Actively Recruiting
A Multicenter, Randomized, Open-Label Study of Haplo-Cord HSCT for the Treatment of Aplastic Anemia
Led by The First Affiliated Hospital of Soochow University · Updated on 2026-03-30
224
Participants Needed
4
Research Sites
156 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Aplastic anemia (AA) is a bone marrow failure disorder characterized by pancytopenia and hypoplastic bone marrow caused by the decrease of hematopoietic stem cells. The pathogenesis of AA is complex and involves an abnormal hematopoietic microenvironment, hematopoietic stem cell/progenitor cell deficiencies and immunity disorders. Currently, the standard treatment for AA includes immunosuppressive therapy (IST) based on anti-thymocyte/lymphocyte globulin (ATG/ALG) and cyclosporine A (CsA) or hematopoietic stem cell transplantation (HSCT). Although HLA-identical sibling allogeneic hematopoietic stem cell transplantation is considered the preferred transplant option for patients with severe aplastic anemia (SAA), only less than 30% of patients have an available HLA-matched sibling donor. In recent years, haploidentical hematopoietic cell transplantation (Haplo-HCT) has developed rapidly and has become an important alternative. However, graft failure and graft-versus-host disease (GVHD) remain significant factors limiting its efficacy. Umbilical cord blood (UCB) contains a diverse population of hematopoietic stem cells. Compared with other sources, cord blood-derived hematopoietic stem cells are more primitive, more viable, and possess higher proliferative capacity. Therefore, cord blood transplantation, with its notable clinical therapeutic effects, has become an effective and reliable alternative to peripheral blood or bone marrow transplantation. Currently, some transplant centers worldwide have adopted the coinfusion of UCB units with haplo-HCT (haplo-cord HCT) achieving preliminary efficacy in promoting engraftment and reducing the incidence of GVHD. A retrospective comparative study of haplo-cord HCT versus IST in patients with SAA identified haplo-cord HCT as the sole independent predictor for superior health-related quality of life (HRQoL) (P \< 0.0001). Based on existing research and clinical experience, this study plans to investigate and further evaluate the safety and efficacy of haplo-cord HCT in the treatment of aplastic anemia. Primary endpoints will include overall survival, engraftment rate, disease-free survival, incidence of GVHD, CMV/EBV reactivation rate, donor chimerism dynamics, and immune reconstitution.
CONDITIONS
Official Title
A Multicenter, Randomized, Open-Label Study of Haplo-Cord HSCT for the Treatment of Aplastic Anemia
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Aged 14 years or older
- Diagnosed with aplastic anemia meeting the Chinese Guidelines for Diagnosis and Management (2024 Edition)
- Eligible for allogeneic bone marrow or peripheral blood hematopoietic stem cell transplantation as per 2022 Clinical Application and Management Specifications
- Understands the study protocol and voluntarily signs informed consent
- Life expectancy of at least 3 months
- Pre-transplant assessment with Karnofsky Performance Status score ≥70, ECOG Performance Status score ≤2, and Hematopoietic Cell Transplantation-Comorbidity Index score ≤2
You will not qualify if you...
- Aplastic anemia secondary to cancer treatment or with active malignancy
- Pregnant or breastfeeding women
- Psychiatric or psychological disorders preventing treatment compliance
- Positive serologic tests for hepatitis B, hepatitis C, syphilis, or HIV
- Systemic or severe localized infection requiring active antimicrobial therapy
- Significant dysfunction of major organs such as heart, lung, liver, or kidney
- Known allergy or hypersensitivity to any drug or component used in the study
- Current participation or planned participation in another clinical trial
- Any other condition making the patient unsuitable for the study as judged by the investigator
AI-Screening
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Trial Site Locations
Total: 4 locations
1
Hematology Department, The First Affiliated Hospital of Soochow University
Suzhou, Jiangsu, China, China 215006
Actively Recruiting
2
Suzhou Hongci Hematology Hospital
Suzhou, Jiangsu, China, China 215006
Actively Recruiting
3
The Affiliated Hospital of Xuzhou Medical University
Xuzhou, Jiangsu, China, China 221006
Actively Recruiting
4
The First Affiliated Hospital of Zhejiang Chinese Medical University
Hangzhou, Zhejiang, China, China 310006
Actively Recruiting
Research Team
X
Xiaojin Wu
CONTACT
D
Depei Wu
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
RANDOMIZED
Model
PARALLEL
Primary Purpose
TREATMENT
Number of Arms
2
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