Phase I/II Study of Fludarabine, Cyclophosphamide Plus TBI Conditioning Regimen for Double Units Cord Blood Transplantation in Severe Aplastic Anemia
试验速览
- 阶段
- 1 期
- 发起方
- 入组人数
- 5
- 试验地点
- 2
- 主要终点
- To evaluate the engraftment potential of fludarabine, busulfan plus thymoglobulin conditioning regimen for cord blood transplantation (CBT) in severe aplastic anemia.
研究概览
简要总结
Severe aplastic anemia is a fatal disease and patients without HLA matched siblings need alternative treatment option. Cord blood transplantation (CBT) has become an alternative treatment means in various diseases, but it has not been proved to be good for severe aplastic anemia. Double units CBT is proposed to have better engraftment potential and and we reported successful double units UCBT after engraftment failure with single unit with promising result. To increase the engraftment potential, fludarabine, cyclophosphamide plus TBI conditioning regimen for double units cord blood transplantation was proposed for the patient with severe aplastic anemia without HLA-matched donor.
研究设计
- 研究类型
- Interventional
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 1 Year 至 21 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Diagnosis of severe aplastic anemia defined by any two or three peripheral blood criteria and either marrow criterion.
- •Peripheral blood
- •Neutrophils < 0.5 x 109/l
- •Platelets < 20 x 109/l
- •Corrected reticulocytes < 1%
- •Bone marrow
- •Severe hypocellularity (< 25%)
- •Moderate hypocellularity (25-30%) with hematopoietic cells representing < 30% of residual cells
- •No prior hematopoietic stem cell transplantation.
- •Age: no limits.
- •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%, 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.
- •No appropriate donor
- •Appropriate cord blood is available: matched at least in 4/6 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.
- •Congenital aplastic anemia including Fanconi anemia.
结局指标
主要结局
To evaluate the engraftment potential of fludarabine, busulfan plus thymoglobulin conditioning regimen for cord blood transplantation (CBT) in severe aplastic anemia.
时间窗: From Oct 2008. to Sep 2011
次要结局
- To evaluate the incidence and severity of toxicity and treatment related mortality(From Oct 2008. to Sep 2011)
- To evaluate overall and event free survival rate(From Oct 2008. to Sep 2011)
- To evaluate acute and chronic GVHD(From Oct 2008. to Sep 2011)
- To evaluate immunologic recovery after CBT(From Oct 2008. to Sep 2011)