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 Baricitinib 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
- 进行中（未招募）
- 入组人数
- 150
- 试验地点
- 1
- 主要终点
- Estimate the probability of developing acute GVHD 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 and Baricitinib in children and young adults with hematoloblastosis after myeloablative conditioning regimen with treosulfan/TBI, cyclophosphamide/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 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
Baricitinib 4 mg/day per os (patient age > 9 years), 2 mg/day (patient age < 9 years), from day -3 to day +90 (after HSCT), orally, once 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
- •following diseases:
- •acute lymphoblastic,
- •myeloblastic,
- •biphenotypic,
- •bilinear leukemia,
- •malignant lymphoma,
- •myelodysplastic syndrome,
- •voluntary fully HLA-matched unrelated,
- •related haploidentical donors
- •Clinical center: National Medical Research Center of pediatric haematology, oncology and immunology named after Dmitry Rogachev" of the Ministry of Health of Russia
- •Availability of consent to conduct a study
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
研究组 & 干预措施
Baricitinib and Cyclophosphamide
Baricitinib 4 mg/day per os (patient age > 9 years), 2 mg/day (patient age < 9 years), from day -3 to day +90 (after HSCT), orally, once a day.
干预措施: Baricitinib (Drug)
结局指标
主要结局
Estimate the probability of developing acute GVHD stage II-IV after HSCT
时间窗: 100 days after HSCT
side effects of conditioning
时间窗: 100 days after HSCT
Explore the safety based on an assessment of the frequency of occurrence of: - severe (3-5 degrees) side effects of conditioning during 1 month
transplant-associated mortality
时间窗: 100 days after HSCT
次要结局
- Probability of engraftment of leukocyte(after HSCT by day + 100)
- Probability of reactivation of CMV(12 months after HSCT)
- Probability of reactivation of AdV(12 months after HSCT)
- cumulative probability of relapse(up to 2 years after HSCT)
- event-free survival(up to 2 years after HSCT)
- Probability of engraftment of platelet(after HSCT by day + 100)
- Probability of reactivation of EBV(12 months after HSCT)
- Probability of reactivation BK(12 months after HSCT)