To Evaluate Different Conditioning Regimens for HLA Matched Donor Transplantation in Severe Aplastic Anemia: a Prospective, Multicenter, Randomized Controlled Study
试验速览
- 阶段
- 4 期
- 状态
- 招募中
- 入组人数
- 160
- 试验地点
- 1
- 主要终点
- Failure free survival
研究概览
简要总结
Hematopoietic stem cell transplantation (HSCT) from a human leukocyte antigen (HLA) -matched donor is an effective option for severe aplastic anemia (SAA), but there is no standardized and recommended conditioning regimen. The occurrence of mixed chimerism after transplantation is associated with secondary graft failure and poor failure-free survival. Previous studies have shown that Fludarabine (Flu)/ Cyclophosphamide (Cy)/ antithymocyte globulin (antithymocyte globulin), ATG) and Cy/ATG conditioning regimens had higher rates of mixed chimerism and poorer failure-free survival. A small cohort study has suggested that adding busulfan to Flu/Cy/ATG or Cy/ATG can reduce the incidence of mixed chimerism and improve failure-free survival. This study was a prospective, multicenter, randomized controlled trial to compare the efficacy and safety of different conditioning regimens in the treatment of severe aplastic anemia (SAA) after hematopoietic stem cell transplantation (HSCT) from HLA-identical sibling or unrelated donor.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 1 Year 至 50 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Diagnosed as SAA/vSAA
- •Indication for hematopoietic stem cell transplantation
- •Available HLA matched sibling or unrelated donor
- •No active infection
- •No serious organ damage: liver and kidney function (ALT and AST < 2.5 times normal value, normal renal function, no cardiac insufficiency)
- •Signed informed consent
- •High risk factors of mixed chimerism, at least one of the following
- •Age < 18 years old
- •Ferritin level ≥2500ng/ml before transplantation
排除标准
- •Age > 50 years old
- •Active infections that were difficult to control
- •Severe liver and kidney dysfunction
- •Mental illness
- •Not signing the informed consent
- •pregnant or lactating women
- •Any condition considered by the investigators to be unsuitable for enrollment
研究组 & 干预措施
Busulfan included group
The conditioning regimens were Bu/Flu/Cy/ATG or Bu/Cy/ATG, depending on the patient's risk factors of regimen related cardiotoxicity.
干预措施: Busulfan (Drug)
Control group
The conditioning regimens were Flu/Cy/ATG or Cy/ATG, depending on the patient's risk factors of regimen related cardiotoxicity.
干预措施: Flu/Cy/ATG or Cy/ATG (Drug)
结局指标
主要结局
Failure free survival
时间窗: 1 year post HSCT
Failure free survival was defined as survival with a response to therapy.
次要结局
- The incidence of mixed chimerism(1 year post HSCT)
- Regimen related toxicity(100 days post HSCT)
- Myeloid and platelet engraftment(100 days post HSCT)
- The incidence of graft versus host disease(100 days post HSCT for aGvHD and 1 year post HSCT for cGvHD)
- The incidence of CMV and EBV reactivation(100 days post HSCT)
- The incidence of Transplantation related mortality(1 year post HSCT)
- The probability of Overall survival(1 year post HSCT)
研究者
Xiao-Jun Huang
Professor
Peking University People's Hospital