Multi-Center Study Using Allogeneic Stem Cell Transplantation Following Reduced Intensity Chemotherapy in Patients With Hemoglobinopathies
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 2
- 试验地点
- 12
- 主要终点
- Stable Engraftment With Donor Stem Cells in Patients With Severe Hemoglobinopathy.
研究概览
简要总结
The purpose of this study is to determine if treatment with reduced-dose busulfex, fludarabine and alemtuzumab (CAMPATH) followed by sten cell infusion will allow for donor stem cells to grow in patients with hemoglobinopathies bone marrow and restore circulating blood counts. In addition the incidence and severity of side effects and of graft vs. host disease (GVHD) will be monitored.
详细描述
- In order to undergo transplant procedure, patients will be admitted to the hospital for approximately 10-14 days.
- To prepare patient's bone marrow to accept donor stem cells, they will receive fludarabine and busulfex. Fludarabine will be given intravenously once daily for 4 days. Busulfex will be given once daily for the same 4 days.
- One day before patients receive busulfex and fludarabine, they will also be given alemtuzumab intravenously once daily for 5 days.
- Three days after the end of chemotherapy, patients will receive the infusion of donor stem cells.
- If patients have thalassemia, they will receive subcutaneous injections of filgrastim starting on day one after the donor stem cell transfusion and will continue receiving filgrastim every day until it appears that the donor stem cells have been accepted. If the patient has sickle cell disease, filgrastim will not be given,
- Additional drugs will be given to help prevent infection (i.e. antibiotics).
- After stem cell infusion patients will be examined and have blood tests weekly for 1 month. Bone marrow biopsies, and blood work will also be performed 1 month, 3 months, 6 months and 1 year after stem cell infusion.
- Patients will be on the study for about 12 months. After study is completed progress will be monitored on an annual basis.
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
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patients with sickle cell disease should have one or more of the following: acute chest syndrome requiring hospitalization; nonhemorrhagic stroke or central nervous system event lasting longer than 24 hours; recurrent caso-occlusive pain or recurrent priapism; sickle neuropathy; bilateral proliferative retinopathy and major visual impairment of at least one eye; osteonecrosis of multiple joints; transfusion dependence; vaso-occlusive.
- •Patients with thalassemia should have one or more of the following: transfusion dependence; iron overload; presence of 2 or more alloantibodies against red cell antigens.
排除标准
- •Pregnancy
- •Acute hepatitis
- •Cardiac ejection fraction < 30%
- •Severe renal impairment
- •Severe residual functional neurologic impairment
- •Evidence of HIV infection
结局指标
主要结局
Stable Engraftment With Donor Stem Cells in Patients With Severe Hemoglobinopathy.
时间窗: 3 years
Outcome was measured by ANC \>500 for three consecutive days prior to day 30 after PBSC infusion, \>25% of hematopoietic cells are donor derived as determined by molecular chimerism assays or cytogenetic methods prior to day 45 after PBSC infusion and \>25% of hematopoietic cells are donor derived as determined by molecular chimerism assays or cytogenetic methods after day 180 after PBSC infusion.
次要结局
- Solid Organ Toxicity Related to the Conditioning Regimen.(3 years)
- The Incidence of Grade II-IV Acute Graft vs. Host Disease.(3 years)
研究者
Catherine Wu, MD
Principal Investigator
Dana-Farber Cancer Institute