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.
• Pregnant or lactating women.
• Concurrent malignancy of other organ system.
• Any other medical conditions deemed inappropriate for study participation by the treating investigators.

Sponsors & Collaborators

• Fujian Medical University Union Hospital: lead

Study Sites (4)

Fujian Medical University Union Hospital

Fuzhou, Fujian, 350001, China

RECRUITING

Guangdong Provincial People's Hospital

Guangzhou, Guangdong, China

NOT YET RECRUITING

Sichuan Provincial People's Hospital

Chengdu, Sichuan, China

NOT YET RECRUITING

Chinese PLA General Hospital

Beijing, China

NOT YET RECRUITING

Related Publications (18)

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MeSH Terms

Conditions

Anemia, Refractory, with Excess of Blasts; Leukemia, Myeloid, Acute; Myelodysplastic Syndromes

Interventions

fludarabine; Cytarabine; Busulfan; Antilymphocyte Serum; Melphalan; Cyclophosphamide

Condition Hierarchy (Ancestors)

Anemia, Refractory; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Bone Marrow Diseases; Leukemia, Myeloid; Leukemia; Neoplasms by Histologic Type; Neoplasms

Intervention Hierarchy (Ancestors)

Cytidine; Pyrimidine Nucleosides; Pyrimidines; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds; Arabinonucleosides; Nucleosides; Nucleic Acids, Nucleotides, and Nucleosides; Butylene Glycols; Glycols; Alcohols; Organic Chemicals; Mesylates; Alkanesulfonates; Alkanesulfonic Acids; Alkanes; Hydrocarbons, Acyclic; Hydrocarbons; Sulfonic Acids; Sulfur Acids; Sulfur Compounds; Immune Sera; Antibodies; Immunoglobulins; Immunoproteins; Blood Proteins; Proteins; Amino Acids, Peptides, and Proteins; Serum Globulins; Globulins; Biological Products; Complex Mixtures; Nitrogen Mustard Compounds; Mustard Compounds; Hydrocarbons, Halogenated; Phenylalanine; Amino Acids, Aromatic; Amino Acids, Cyclic; Amino Acids; Phosphoramide Mustards; Phosphoramides; Organophosphorus Compounds

Central Study Contacts

Nainong Li, MD

CONTACT

+86 13365910189; nainli@aliyun.com

Lihua Wu, MD

CONTACT

+86 18359180265; 877998423@qq.com

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 28, 2026
• First Posted: May 14, 2026
• Study Start: October 20, 2025
• Primary Completion (Estimated): October 20, 2028
• Study Completion (Estimated): June 30, 2029
• Last Updated: May 14, 2026

Record last verified: 2025-10

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP, CSR, ANALYTIC CODE
• Time Frame: Beginning 3 months and ending 3 years after the publication of results
• Access Criteria: Access to de-identified IPD and supporting documents will be available to all researchers upon application to the principal investigator. Approval will be granted following review, and data will be provided via direct contact with the study team.

Locations

CN (4)