A Prospective Randomized Controlled Study for Intervention of Prolonged Isolated Thrombocytopenia After Allogeneic Hematopoietic Stem Cell Transplantation: N-acetyl-L-cysteine (NAC) Versus Supportive Therapy
试验速览
- 阶段
- 2 期
- 状态
- 终止
- 入组人数
- 15
- 试验地点
- 1
- 主要终点
- response rate
研究概览
简要总结
Isolated prolonged thrombocytopenia (PT) is a common complication after allogeneic stem cell transplantation with significant poor prognosis. No standard treatment is available. The current study assigned PT randomly to 2 arms: intervention arm with N-acetyl-L-cysteine (NAC) and control arm with supportive therapy.This is a prospective randomized controlled study.
详细描述
- patients diagnosed with PT at day 60 post transplant will be randomized assigned to intervention arm (NAC) or controlled arm (supportive therapy: prophylactic platelet transfusion was given when platelet count <20000/ul.)
- Response will be evaluated at day 90. Response was defined as platelet recovery to >= 20000/ul for 7 consecutive days without transfusion support during the enrollment period. All the other patients not achieved above criteria was defined as no response.
- For those without response in both arms, patients will received NAC plus recombinant human thrombopoietin (rhTPO) for an additional 30 days. rhTPO was given at 300IU/kg/d for 28 consecutive days or platelet > 50000/ul independent of platelet transfusion.
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 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •age>= 14 years
- •diagnosed as PT (defined as platelet count in peripheral blood never achieve 20000/miro-liter for 7 consecutive days without transfusion at day 60 post transplantation)
- •serum creatine level < ULN (upper limit of normal), serum ALT/AST /TBil<=2 ULN
- •without active CMV/EBV/ADV reactivation, active GVHD, without relapse or minimal residual disease at enrollment
排除标准
- •history of asthma
- •allergy to NAC
- •refuse to participate
研究组 & 干预措施
intervention arm
NAC 400mg p.o tid from day 60 to day 90 post transplant
干预措施: N-acetyl-L-cysteine (NAC) (Drug)
controlled arm
Supportive therapy including platelet infusion:prophylactic platelet transfusion was given when platelet count <20000/ul
干预措施: supportive therapy (Other)
结局指标
主要结局
response rate
时间窗: 30 days after the start of enrollement
rate of response after 30 days of treatment
次要结局
- non-relapse mortality(the day of last follow-up)
- grade 2-4 acute graft versus host disease(day 100 after transplant)
- overall survival(the day of last follow-up)
- chronic graft versus host disease(the day of last follow-up)
研究者
Xiaojun Huang,MD
Head of Peking University Institute of Hematology
Peking University People's Hospital