Prospective Pilot Study of the Clinical Efficacy and Safety of the Method for Preventing a Graft-versus-host Disease Through the Agency of Using the Combination of Post-transplantation Cyclophosphamide with Abatacept, Vedolizumab and Ruxolitinib At Children and Young Adults with Hemoblastosis After Hematopoietic Stem Cell Transplantation from an Unrelated or Haploidentic Donor
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 入组人数
- 50
- 试验地点
- 2
- 主要终点
- 1. Cumulative Incidence stage II-IV after HSCT
研究概览
简要总结
GVHD prevention using a combination of post-transplantation cyclophosphamide in combination with abatacept, vedolizumab and Ruxolitinib in children and young adults with hematoloblastosis after myeloablative conditioning regimen with treosulfan/TBI, etoposide, fludarabine after HSCT from matched unrelated and haploidentical donors
详细描述
Conditioning regimen:
Treosulfan 42 g/m2/course on the days -5, -4, -3 or total body irradiation 12 Gray/course on the days -8, -7, -6 Etoposide 60 mg/kg on the days -6, -5. or Thiotepa 10 mg/kg -6,-5 Fludarabine 150 mg/m2/course on the days -6, -5, -4, -3, -2
Prevention of GVHD:
Cyclophosphamide 80 mg/kg/course on the days +3, +4 Abatacept 10 mg/kg/day on the days +5, +14, +28, +60, +90 Vedolizumab 10 mg/kg/day, max. 300 mg on the days 0, +14, +28, +60
Ruxolitinib 10 mg/m2 per os, from day -3 to day +90 (after HSCT), orally, twice a day.
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
- 1 Day 至 21 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patients under the age of 21 years with following diseases:
- •acute lymphoblastic,
- •myeloblastic,
- •biphenotypic,
- •bilinear leukemia,
- •malignant lymphoma,
- •myelodysplastic syndrome,
排除标准
- •Age over 21 years
- •Patients with ALL outside clinical and hematological remission
- •Clinical status:
- •Lansky/Karnowski index <70% (supplement No.1)
- •Heart function: left ventricular ejection fraction <40% according to ultrasound of the heart1
- •Kidney function: clearance of endogenous creatinine < 70 ml / min
- •Liver function: total bilirubin, ALT, AST, ALP > 2 norms
- •Lung function: lung capacity <50%, for children who cannot carry out of respiratory function - oxygen saturation during pulse oximetry <92%
- •Uncontrolled viral, fungal or bacterial infection.
- •Mental illness of the patient or caregivers, making it impossible to realize the essence of the study and compromising compliance with medical appointments and sanitary and hygienic regime 1 These patients may receive treatment according to the protocol, but the results will be evaluated separately
结局指标
主要结局
1. Cumulative Incidence stage II-IV after HSCT
时间窗: up to 100-day
Estimate the probability of developing acute GVHD stage II-IV after HSCT
Kaplan Meier overal survival
时间窗: up to 100 days
Explore the safety based on an assessment of the frequency of occurrence severe (3-5 degrees) side effects of conditioning- 100-day transplant-associated mortality
Kaplan Meier event free survival
时间窗: up to 100 days
Explore the safety based on an assessment of the frequency of occurrence severe (3-5 degrees) side effects of conditioning- 100-day transplant-associated mortality
次要结局
- event-free survival(up to 100 days)
- Cumulative Incidence of leukocyte engraftment(up to 30 days)
- Cumulative Incidence of platelet engraftment(up to 30 days)
- Cumulative Incidence reactivation of CMV(up to 6 mouth or up to immunreconstitution)
- box plot(up to 1 year)
- Cumulative Incidence of chronic GVHD(up to 1 year)
- Cumulative Incidence reactivation of EBV(up to 6 mouth or up to immunreconstitution)
- Cumulative Incidence reactivation of AdV(up to 6 mouth or up to immunreconstitution)
- Cumulative Incidence reactivation of HHV6(up to 6 mouth or up to immunreconstitution)