Unrelated Umbilical Cord Blood Following HLA-haploidentical Hematopoietic Stem Cell Transplantation in Patients With β-thalassemia Major
试验速览
- 阶段
- 不适用
- 入组人数
- 30
- 试验地点
- 1
- 主要终点
- TM-free survival（TFS）
研究概览
简要总结
Allo-hematopoietic stem cell transplantation(HSCT) is the only way to cure β-thalassemia major at present. To expand donor pool，we developed a haplo-identical HSCT (Hi-HSCT) platform. But in prior Hi-HSCT using high dose post-transplant Cyclophosphamide in patients with leukemia, cytopenia post-transplant often developed, which was considered as a symptom of GVHD. Therefore, the investigators add unrelated umbilical cord blood (UCB) to the Hi-HSCT. It has reported that, as third-party cells, UCB will reduce GVHD.The purpose of this study is to determine whether unrelated UCB following Hi-HSCT can improve outcomes of Hi-HSCT in patients with β-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
- 12 Months 至 18 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •β-thalassemia major
- •< 18 year old
- •Unrelated umbilical cord blood following Haplo-identical HSCT
排除标准
- •≥ 18 year old
- •HLA- matched related donors
- •Unrelated donor transplants
- •Unrelated umbilical cord blood transplants
- •Severe iron overload in heart by T2*
结局指标
主要结局
TM-free survival（TFS）
时间窗: 12 months
the measure is a composite
Transplant Related Martality (TRM)
时间窗: 12 months
the measure is a composite
overall survival（OS）
时间窗: 12 months
the measure is a composite
Primary or Secondary Graft Rejection (GR)
时间窗: 12 months
the measure is a composite
次要结局
- The cumulative incidences of acute graft-versus-host disease（GVHD）(12 months)
- The cumulative incidences of chronic graft vesus host disease (cGVHD)(12 months)
研究者
Chunfu Li
Director and professor
Nanfang Hospital, Southern Medical University