A Prospective, Multi-center, Single-arm Clinical Study to Evaluate the Safety and Efficacy of a Modified Allogeneic Hematopoietic Stem Cell Transplantation Regimen for Severe Aplastic Anemia
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 发起方
- 入组人数
- 30
- 试验地点
- 1
- 主要终点
- Acute graft-versus-host disease incidence
研究概览
简要总结
The aim of this study was to evaluate the safety and efficacy of a modified allogeneic hematopoietic stem cell transplantation regimen for aplastic anemia.
详细描述
ObjectiveTo evaluate the safety and efficacy of a modified allogeneic hematopoietic stem cell transplantation regimen in aplastic anemia. 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%. We designed this study to improve the conditioning regimen and optimize the GHVD prevention measures to improve the transplant success rate and prolong patient survival, while minimizing the occurrence of GVHD and reducing the recurrence rate of the disease.30 patients with aplastic anemia were planned to be enrolled.
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
- 3 Years 至 65 Years（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 是
入选标准
- •Patients with benign or malignant hematological diseases such as leukemia, lymphoma, thalassemia, aplastic anemia, etc. diagnosed by NCCN guidelines, and requiring allogeneic hematopoietic stem cell transplantation as determined by the researchers;
- •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 nonhematologic 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.
研究组 & 干预措施
Experimental group
干预措施: Modified transplantation system (Drug)
结局指标
主要结局
Acute graft-versus-host disease incidence
时间窗: [Time Frame: 2 years after allogeneic transplantation with improved conditioning regimen]
overall survival (OS)
时间窗: [Time Frame: 2 years after allogeneic transplantation with improved conditioning regimen]
2-year overall survival (OS) and graft-versus-host disease and relapse-free survival (GRFS) after transplantation
次要结局
- Transplantation-related motality([Time Frame: 2 years after allogeneic transplantation with improved conditioning regimen])
- CMV reactivation rate([Time Frame: 2 years after allogeneic transplantation with improved conditioning regimen])
- Chronic graft-versus-host disease incidence([Time Frame: 2 years after allogeneic transplantation with improved conditioning regimen])
- incidence of Intensive fungal disease([Time Frame: 2 years after allogeneic transplantation with improved conditioning regimen])
- EB virus reactivation rate([Time Frame: 2 years after allogeneic transplantation with improved conditioning regimen])