A Prospective, Single-Center, Single-Arm Interventional Study of a Thiotepa-Based Conditioning Regimen for Haploidentical Hematopoietic Stem Cell Transplantation in Patients Aged 50 Years or Older With Severe Aplastic Anemia
试验速览
- 阶段
- 不适用
- 状态
- 尚未招募
- 入组人数
- 10
- 主要终点
- 1-year transplant-related mortality (TRM)
研究概览
简要总结
This prospective, single-center, single-arm interventional study evaluates the safety and preliminary efficacy of a thiotepa-based conditioning regimen in patients aged 50 years or older with severe or very severe aplastic anemia undergoing haploidentical hematopoietic stem cell transplantation.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 50 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Age 50 years or older.
- •Diagnosis of severe aplastic anemia or very severe aplastic anemia according to institutional diagnostic criteria.
- •Planned to undergo haploidentical hematopoietic stem cell transplantation with an eligible haploidentical donor.
- •Meets institutional performance status and cardiac, hepatic, renal, and pulmonary function requirements for transplantation.
- •Provides written informed consent.
排除标准
- •Previous allogeneic hematopoietic stem cell transplantation.
- •Uncontrolled active infection at the time of enrollment or transplantation.
- •Known hypersensitivity to busulfan, fludarabine, thiotepa, basiliximab, or any relevant excipient.
- •Concurrent hematologic malignancy or another condition that, in the investigator's judgment, makes transplantation unsafe.
- •Pregnancy or breastfeeding.
- •Any serious uncontrolled medical, psychiatric, or social condition that could compromise protocol compliance or participant safety.
研究组 & 干预措施
Thiotepa-Based Conditioning Followed by Haploidentical HSCT
Participants receive thiotepa-based conditioning consisting of busulfan 3.2 mg/kg on Days -8 and -7, thiotepa 10 mg/kg on Day -6, and fludarabine at a total dose of 150 mg/m² divided over Days -6 through -2. Basiliximab 20 mg is administered on Day -1 and Day +4. Haploidentical hematopoietic stem cell transplantation is performed on Day 0.
干预措施: Anti-thymocyte globulin (ATG) (Drug)
Thiotepa-Based Conditioning Followed by Haploidentical HSCT
Participants receive thiotepa-based conditioning consisting of busulfan 3.2 mg/kg on Days -8 and -7, thiotepa 10 mg/kg on Day -6, and fludarabine at a total dose of 150 mg/m² divided over Days -6 through -2. Basiliximab 20 mg is administered on Day -1 and Day +4. Haploidentical hematopoietic stem cell transplantation is performed on Day 0.
干预措施: Thiotepa (Drug)
Thiotepa-Based Conditioning Followed by Haploidentical HSCT
Participants receive thiotepa-based conditioning consisting of busulfan 3.2 mg/kg on Days -8 and -7, thiotepa 10 mg/kg on Day -6, and fludarabine at a total dose of 150 mg/m² divided over Days -6 through -2. Basiliximab 20 mg is administered on Day -1 and Day +4. Haploidentical hematopoietic stem cell transplantation is performed on Day 0.
干预措施: Busulfan (BU) (Drug)
Thiotepa-Based Conditioning Followed by Haploidentical HSCT
Participants receive thiotepa-based conditioning consisting of busulfan 3.2 mg/kg on Days -8 and -7, thiotepa 10 mg/kg on Day -6, and fludarabine at a total dose of 150 mg/m² divided over Days -6 through -2. Basiliximab 20 mg is administered on Day -1 and Day +4. Haploidentical hematopoietic stem cell transplantation is performed on Day 0.
干预措施: Fludarabine (Drug)
Thiotepa-Based Conditioning Followed by Haploidentical HSCT
Participants receive thiotepa-based conditioning consisting of busulfan 3.2 mg/kg on Days -8 and -7, thiotepa 10 mg/kg on Day -6, and fludarabine at a total dose of 150 mg/m² divided over Days -6 through -2. Basiliximab 20 mg is administered on Day -1 and Day +4. Haploidentical hematopoietic stem cell transplantation is performed on Day 0.
干预措施: Basiliximab (Simulect) (Drug)
结局指标
主要结局
1-year transplant-related mortality (TRM)
时间窗: From Day 0 through 1 year after transplantation
The proportion of participants who die from transplant-related causes, including regimen-related toxicity, infection, and graft-versus-host disease, as defined in the study protocol.
次要结局
- Neutrophil Engraftment(From Day 0 through Day 28)
- Platelet Engraftment(From Day 0 through Day 100)
- Primary Graft Failure(From Day 0 through Day 100)
- Overall Survival(From Day 0 through 1 year after transplantation)
- Grade II-IV Acute GVHD(From Day 0 through Day 100)
- Moderate or Severe Chronic GVHD(From Day 0 through 1 year after transplantation)
研究者
Xiao-Jun Huang
Professor
Peking University People's Hospital