A Prospective, Single-Center, Observational Cohort Study of Thiotepa-Based Conditioning in Older Participants With Severe or Very Severe Aplastic Anemia Undergoing Haploidentical Hematopoietic Stem Cell Transplantation
试验速览
- 阶段
- 不适用
- 状态
- 尚未招募
- 入组人数
- 20
- 主要终点
- 1-year transplant-related mortality (TRM)
研究概览
简要总结
This prospective, single-center observational cohort study will follow participants aged 50 years or older with severe or very severe aplastic anemia who receive haploidentical hematopoietic stem cell transplantation with a thiotepa-based conditioning regimen as part of routine clinical care. Treatment decisions, including conditioning, prevention of graft-versus-host disease, and supportive care, are made by the clinical care team and are not assigned or changed by this study. The study will collect routine clinical and follow-up information for 1 year after transplantation to describe transplant-related mortality, survival, engraftment, graft failure, graft-versus-host disease, infections, and serious adverse events.
详细描述
Severe aplastic anemia is a bone marrow failure disorder characterized by reduced blood cell production. For participants who need transplantation and do not have a fully matched sibling donor, haploidentical hematopoietic stem cell transplantation is an important treatment option. Older participants may have reduced organ function, a higher risk of infection, and lower tolerance of intensive treatment.
Thiotepa-based conditioning regimens are used in routine clinical practice before transplantation. In this study, the treating clinical team independently determines the conditioning regimen, medication doses and schedules, prevention of graft-versus-host disease, and supportive care according to each participant's clinical condition. Study participation does not assign, require, or change any treatment.
This is a prospective, single-center observational cohort study. The study will consecutively include participants aged 50 years or older with severe or very severe aplastic anemia who receive haploidentical hematopoietic stem cell transplantation with thiotepa-based conditioning at Peking University People's Hospital. Routine clinical information, transplantation information, and routinely collected follow-up information will be recorded through 1 year after transplantation.
The primary outcome is transplant-related mortality within 1 year after transplantation. Secondary outcomes include overall survival, hematopoietic engraftment, graft failure, acute and chronic graft-versus-host disease, cytomegalovirus and Epstein-Barr virus reactivation, serious infections, and serious adverse events. The study will not add treatment, examinations, blood collection, or follow-up visits beyond routine care.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Prospective
入排标准
- 年龄范围
- 50 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Age 50 years or older at transplantation.
- •Diagnosis of severe aplastic anemia or very severe aplastic anemia.
- •Planned to undergo haploidentical hematopoietic stem cell transplantation at Peking University People's Hospital.
- •The treating clinical team has independently selected a thiotepa-based conditioning regimen as part of routine clinical care before study enrollment.
- •Required baseline and transplantation information is available in the medical record.
排除标准
- •Diagnosis or transplantation type does not meet the study eligibility criteria.
- •The conditioning regimen does not include thiotepa.
- •Essential baseline information is unavailable, such that study eligibility cannot be determined.
- •For a participant with more than one transplantation episode, only the first episode meeting all eligibility criteria will be included in the analysis.
研究组 & 干预措施
Thiotepa-Based Conditioning Cohort
Participants aged 50 years or older with severe aplastic anemia or very severe aplastic anemia who receive haploidentical hematopoietic stem cell transplantation with a thiotepa-based conditioning regimen. The conditioning regimen is selected by the treating clinical team before study enrollment. The study does not assign or alter treatment.
干预措施: Thiotepa (Drug)
结局指标
主要结局
1-year transplant-related mortality (TRM)
时间窗: From Day 0 through 1 year after transplantation
The proportion of participants who die from transplant-related causes, including regimen-related toxicity, infection, and graft-versus-host disease, as defined in the study protocol.
次要结局
- Neutrophil Engraftment(From Day 0 through Day 28)
- Platelet Engraftment(From Day 0 through Day 100)
- Primary Graft Failure(From Day 0 through Day 100)
- Overall Survival(From Day 0 through 1 year after transplantation)
- Grade II-IV Acute graft-versus-host disease（ GVHD）(From Day 0 through Day 100)
- Moderate or Severe Chronic graft-versus-host disease（GVHD）(From Day 0 through 1 year after transplantation)
研究者
Xiao-Jun Huang
Professor
Peking University People's Hospital