A Prospective Study of Patients With Isolated Thrombocytopenia Following Hematopoietic Stem Cell Transplantation
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 发起方
- 入组人数
- 97
- 试验地点
- 1
- 主要终点
- Number of Participants With Platelet Count Recovery
研究概览
简要总结
Isolated thrombocytopenia is a common and severe complication of HSCT, which often leads to an increased risk of life-threatening hemorrhage, frequent requirement of platelet transfusions and extended hospital stays, representing a challenging clinical problem. Current treatments for thrombocytopenia after HSCT are frequently unsatisfactory in platelet recovery and for preventing potentially fatal bleeding complications. Therefore, it is urgent to explore an effective therapy to improve the outcomes of thrombocytopenia after HSCT. Previous studies have demonstrated that decitabine, a hypomethylating agent, may reduce platelet transfusions in myelodysplastic syndrome (MDS) patients. The investigators conducted an prospective clinical trial to evaluate the safety and efficiency of rhTPO and decitabine in the treatment of thrombocytopenia following HSCT.
详细描述
Isolated thrombocytopenia is a frequent and severe complication of hematopoietic stem cell transplantation (HSCT). It often leads to an increased risk of life-threatening hemorrhage, frequent requirement of platelet transfusions and extended hospital stays, representing a challenging clinical problem. Current treatments for thrombocytopenia after HSCT, including thrombopoietin, interleukin-11, immunoglobulin, methylprednisolone and rituximab, are frequently unsatisfactory in platelet recovery. Therefore, it is urgent to explore an effective therapy to improve the outcomes of thrombocytopenia after HSCT. Thrombopoietin (TPO) is a cytokine that drives thrombopoiesis by stimulating the differentiation of stem cells into megakaryocytes and promoting megakaryocyte proliferation and polyploidization. Decitabine was approved for the treatment of myelodysplastic syndrome (MDS) as a DNA methylation inhibitors. Studies in vitro show that decitabine enhances platelet release and megakaryocyte maturation. Here, the investigators performed a prospective clinical trial, in order to investigate the safety and efficiency of rhTPO and decitabine in the treatment of thrombocytopenia following 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
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Platelet count ≤ 30 × 109/L persistently at day 60 post-HSCT or later;
- •Neutrophil and hemoglobin were well recovered;
- •Full donor chimerism was achieved;
- •No response to conventional treatments (e.g. thrombopoietin, immunoglobulin, glucocorticoid alone or in combination) for a duration of at least 4 weeks;
排除标准
- •Patients with malignancy relapse;
- •Active infections;
- •Grade Ⅲ-Ⅳ acute GVHD or severe chronic GVHD according to National Institute of Health criteria;
- •Severe organ damage;
- •Thrombosis requiring treatment;
- •Received decitabine following the current transplantation.
研究组 & 干预措施
Experimental Group 1
Decitabine in combination with rhTPO.
干预措施: rhTPO (Drug)
Experimental Group 1
Decitabine in combination with rhTPO.
干预措施: Decitabine (Drug)
Experimental Group 2
Decitabine
干预措施: Decitabine (Drug)
Control Group
Conventional treatment except decitabine.
干预措施: Conventional Treatment (Other)
结局指标
主要结局
Number of Participants With Platelet Count Recovery
时间窗: Up to 4 weeks after the treatment
Platelet response refers to a sustained increase (stable or increasing level) of at least 30×10E9/L independent of transfusion for 3 days.
次要结局
- Megakaryocyte Count(Up to 4 weeks after the treatment)