Thiotepa Incorporating TBI/Cy Conditioning Regimen Followed by HSCT for EBV-HLH With Central Nervous System Involvement: a Prospective Single-arm Clinical Study
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 入组人数
- 20
- 试验地点
- 1
- 主要终点
- The clinical remission rate
研究概览
简要总结
EBV-HLH is the most common in virus-associated hemophagocytic syndrome (VAHS). There are no uniform clinical criteria for the diagnosis of CNS-HLH. And there is still a lack of international consensus on the treatment for CNS-HLH. Following allogeneic HSCT after HLH induction therapy is recommended for CNS-HLH. One of the major factors influencing the transplantation effect is conditioning. Therefore, we conduct a prospective clinical study to explore the efficacy and safety of thiotepa incorporating TBI/Cy conditioning regimen followed by allogeneic hematopoietic stem cell transplantation for EBV-HLH with central nervous system involvement.
详细描述
HLH is an immune disorder characterized by a potentially fatal cytokine storm induced by uncontrolled T lymphocyte and macrophage activation. EBV-HLH is the most common in virus-associated hemophagocytic syndrome (VAHS). There are no uniform clinical criteria for the diagnosis of CNS-HLH. And there is still a lack of international consensus on the treatment for CNS-HLH. CNS involvement is one of the factors in the poor prognosis for HLH. Following allogeneic HSCT after HLH induction therapy is recommended for CNS-HLH. One of the major factors influencing the transplantation effect is conditioning. Therefore, we conduct a prospective clinical study to explore the efficacy and safety of thiotepa incorporating TBI/Cy conditioning regimen followed by allogeneic hematopoietic stem cell transplantation for EBV-HLH with central nervous system involvement.
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
- 18 Years 至 60 Years（Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Diagnosed as HLH according to HLH-04 diagnostic criteria.
- •a significant increase of EBV-DNA copies/ml in peripheral blood or tissue or EBER(+) in tissue specimens or peripheral blood.
- •Cerebrospinal fluid EBV-DNA ≥5×10^2 copies/ml.
- •Estimated survival time ≥ 1 month.
- •Age 18~60， gender is not limited.
- •Total bilirubin ≤ 2 times the upper limit of normal; serum creatinine ≤ normal value before study entry.
- •Serum HIV antigen or antibody negative.
- •HCV antibody negative.
- •HBsAg negative, HBcAb negative. If any of the above is positive, then HBV-DNA titer in peripheral blood is required less than 1×102 copies/ml before enrollment.
- •Cardiac ultrasound LVEF≥50%.
- •Women in the child-bearing period must not be pregnant as determined by a pregnancy test and must be willing to use effective contraception during the trial and for ≥ 12 months after the last dose. Pregnant and lactating women cannot participate. All male subjects accept contraception during the trial and for ≥6 months after the last dose.
- •Signed informed consent.
排除标准
- •Heart function above grade II (NYHA).
- •Active bleeding of the internal organs(digestive tract, lung, brain, etc.).
- •Uncontrolled infection (pulmonary infection, intestinal infection, etc.).
- •Severe mental illness.
- •Patients are unable to comply during the trial and/or follow-up phase.
- •Participate in other clinical research at the same time.
研究组 & 干预措施
single-arm
a prospective single-arm clinical study
干预措施: Thiotepa (Drug)
结局指标
主要结局
The clinical remission rate
时间窗: 24 weeks
The number of cases with complete response (CR) and partial response (PR) after transplantation as a percentage of the total cases.
次要结局
未报告次要终点
研究者
Zhao Wang
Professor and Head of hematology
Beijing Friendship Hospital