Hematopoietic Stem Cell Transplantation for Patients With Thalassemia Major: A Multicenter, Prospective Clinical Study
试验速览
- 阶段
- 4 期
- 状态
- 已完成
- 发起方
- 入组人数
- 823
- 试验地点
- 1
- 主要终点
- Overall survival
研究概览
简要总结
The only curative therapy for thalassemia major remains the replacement of the defective erythropoiesis by allogeneic hematopoietic stem cell transplantation(allo-HSCT). We conduct a prospective multicenter study to evaluate the efficacy of allo-HSCT in the treatment of thalassemia major.
详细描述
Allogeneic hematopoietic stem cell transplantation (allo-HSCT) is one of the established curative option for thalassemia major (TM). Previous study has predicted that more than 90% of TM patients can survive after allo-HSCT with a thalassemia-free survival (TFS) in around 80% of them.The purpose of this study is to evaluate the efficacy of allo-HSCT in the treatment of thalassemia major.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Sequential
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 2 Years 至 20 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Diagnosed with thalassemia major.
- •Indication of hematopoietic stem cell transplantation.
- •A cardiac ejection fraction of >50%; normal pulmonary function tests and pulmonary examination results; and normal kidney function.
排除标准
- •Aspartate aminotransferase levels > 4-fold the upper limit of the normal range for our institution's lab criteria;
- •Uncontrolled bacterial, viral or fungal infections;
- •Any other restriction for transplantation.
研究组 & 干预措施
URD-HSCT
unrelated donor hematopoietic stem cell transplantation
干预措施: Fludarabine (Drug)
MSD-HSCT
matched sibling donors hematopoietic stem cell transplantation
干预措施: Busulfan (Drug)
MSD-HSCT
matched sibling donors hematopoietic stem cell transplantation
干预措施: Cyclophosphamide (Drug)
MSD-HSCT
matched sibling donors hematopoietic stem cell transplantation
干预措施: Fludarabine (Drug)
MSD-HSCT
matched sibling donors hematopoietic stem cell transplantation
干预措施: Thymoglobulin (Drug)
MSD-HSCT
matched sibling donors hematopoietic stem cell transplantation
干预措施: cyclosporine A (Drug)
MSD-HSCT
matched sibling donors hematopoietic stem cell transplantation
干预措施: Mycophenolate mofetil (Drug)
MSD-HSCT
matched sibling donors hematopoietic stem cell transplantation
干预措施: Methotrexate (Drug)
URD-HSCT
unrelated donor hematopoietic stem cell transplantation
干预措施: Busulfan (Drug)
URD-HSCT
unrelated donor hematopoietic stem cell transplantation
干预措施: Cyclophosphamide (Drug)
URD-HSCT
unrelated donor hematopoietic stem cell transplantation
干预措施: Thymoglobulin (Drug)
URD-HSCT
unrelated donor hematopoietic stem cell transplantation
干预措施: Mycophenolate mofetil (Drug)
URD-HSCT
unrelated donor hematopoietic stem cell transplantation
干预措施: Tacrolimus (Drug)
URD-HSCT
unrelated donor hematopoietic stem cell transplantation
干预措施: Methotrexate (Drug)
haplo-HSCT
haplo-identical hematopoietic stem cell transplantation
干预措施: Busulfan (Drug)
haplo-HSCT
haplo-identical hematopoietic stem cell transplantation
干预措施: Cyclophosphamide (Drug)
haplo-HSCT
haplo-identical hematopoietic stem cell transplantation
干预措施: Fludarabine (Drug)
haplo-HSCT
haplo-identical hematopoietic stem cell transplantation
干预措施: Thymoglobulin (Drug)
haplo-HSCT
haplo-identical hematopoietic stem cell transplantation
干预措施: Mycophenolate mofetil (Drug)
haplo-HSCT
haplo-identical hematopoietic stem cell transplantation
干预措施: Tacrolimus (Drug)
haplo-HSCT
haplo-identical hematopoietic stem cell transplantation
干预措施: Methotrexate (Drug)
结局指标
主要结局
Overall survival
时间窗: 2 years
2-years overall survival
Thalassemia-free survival
时间窗: 2 years
2-years thalassemia-free survival
次要结局
- Engraftment(30 days)
- Transplant Related Mortality(2 year)
- Cumulative Incidence of acute Graft Versus Host Disease(100 days)
- Cumulative Incidence of chronic Graft Versus Host Disease(2 years)
- Cumulative Incidence of Infectious Complications(2 years)
研究者
Yongrong Lai
Director of the Hematology Department
First Affiliated Hospital of Guangxi Medical University