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Umbilical Cord Blood-Supported Haplo-HSCT for Aplastic Anemia Treatment Study
Sponsor: Shanxi Bethune Hospital
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.
Official title: Clinical Study on Umbilical Cord Blood-Supported Haploidentical Hematopoietic Stem Cell Transplantation for the Treatment of Aplastic Anemia
Key Details
Gender
All
Age Range
6 Years - 75 Years
Study Type
INTERVENTIONAL
Enrollment
110
Start Date
2024-06-01
Completion Date
2027-07
Last Updated
2025-08-14
Healthy Volunteers
No
Conditions
Interventions
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.
Locations (1)
Shanxi Bethune Hospital
Taiyuan, Shanxi, China