An Observation of the Safety and Efficacy of a Modified Transplantation Conditioning and aGVHD Prophylaxis for Severe Aplastic Anemia--a Retrospective, Double-center, Single-arm Clinical Study.
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 发起方
- 入组人数
- 72
- 试验地点
- 1
- 主要终点
- Primary graft falure rate
研究概览
简要总结
The investigators retrospectively evaluted the safety and efficacy of the modified transplantation conditioning and aGVHD prophylaxis in severe aplastic anemia in 4 transplantation centers.
详细描述
Aplastic anemia (AA) is a group of myelo-hemopoietic failure syndromes caused by a variety of etiologies. If not intervened, the average expected survival time is less than half a year.Allogeneic hematopoietic stem cell transplantation (allo-HSCT) is one of the possible cures for this disease. The success rate of treatment for this disease can be further improved under the previous regimen system. The survival rate reported in the literature is 60%-90%. From Jun. 2020 to Dec. 2023, 72 patients in 4 transplantation center received this modified transplantation system, The investigators designed this clinical trial to retrospectively evaluted the safety and efficacy of the modified transplantation conditioning and aGVHD prophylaxis in severe aplastic anemia in the 4 clinical center.
研究设计
- 研究类型
- Observational
- 观察模型
- Case Only
- 时间视角
- Retrospective
入排标准
- 年龄范围
- 3 Years 至 65 Years（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patients who were diagnosed with severe aplastic anemia by NCCN guidelines, and has received HSCT with this transplantation preconditioning and aGVHD prophylaxis regimen;
- •Age 3-65 years old;
- •Weight 10Kg-100Kg;
- •Eastern Cooperative Oncology Group (ECOG) score ≤3;
- •No major organ injury (ECG ejection fraction >45%; bilirubin < 2 times the upper limit of normal value; AST and ALT < 3 times the upper limit of normal value; serum creatinine < 2 times the upper limit of normal value);
- •No severe infection;
- •Subjects voluntarily participated in this clinical trial and signed the informed consent.
排除标准
- •Patients with other hematologic diseases who are not eligible for transplantation or who do not wish to receive transplantation;
- •Patients with an expected survival of less than 1 month;
- •Patients with previous autologous or allogeneic hematopoietic stem cell transplantation;
- •pregnant patients;
- •Patients with severe mental or neurological disorders that would affect the ability to provide informed consent and/or to report or observe adverse events;
- •Other conditions that the investigator determines to be inappropriate for enrollment.
结局指标
主要结局
Primary graft falure rate
时间窗: From the day of HSCs transfusion to +100 day after HSCT.
no apperance or complete loss of donor-derived neutrophils by +28 days
aGVHD incidence
时间窗: From the day of HSCT transfusion to 100 days after HSCT
proportion of patients who developed aGVHD within 100 days after HSCT
次要结局
- Poor graft function rate(From the day of HSCs transfusion to 24 months after HSCT)
- cGVHD incidence(From +100 days after HSCT to the follow-up date)
- adverse events(From the first day of preconditioning to +100 days after hematopoietic stem cell Transfusion)
- 2-year Overall survival rate(24 months)
- Disease-free survival(24 months)