Sirolimus+Ruxolitinib+Mycophenolate Mofetil Regimen for Prophylaxis of Acute Graft-versus-host Disease (aGvHD) in Patients Receiving Haploidentical Hematopoietic Stem Cell Transplantation (Haplo-HSCT) Who Are Intolerant to Calcineurin Inhibitor
试验速览
- 阶段
- 1 期
- 状态
- 尚未招募
- 入组人数
- 40
- 主要终点
- Incidence of Grade 2-4 aGVHD within 100 days post transplantation
研究概览
简要总结
Graft-versus-host disease (GVHD) is an important complication after transplantation, with an incidence of 40-60%, which can increase non-relapse mortality if poorly controlled. At present, the standard prophylaxis for GVHD is cyclosporine combined with methotrexate. However, calcineurin inhibitors (CNI) can cause some vital side effects, which are not tolerated by some patients. Therefore, this study aims to explore the safety and efficacy of Sirolimus in combination with Ruxolitinib and Mycophenolate Mofetil for the prophylaxis of GVHD in patients with haplo-HSCT who are intolerant to calcineurin inhibitors.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Prevention
- 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
- •Primary disease: hematological malignancies (including acute leukemia, myelodysplastic syndromes), nonmalignant disorders (including severe aplastic anaemia)
- •Renal injury or inability to tolerate the side effects of CNI: such as CNI renal toxicity (creatinine levels above the upper limit of normal), uncontrolled hypertension, and neurotoxicity rrom the time of hematopoietic stem cell infusion until +90 days after transplantation
- •Receiving haplo-HSCT for the first time
排除标准
- •Allergy or intolerance to study drugs
- •Active infection
- •Active GVHD
- •Transplantation-associated thrombotic microangiopathy
- •Key organ dysfunction: liver injury (total bilirubin more than 2 upper limit of normal) or heart injury (symptomatic heart failure or ejection fraction<50%)
- •Eastern Cooperative Oncology Group (ECOG) score >2
- •Expected survival time <30 days
- •Patients could not cooperate
研究组 & 干预措施
Sirolimus+Ruxolitinib+Mycophenolate mofetil (MMF)+anti-thymocyte globulin (ATG)
Patients receiving haplo-HSCT who are intolerant to calcineurin inhibitors would receive Sirolimus+Ruxolitinib+MMF+ATG (SRMA) for prophylaxis of aGVHD
干预措施: Sirolimus (Drug)
Sirolimus+Ruxolitinib+Mycophenolate mofetil (MMF)+anti-thymocyte globulin (ATG)
Patients receiving haplo-HSCT who are intolerant to calcineurin inhibitors would receive Sirolimus+Ruxolitinib+MMF+ATG (SRMA) for prophylaxis of aGVHD
干预措施: Ruxolitinib (Drug)
Sirolimus+Ruxolitinib+Mycophenolate mofetil (MMF)+anti-thymocyte globulin (ATG)
Patients receiving haplo-HSCT who are intolerant to calcineurin inhibitors would receive Sirolimus+Ruxolitinib+MMF+ATG (SRMA) for prophylaxis of aGVHD
干预措施: ATG (Drug)
Sirolimus+Ruxolitinib+Mycophenolate mofetil (MMF)+anti-thymocyte globulin (ATG)
Patients receiving haplo-HSCT who are intolerant to calcineurin inhibitors would receive Sirolimus+Ruxolitinib+MMF+ATG (SRMA) for prophylaxis of aGVHD
干预措施: MMF (Drug)
结局指标
主要结局
Incidence of Grade 2-4 aGVHD within 100 days post transplantation
时间窗: Participants will be followed for an expected average of 100 days post transplantation
次要结局
- Transplant-related mortality(Participants will be followed for an expected average of 1 year)
- Overall survival(Participants will be followed for an expected average of 1 year)
- Incidence of cytomegalovirus (CMV) and Epstein-Barr virus (EBV)(Participants will be followed for an expected average of 1 year)
- Incidence of chronic GVHD (cGVHD) within 1 year post transplantation(Participants will be followed for an expected average of 1 year)
- Incidence of thrombotic microangiopathy within 1 year post transplantation(Participants will be followed for an expected average of 1 year)
- Cumulative incidence of relapse(Participants will be followed for an expected average of 1 year)
研究者
Xiao-Jun Huang
Chief of the Department of Hematology
Peking University People's Hospital