Prospective Cohort Study of Age-Stratified Busulfan-Based Conditioning Regimens in Adult Patients With Myelodysplastic Syndrome Receiving Haploidentical Hematopoietic Stem Cell Transplantation
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 入组人数
- 120
- 试验地点
- 2
- 主要终点
- transplant-related mortality (TRM)
研究概览
简要总结
This study plan aims to enroll adult patients diagnosed with myelodysplastic syndrome (MDS) who are scheduled to receive T-cell-replete haploidentical hematopoietic stem cell transplantation. After obtaining written informed consent, participants will receive either reduced-toxicity Bu/Flu/Cy/ATG conditioning regimen (for patients aged ≥55 years) or standard myeloablative modified Bu/Cy+ATG conditioning regimen (for patients aged <55 years) followed by unified post-transplant immunosuppression and supportive care. The objective is to prospectively characterize the 1-year transplant-related mortality and comprehensively evaluate hematopoietic engraftment, graft-versus-host disease, infection, relapse, survival outcomes and conditioning-related organ toxicity among all enrolled patients undergoing haploidentical 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
- 18 Years 至 65 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patients who had low- and intermediate-risk MDS without ISD nor URD receiving haploidentical hematopoietic stem cell transplantation
排除标准
- •Patients having ISD or URD; patients having high-risk MDS; patients with active infection; patients with poor compliance; patients with organ failure
研究组 & 干预措施
Age-Stratified Conditioning Regimen
MDS patients without identical sibling donor or unrelated donor would receive haplo-HSCT. Participants will receive either reduced-toxicity Bu/Flu/Cy/ATG conditioning regimen (for patients aged ≥55 years) or standard myeloablative modified Bu/Cy+ATG conditioning regimen (for patients aged <55 years) followed by unified post-transplant immunosuppression and supportive care.
干预措施: RTC conditioning regimen and Modified Bu/Cy conditioning regimen (Drug)
结局指标
主要结局
transplant-related mortality (TRM)
时间窗: Participants will be followed for an expected average of 1 years
Death without disease progression or relapse
次要结局
- Hematopoietic engraftment(Day 30 and Day 90 post-transplantation)
- Cumulative incidence of graft-versus-host disease (GVHD)(Day 100 and 3 years after transplantation)
- Cumulative incidence of CMV and EBV reactivation(1 year after transplantation)
- Cumulative incidence of hematologic relapse(1 year after transplantation)
- Disease-free survival (DFS)(1 year after transplantation)
- Overall survival（OS）(1 year after transplantation)
- Conditioning-related organ toxicities(From conditioning start to Day 30 post-transplantation)
研究者
Xiao-Jun Huang
director
Peking University People's Hospital