Haploidentical Hematopoietic Stem Cell Transplantation for Patients With Thalassemia Major
试验速览
- 阶段
- 4 期
- 发起方
- 入组人数
- 30
- 试验地点
- 1
- 主要终点
- Overall Survival
研究概览
简要总结
The purpose of the study is to assess the safety and efficacy of haploidentical hematopoietic stem cell transplantation for patients with thalassemia major.
详细描述
The only curative therapy for thalassemia major remains the replacement of the defective erythropoiesis by allogeneic hematopoietic stem cell transplantation(HSCT). However, this option is unavailable to many patients as a result of a lack of compatible donors.Haploidentical transplantation has been certified as a valuable alternative for hematological malignancy patients lacking a well-matched donor, with results comparable matched HLA-identical sibling donors or unrelated donors.The objective of this study is to test the feasibility of haploidentical HSCT for patients with thalassemia major.
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
- 2 Years 至 18 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Diagnosed with thalassemia major
- •Lacking of a HLA-identical sibling donor or unrelated donor
- •Indication of haploidentical hematopoietic stem cell transplantation
- •No restrictions for transplantation
排除标准
- •Any restriction for transplantation
- •No indication of haploidentical hematopoietic stem cell transplantation
研究组 & 干预措施
Haploidentical HSCT
Procedure: Haploidentical hematopoietic stem cell transplantation from a related donor (partially matched sibling, father or mother).
Conditioning: Busulfan （4 mg/kg/day,4 days） + Cyclophosphamide （50 mg/kg/day,4 days）+ Fludarabine （50 mg/m2/day,3 days）
GVHD Prophylaxis：Mycophenolate mofetil（0.25g/day）+ Tacrolimus（0.03mg/kg/day）+ Methotrexate（15mg/m2 on day +1,10mg/m2 on day +3,+6,+11）+ Thymoglobulin（2.5 mg/kg/day,4 days）+Basiliximab（10mg on day 0 and +4）
干预措施: Busulfan (Drug)
Haploidentical HSCT
Procedure: Haploidentical hematopoietic stem cell transplantation from a related donor (partially matched sibling, father or mother).
Conditioning: Busulfan （4 mg/kg/day,4 days） + Cyclophosphamide （50 mg/kg/day,4 days）+ Fludarabine （50 mg/m2/day,3 days）
GVHD Prophylaxis：Mycophenolate mofetil（0.25g/day）+ Tacrolimus（0.03mg/kg/day）+ Methotrexate（15mg/m2 on day +1,10mg/m2 on day +3,+6,+11）+ Thymoglobulin（2.5 mg/kg/day,4 days）+Basiliximab（10mg on day 0 and +4）
干预措施: Cyclophosphamide (Drug)
Haploidentical HSCT
Procedure: Haploidentical hematopoietic stem cell transplantation from a related donor (partially matched sibling, father or mother).
Conditioning: Busulfan （4 mg/kg/day,4 days） + Cyclophosphamide （50 mg/kg/day,4 days）+ Fludarabine （50 mg/m2/day,3 days）
GVHD Prophylaxis：Mycophenolate mofetil（0.25g/day）+ Tacrolimus（0.03mg/kg/day）+ Methotrexate（15mg/m2 on day +1,10mg/m2 on day +3,+6,+11）+ Thymoglobulin（2.5 mg/kg/day,4 days）+Basiliximab（10mg on day 0 and +4）
干预措施: Fludarabine (Drug)
Haploidentical HSCT
Procedure: Haploidentical hematopoietic stem cell transplantation from a related donor (partially matched sibling, father or mother).
Conditioning: Busulfan （4 mg/kg/day,4 days） + Cyclophosphamide （50 mg/kg/day,4 days）+ Fludarabine （50 mg/m2/day,3 days）
GVHD Prophylaxis：Mycophenolate mofetil（0.25g/day）+ Tacrolimus（0.03mg/kg/day）+ Methotrexate（15mg/m2 on day +1,10mg/m2 on day +3,+6,+11）+ Thymoglobulin（2.5 mg/kg/day,4 days）+Basiliximab（10mg on day 0 and +4）
干预措施: Mycophenolate mofetil (Drug)
Haploidentical HSCT
Procedure: Haploidentical hematopoietic stem cell transplantation from a related donor (partially matched sibling, father or mother).
Conditioning: Busulfan （4 mg/kg/day,4 days） + Cyclophosphamide （50 mg/kg/day,4 days）+ Fludarabine （50 mg/m2/day,3 days）
GVHD Prophylaxis：Mycophenolate mofetil（0.25g/day）+ Tacrolimus（0.03mg/kg/day）+ Methotrexate（15mg/m2 on day +1,10mg/m2 on day +3,+6,+11）+ Thymoglobulin（2.5 mg/kg/day,4 days）+Basiliximab（10mg on day 0 and +4）
干预措施: Tacrolimus (Drug)
Haploidentical HSCT
Procedure: Haploidentical hematopoietic stem cell transplantation from a related donor (partially matched sibling, father or mother).
Conditioning: Busulfan （4 mg/kg/day,4 days） + Cyclophosphamide （50 mg/kg/day,4 days）+ Fludarabine （50 mg/m2/day,3 days）
GVHD Prophylaxis：Mycophenolate mofetil（0.25g/day）+ Tacrolimus（0.03mg/kg/day）+ Methotrexate（15mg/m2 on day +1,10mg/m2 on day +3,+6,+11）+ Thymoglobulin（2.5 mg/kg/day,4 days）+Basiliximab（10mg on day 0 and +4）
干预措施: Methotrexate (Drug)
Haploidentical HSCT
Procedure: Haploidentical hematopoietic stem cell transplantation from a related donor (partially matched sibling, father or mother).
Conditioning: Busulfan （4 mg/kg/day,4 days） + Cyclophosphamide （50 mg/kg/day,4 days）+ Fludarabine （50 mg/m2/day,3 days）
GVHD Prophylaxis：Mycophenolate mofetil（0.25g/day）+ Tacrolimus（0.03mg/kg/day）+ Methotrexate（15mg/m2 on day +1,10mg/m2 on day +3,+6,+11）+ Thymoglobulin（2.5 mg/kg/day,4 days）+Basiliximab（10mg on day 0 and +4）
干预措施: Thymoglobulin (Drug)
Haploidentical HSCT
Procedure: Haploidentical hematopoietic stem cell transplantation from a related donor (partially matched sibling, father or mother).
Conditioning: Busulfan （4 mg/kg/day,4 days） + Cyclophosphamide （50 mg/kg/day,4 days）+ Fludarabine （50 mg/m2/day,3 days）
GVHD Prophylaxis：Mycophenolate mofetil（0.25g/day）+ Tacrolimus（0.03mg/kg/day）+ Methotrexate（15mg/m2 on day +1,10mg/m2 on day +3,+6,+11）+ Thymoglobulin（2.5 mg/kg/day,4 days）+Basiliximab（10mg on day 0 and +4）
干预措施: Basiliximab (Drug)
结局指标
主要结局
Overall Survival
时间窗: 2 years
2-years overall survival
次要结局
- Disease Free Survival(2 years)
- Engraftment(30 days)
- Transplant Related Mortality(1 year)
- Cumulative Incidence of acute Graft Versus Host Disease(180 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