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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.
This study is designed to assess the safety and efficacy of haplo-cord HCT versus haplo HCT in patients with aplastic anemia, with particular emphasis on factors associated with treatment outcome and adverse events.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Experimental group: 112 patients with aplastic anemia undergo haplo-cord HCT | Experimental |
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| Comparator group: 112 patients with aplastic anemia undergo haplo HCT | Active Comparator |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Haploidentical hematopoietic cell transplantation combined with unrelated cord blood stem cells | Procedure | 1. Donor Stem Cells Infusion (Haploidentical hematopoietic cell transplantation combined with unrelated cord blood stem cells) Day 0: Intravenous infuse a single unit of unrelated cord blood stem cells( TNC≤2×10⁷/kg, CD34+ cells≤0.8×10⁵/kg, HLA match≥5/10 loci, and either matched ABO type or a cord blood unit of type O). This is followed, 6 hours after the cord blood infusion, by the infusion of donor haploidentical stem cells (TNC≥8×10⁸/kg, CD34+ cells≥4.0×10⁶/kg, and HLA match≥6/12 loci). 2. Conditioning Regimen: Day -7 to Day -6: Busulfan (Bu) 3.2mg/kg/day; Day -5 to Day -2: Cyclophosphamide (CTX) 160-200mg/kg. 3. Graft-versus-Host Disease Prophylaxis Regimen: Starting on the day before transplantation (Day -1): Cyclosporin A (CsA) 3mg/kg/day; Day +1 to Day +28: Mycophenolate Mofetil (MMF) 600mg/㎡/12h. 4. Infection Prophylaxis Regimen: Day -7 to Day -3: Ganciclovir (GCV): 5mg/kg/12h; Starting two days before transplantation (Day -2):Aciclovir (ACV): 250mg/㎡/8h. |
| Measure | Description | Time Frame |
|---|---|---|
| Overall survival rate | We estimated OS from the time of transplant until the date of death of any cause or last follow-up for patients still alive. | within 1 year following HSCT |
| Measure | Description | Time Frame |
|---|---|---|
| The cumulative incidence of neutrophil engraftment and platelet engraftment | Neutrophil and platelet engraftment is defined as the first occurence of 3 consecutive days with an absolute neutrophil count of at least 0.5x10⁹/L and a platelet count of over 20x10⁹/L/L for 7 consecutive days without transfusion support. | on day 28+7 following HSCT |
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Inclusion Criteria: (1)Aged ≥14 years. (2) Patients diagnosed with aplastic anemia meeting the diagnostic criteria of the Chinese Guidelines for the Diagnosis and Management of Aplastic Anemia (2024 Edition). (3) Patients who were eligible for allogeneic bone marrow or peripheral blood hematopoietic stem cell transplantation in accordance with the Clinical Application and Management Specifications for Allogeneic Hematopoietic Stem Cell Transplantation Technology (2022 Edition). (4) The patient understands the study protocol and voluntarily signs the informed consent form. (5) Life expectancy of ≥3 months; (6) Pre-transplant assessment meets the following criteria: Karnofsky Performance Status (KPS) score≥70, Eastern Cooperative Oncology Group Performance Status (ECOG PS) score≤2, and Hematopoietic Cell Transplantation-Comorbidity Index (HCT-CI) score≤2.
Exclusion Criteria: (1)Patients with aplastic anemia secondary to malignancy treatment or those with concurrent active malignancy. (2) Women who are pregnant or lactating. (3) Patients with psychiatric or psychological disorders that preclude adequate compliance with the treatment protocol. (4) Patients with positive serologic testing for any of the four major infectious diseases (hepatitis B, hepatitis C, syphilis, HIV). (5) Patients with systemic infection or localized severe infection requiring active antimicrobial therapy. (6) Patients with significant dysfunction of major organs (e.g., heart, lung, liver, kidney). (7) Patients with a known allergy or hypersensitivity to any drug or component used in this study. (8) Patients who are currently participating in or plan to participate in any other clinical trial. (9) Any other condition deemed by the investigator to render the patient unsuitable for study participation.
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| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Xiaojin Wu | Contact | +8613057493105 | wuxiaojin@suda.edu.cn | |
| Depei Wu | Contact | +8613951102021 | wudepei@suda.edu.cn |
| Name | Affiliation | Role |
|---|---|---|
| Depei Wu | The First Affiliated Hospital of Soochow University | Study Chair |
| Xiaojin Wu | The First Affiliated Hospital of Soochow University | Study Chair |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Hematology Department, The First Affiliated Hospital of Soochow University | Recruiting | Suzhou | Jiangsu | China 215006 | China |
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| Haploidentical hematopoietic cell transplantation | Procedure | 1. Donor Stem Cell Infusion (Haploidentical hematopoietic cell transplantation) Day 0: Intravenous infuse of donor haploidentical stem cells (TNC≥8×10⁸/kg, CD34+ cells≥4.0×10⁶/kg, and HLA match≥6/12 loci). 2. Conditioning Regimen: Day -7 to Day -6: Busulfan (Bu) 3.2mg/kg/day; Day -5 to Day -2: Cyclophosphamide (CTX) 160-200mg/kg. 3. Graft-versus-Host Disease Prophylaxis Regimen: Starting on the day before transplantation (Day -1): Cyclosporin A (CsA) 3mg/kg/day; Day +1 to Day +28: Mycophenolate Mofetil (MMF) 600mg/㎡/12h. 4. Infection Prophylaxis Regimen: Day -7 to Day -3: Ganciclovir (GCV): 5mg/kg/12h; Continuous medication starting from two days before transplantation (Day -2): Aciclovir (ACV): 250mg/㎡/8h. |
|
| Disease Free Survival | We defined DFS as the time from the initiation of transplant to the occurrence of any treatment failure event (including graft failure or disease relapse requiring therapeutic intervention), or death from any cause, whichever occurred first. Patients who were alive and event-free at the last follow-up were censored. | within 1 year following HSCT |
| The cumulative incidence and grade of graft-versus-host disease (GVHD) | Graft-versus-host disease (GvHD) is a medical complication following the receipt of transplanted tissue from a genetically diferent person. | within 1 year following HSCT |
| Suzhou Hongci Hematology Hospital | Recruiting | Suzhou | Jiangsu | China 215006 | China |
| The Affiliated Hospital of Xuzhou Medical University | Recruiting | Xuzhou | Jiangsu | China 221006 | China |
| The First Affiliated Hospital of Zhejiang Chinese Medical University | Recruiting | Hangzhou | Zhejiang | China 310006 | China |
| ID | Term |
|---|---|
| D000741 | Anemia, Aplastic |
| ID | Term |
|---|---|
| D000740 | Anemia |
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
| D000080983 | Bone Marrow Failure Disorders |
| D001855 | Bone Marrow Diseases |
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