A Phase II Trial of Myeloablative Conditioning and Transplantation of HLA-matched, Partially HLA-mismatched, HLA-haploidentical or Matched Unrelated (MUD) Bone Marrow for Patients With Refractory Severe Aplastic Anemia
试验速览
- 阶段
- 2 期
- 状态
- 终止
- 入组人数
- 4
- 试验地点
- 2
- 主要终点
- To estimate the overall survival (OS) at 1 year following HLA-matched, Partially HLA-mismatched, HLA-haploidentical or matched unrelated Bone Marrow for Patients with Refractory Severe Aplastic Anemia
研究概览
简要总结
Patients with severe, refractory aplastic anemia have a severe, life threatening disease in their bone marrow. Refractory disease means that disease has come back or not responded after receiving one or more immunosuppressive treatments. High dose chemotherapy followed by bone marrow transplantation (BMT) has been used to treat blood diseases like aplastic anemia but complications from Graft vs Host disease (GVHD) and graft failure have limited the survival for those patients.
Another study done here at Johns Hopkins has shown that in patients with other diseases (blood cancers) some immunosuppressive drugs given after the BMT has decreased how often patients had complications of GVHD and engraftment failure.
This research is being done to find if this approach will help patients with aplastic anemia who have failed other treatments will have better outcomes.
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
- 6 Months 至 70 Years（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patients who have refractory or relapsed SAA following treatment with one or more immunosuppressive regimens.
- •Age 6 months - 70 years
- •Patients must meet medical criteria for myeloablative BMT
- •Patients or their parents/guardians and donors must be able to sign consent forms.
- •Patients must be geographically accessible and willing to participate in all stages of treatment.
排除标准
- •Poor cardiac function: left ventricular ejection fraction <45% as determined by MUGA or ECHO.
- •Poor pulmonary function: FEV1 and FVC <50% predicted.
- •Poor renal function
- •Positive leukocytotoxic crossmatch
- •Women of childbearing potential who currently are pregnant (Β-HCG+) or who are not practicing adequate contraception
- •Uncontrolled viral, bacterial, or fungal infections
结局指标
主要结局
To estimate the overall survival (OS) at 1 year following HLA-matched, Partially HLA-mismatched, HLA-haploidentical or matched unrelated Bone Marrow for Patients with Refractory Severe Aplastic Anemia
时间窗: 1 year
To estimate the event-free survival (EFS) at 1 year following HLA-matched, Partially HLA-mismatched, HLA-haploidentical or matched unrelated Bone Marrow for Patients with Refractory Severe Aplastic Anemia
时间窗: 1 year
次要结局
未报告次要终点