Targeted Busulfan, Fludarabine, Etoposide Conditioning Regimen for Hematopoietic Stem Cell Transplantation in Childhood and Adolescent Acute Lymphoblastic Leukemia
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 入组人数
- 38
- 试验地点
- 1
- 主要终点
- 1-year event free survival after hematopoietic stem cell transplantation.
研究概览
简要总结
To evaluate the outcome of hematopoietic stem cell transplantation using targeted busulfan, fludarabine, etoposide conditioning regimen in childhood and adolescent ALL.
详细描述
Busulfan is a highly toxic drug with narrow therapeutic window used for the conditioning of hematopoietic stem cell transplantation. High exposure is associated with systemic toxicity such as veno-occlusive disease (VOD) and underexposure is associated with graft failure or relapse. In this study the investigators plan to improve the outcome of hematopoietic stem cell transplantation by using optimal busulfan dose through pharmacokinetic study.
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
- — 至 21 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patients who are diagnosed as ALL.
- •Patients who need hematopoietic stem cell transplantation
- •Age: up to 21 years
- •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: Matched in 6/6 of A, B, DR loci.
- •Patients (or one of parents if patients age < 19) should sign informed consent.
排除标准
- •Pregnant or nursing women.
- •Malignant or nonmalignant illness that is uncontrolled or whose control may be jeopardized by complications of study therapy.
- •Psychiatric disorder that would preclude compliance.
研究组 & 干预措施
Busulfan
Drug: 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
1-year event free survival after hematopoietic stem cell transplantation.
时间窗: at 1 year
To evaluate 1-year event free survival after hematopoietic stem cell transplantation.
次要结局
- Engraftment rate(1, 3, 6 and 12 months after transplantation)