Fludarabine, Cyclophosphamide Plus Thymoglobulin Conditioning Regimen for Unrelated Bone Marrow (or Mobilized Peripheral Blood) Transplantation in Severe Aplastic Anemia
试验速览
- 阶段
- 2 期
- 发起方
- 入组人数
- 30
- 试验地点
- 1
- 主要终点
- To evaluate the engraftment potential, incidence and severity of acute graft versus host disease,toxicity of conditioning regimen for UBMT in SAA.
研究概览
简要总结
Anti-thymocyte globulin (ATG) has been used in severe aplastic anemia as a part of the conditioning regimen. Among the many kinds of ATG preparations, thymoglobulin had been found to be more effective in preventing GVHD and rejection of organ transplants. As the fludarabine based conditioning regimens without total body irradiation have been reported to be promising for BMT/PBSCT from alternative donors in SAA, thymoglobulin was added to fludarabine and cyclophosphamide conditioning to reduce GVHD and to allow good engraftment in UBMT/UPBSCT.
详细描述
GVHD prophylaxis recommendation tacrolimus (0.03 mg/kg/day i.v. by continuous infusion from day -2 and taper with an oral form until 1 year after BMT/PBSCT) methotrexate (15 mg/m2 i.v. on days 1 and 10 mg/m2 i.v. on days 3, 6, 11)
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- — 至 25 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.
- •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.
- •Congenital aplastic anemia including Fanconi anemia.
- •Manipulated bone marrow.
研究组 & 干预措施
Fludarabine
干预措施: cyclophosphamide, fludarabine , thymoglobulin (Drug)
结局指标
主要结局
To evaluate the engraftment potential, incidence and severity of acute graft versus host disease,toxicity of conditioning regimen for UBMT in SAA.
时间窗: From Jan. 1. 2006 to Dec. 31. 2008. For 3 years.
To evaluate overall and EFS follow-up of 1 year after UBMT/PBSCT.
时间窗: From Jan. 1. 2006 to Dec. 31. 2008. For 3 years
次要结局
- To evaluate chronic GVHD and immunologic recovery after UBMT/PBSCT. and the efficacy of UBMT/PBSCT before immuno-suppressive therapy with anti-thymocyte globulin in severe aplastic anemia and long term toxicity of non-TBI based conditioning(From Jan. 1. 2006 to Dec. 31. 2008. For 3 years.)