Targeted Busulfan, Fludarabine Conditioning Regimen for Hematopoietic Stem Cell Transplantation in Chronic Granulomatous Disease(CGD)
试验速览
- 阶段
- 1 期
- 入组人数
- 5
- 试验地点
- 1
- 主要终点
- Engraftment rate
研究概览
简要总结
In this study the investigators plan to use optimal busulfan dose through pharmacokinetic study in stem cell transplantation of CGD patients.
详细描述
Chronic granulomatous disease is one of the rare congenital immunodeficiency which can be cured by hematopoietic stem cell transplantation. Previous myeloablative conditioning regimen has problems related to the severe toxicities, and non-myeloablative conditioning regimen has the risk of graft failure. Recently, reduced-intensity myeloablative conditioning regimen with busulfan and fludarabine was used usually in leukemia patients.
Busulfan is a highly toxic drug with narrow therapeutic window. In this study we plan to use optimal busulfan dose through pharmacokinetic study in stem cell transplantation of CGD patients.
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
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patients who are diagnosed as CGD.
- •Patients who need hematopoietic stem cell transplantation with busulfan based conditioning regimen.
- •Age: No limit.
- •Performance status: ECOG 0-
- •Patients must be free of significant functional deficits in major organs, but the following eligibility criteria may be modified in individual cases.
- •Heart: a shortening fraction > 30% and ejection fraction > 45%.
- •Liver: total bilirubin < 2 × upper limit of normal; ALT < 3 × upper limit of normal.
- •Kidney: creatinine <2 × normal or a creatinine clearance (GFR) > 60 ml/min/1.73m
- •Patients must lack any active viral infections or active fungal infection.
- •Appropriate donor is available.
- •Patients (or one of parents if patients age < 20) should sign informed consent.
排除标准
- 未提供
研究组 & 干预措施
Busulfan
First dose: busulfan (120 mg/m2 ivs once daily) (if age<1 yr: 80 mg/ m2) Second to forth dose: according to the daily pharmacokinetic study
干预措施: Busulfan (Drug)
结局指标
主要结局
Engraftment rate
时间窗: 1 month after transplantation
To evaluate engraftment rate after hematopoietic stem cell transplantation.
次要结局
- Transplantation-related mortality and toxicities(1, 3, 6 and 12 months after transplantation)