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Umbilical Cord Blood-Supported Haplo-HSCT for Aplastic Anemia Treatment Study

NCT06650553 · Status: RECRUITING · Phase: NA · Type: INTERVENTIONAL · Enrollment: 110

Last updated 2025-08-14

No results posted yet for this study

Summary

Aplastic anemia (AA) is a rare bone marrow failure syndrome with an annual incidence of about 0.74/100,000, affecting all ages but more common in the elderly. It's divided into congenital and acquired forms, with the latter being more prevalent. The primary acquired form is linked to T lymphocyte activation and genetic factors. The best treatment is allogeneic hematopoietic stem cell transplantation (allo-HSCT), with a near 90% cure rate. Sibling allo-HSCT is ideal but finding a match is challenging. For those who relapse after immunosuppressive therapy, haploidentical HSCT is a viable option despite risks like graft failure and GVHD. Advances in transplantation have made haplo-HSCT's efficacy comparable to other methods. Recent studies suggest co-transplantation with umbilical cord blood cells can improve outcomes by hastening hematopoietic recovery and prognosis. Our study will evaluate the feasibility and safety of this approach in AA treatment, comparing it to sibling fully matched transplantation, with a focus on infection rates, GVHD incidence, TRM, and EFS, aiming to enhance treatment practices and benefit patients and the medical industry.

Conditions

  • Aplastic Anemias

Interventions

PROCEDURE

Clinically diagnosed AA patients are divided into HLA-matched HSCT group and umbilical cord blood-supported haplo-HSCT group.

Conditioning regimen consisted of the following1)Severe aplastic anemia(SAA):FC-ATG:Flu 30 mg/m\^2/d ×4d (- 7d- -4d), CTX 22.5 -25mg/kg/d × 4d (-7d- -4d), ATG 7.5mg in total（- 5d- -2d).2)Transfusion-dependent non-severe aplastic anemia（TD-NSAA) and Paroxysmal nocturnal hemoglobinuria (PNH) acquired clonal-aplastic anemia(AA):BU/FC-ATG:Bu 0.8mg/kg(q6h -8d）+FC-ATG. The GVHD prophylaxis program was Application of Anti-CD25 Humanized Monoclonal Antibody 50mg (+1d,+4d);cyclosporine A + mycophenolate mofetil (MMF)+short course methotrexate (MTX).The infusion time of umbilical cord blood (UCB) stem cells in the UCB-assisted haploidentical hematopoietic stem cell transplantation (UCB-Haplo-HSCT) group: 4 hours before the infusion of peripheral blood stem cells.In the follow-up phase, outcome evaluation indicators must be collected according to the follow-up plan, ensuring data quality.

Sponsors & Collaborators

  • Shanxi Bethune Hospital

    lead OTHER

Principal Investigators

  • Tao Wang, Dr. · Shanxi Bethune Hospital Regulatory Authority

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
6 Years
Max Age
75 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2024-06-01
Primary Completion
2027-06-30
Completion
2027-07-31

Countries

  • China

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT06650553 on ClinicalTrials.gov