A Trial of Plerixafor With G-CSF as Additional Agents in Conditioning Regimen for Prevention of Graft Failure After Transplantation With TCR Alpha/Beta Grafts Depletion in Patients With Wiskott-Aldrich Syndrome.
试验速览
- 阶段
- 2 期
- 入组人数
- 30
- 试验地点
- 2
- 主要终点
- Event free survival (EFS)
研究概览
简要总结
Treatment Study to assess of safety and efficiency of conditioning with Plerixafor and G-CSF as additional agents for prevention of graft failure after transplantation with TCR alpha/beta grafts depletion in patient with Wiskott-Aldrich syndrome.
详细描述
Severe graft dysfunction, such as the degree of donor chimerism predominantly in the myeloid compartment is one of major problem in patients with Wiskott-Aldrich syndrome (WAS), especially after hematopoietic stem cell transplantation (HSCT) from alternative donor. It often leads to the development of severe thrombocytopenia or even transplants rejection. In this study the hypothesis is that the use of plerixafor and G-CSF as additional agents in conditioning regimen would offers advantages due to lowing risk of mixed chimerism after HSCT. This effect is based on the fact that simultaneous use of plerixafor with G-CSF is efficient in inducing stem cell release and opening of bone marrow (BM) niches. Moreover, stem cell release probably leads to liberation of host stem cells from the anti-apoptotic effects of the BM stroma for the more powerful effect of chemotherapy.
In this study, the investigators use TCR alpha/beta grafts depletion of the grafts as basic technology for HSCT from haploidentical and unrelated donors approved in Institution.
Thus, the purpose of this study is to evaluate the safety and efficiency of myeloablative conditioning with Plerixafor and G-CSF as additional agents for prevention of graft failure after transplantation with TCR alpha/beta grafts depletion in patients with Wiskott-Aldrich syndrome.
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
- 1 Month 至 19 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patients aged ≥ 1 months and < 19 years
- •Patients diagnosed with Wiskott-Aldrich syndrome eligible for an allogeneic transplantation and lacking a related HLA-matched donor
- •Lansky/Karnofsky score > 40, WHO > 4
- •Signed written informed consent
排除标准
- •Dysfunction of liver (ALT/AST > 5 times normal value, or bilirubin > 3 times normal value), or of renal function (creatinine clearance < 30 ml / min)
- •Severe cardiovascular disease (arrhythmias requiring chronic treatment, congestive heart failure or left ventricular ejection fraction <40%)
- •Serious concurrent uncontrolled medical disorder
- •Lack of parents' informed consent.
研究组 & 干预措施
Plerixafor/G-CSF for HSCT conditioning
Myeloablative conditioning regimen with Plerixafor and G-CSF as addition agents before stem cell transplantation in WAS patients.
干预措施: G-CSF for Conditioning before HSCT. (Biological)
Plerixafor/G-CSF for HSCT conditioning
Myeloablative conditioning regimen with Plerixafor and G-CSF as addition agents before stem cell transplantation in WAS patients.
干预措施: Plerixafor for Conditioning before HSCT. (Biological)
结局指标
主要结局
Event free survival (EFS)
时间窗: 24 months
The EFS probability compared with historical control. We mean event as patient's death, second transplantation or persistence of severe thrombocytopenia
次要结局
- Chronic Graft Versus Host Diseases (cGVHD)(24 months)
- Overall survival (OS)(24 months)
- Percentage of patients with full/mixed donor chimerism(12 months)
- Transplant related mortality (TRM)(24 months)
- Severe thrombocytopenia (ST)(24 months)
- Autoimmune complications (AC)(24 months)
- Acute Graft Versus Host Diseases (aGVHD)(12 months)
- Plerixafor related complications (PRC)(2 week)