Mesenchymal Stem Cells With or Without G-CSF Mobilized Peripheral Blood Stem Cell for Treatment of Poor Graft Function and Delayed Platelet Engraftment After Allogeneic Hematopoietic Stem Cell Transplant
试验速览
- 阶段
- 2 期
- 入组人数
- 120
- 试验地点
- 1
- 主要终点
- Percentage of Participants with Hematopoietic Recovery
研究概览
简要总结
The purpose of this study is to compare the efficacy of mesenchymal stem cells (MSCs) with or without granulocyte colony-stimulating factor (G-CSF) mobilized peripheral stem cells (PBSC) in treating patients experiencing poor graft function or delayed platelet engraftment after allogeneic hematopoietic stem cell transplantation.
详细描述
Allogeneic hematopoietic stem cell transplantation(allo-HSCT) is the only cure for many hematologic diseases. However, about 5-27% of patients would suffer from poor graft function (PGF) and more recipients might develop delayed platelet engraftment (DPE) after allo-HSCT. These complications are associated with considerable mortality related to infections or hemorrhagic complications. Treatment of PGF and DPE usually involves hematopoietic growth factors such as granulocyte colony-stimulating factor (G-CSF) and thrombopoietin (TPO), or second transplantation, but these methods have dismal effect or even a significant risk of graft-versus-host disease (GVHD).
Mesenchymal stem cells (MSCs) are a form of multipotent adult stem cells that can be isolated from bone marrow (BM), adipose tissue, and cord blood. Clinical applications of human MSCs include improving hematopoietic engraftment, preventing and treating graft-versus-host disease after allo-HSCT and so on. Some studies have shown that MSCs combined with PBSC or cord blood could be useful to improve engraftment after HSCT. Several reports suggested MSCs might be effective in the treatment of PGF.
However, the efficacy of MSCs as single-drug treatment for PGF or DPE is unsatisfactory in our previous study. Therefore, in the present study, G-CSF mobilized PBSC will be used combined with MSCs in the patients with PGF or DPE after allo-HSCT.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 14 Years 至 65 Years（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Age：14-65 years
- •PGF or DPE after allo-HSCT
- •Subjects (or their legally acceptable representatives) must have signed an informed consent document
排除标准
- •Any abnormality in a vital sign (e.g., heart rate, respiratory rate, or blood pressure)
- •Patients with any conditions not suitable for the trial (investigators' decision)
研究组 & 干预措施
MSCs&PBSC
PBSC will be intravenously infused at a dose of 2×10^8/kg. MSCs will be intravenously infused at a dose of 1×10^6 cells/kg once per week or until complete response(CR) . If the patients do not achieve CR or partial remission (PR) within 4 weeks, they will swithed to other therapy. If the patients achieve PR within 4 weeks, a second course of the same treatment will be given.
干预措施: PBSC (Biological)
MSCs
MSCs will be intravenously infused at a dose of 1×10^6 cells/kg once per week or until CR. If the patients have no response (NR) within 4 weeks, they will switch to other therapy. If the patients achieve PR within 4 weeks, a second course of the same treatment will be given.
干预措施: MSCs (Biological)
MSCs&PBSC
PBSC will be intravenously infused at a dose of 2×10^8/kg. MSCs will be intravenously infused at a dose of 1×10^6 cells/kg once per week or until complete response(CR) . If the patients do not achieve CR or partial remission (PR) within 4 weeks, they will swithed to other therapy. If the patients achieve PR within 4 weeks, a second course of the same treatment will be given.
干预措施: MSCs (Biological)
结局指标
主要结局
Percentage of Participants with Hematopoietic Recovery
时间窗: 1 year
Hematopoietic reconstitution post-transplantation is defined as reconstitution of both neutrophil and platelet numbers. Neutrophil reconstitution is defined as occurring on the first 3 consecutive days with an neutrophil(NEU)\>0.5×10\^9/L, and platelet (PLT) reconstitution is defined as the first \>20×10\^9/L for 3 consecutive days.
次要结局
- Incidence of primary underlying disease relapse(1 year)
- Incidence of acute toxicity(up to 100 days)
- Incidence of graft-versus-host disease(1 year)
- Incidence of infections(1 year)
研究者
Qifa Liu
Professor
Nanfang Hospital, Southern Medical University