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 Calcineurin Inhibitor at Children and Young Adults With Hemoblastosis After Hematopoietic Stem Cell Transplantation From an Unrelated or Haploidentic Donor
试验速览
- 阶段
- 3 期
- 状态
- 进行中（未招募）
- 入组人数
- 56
- 试验地点
- 2
- 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 calcineurin inhibitor 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 Cyclophosphamide 50 mg/kg/course on the days -3, -2 or 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 100 mg/kg/course on the days +3, +4 Abatacept 10 mg/kg/day on the days +5, +14, +28, +45, +60, +90, +120 Vedolizumab 10 mg/kg/day, max. 300 mg on the days -1, +14, +28 Cyclosporine A or Tacrolimus 3 mg/kg/day from -1 to 120 (in case of high risk of relapse: patients with JMML, without of remission o positive MRD after HSCT)/180 days or Ruxolitinib (in case of intolerance to calcineurin inhibitors) 5m/day for patients <12 years old and 10 mg/day for patients >12 years old by scheme of CNI.
Donor selection criteria
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%
- •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
Estimate the probability of developing acute GVHD stage II-IV after HSCT
时间窗: evaluation period is 120 days after HSCT
severe (3-5 degrees) side effects of conditioning
时间窗: evaluation period is 30 days after HSCT
次要结局
- transplantation-associated mortality(up to 100 days after HSCT)
- pathogen-specific immunoreconstitution(after HSCT up to 180 days)
- relapse free survival(up to 100 days after HSCT)
- event free survival(up to 100 days after HSCT)
- reactivation of CMV(after HSCT up to 180 days)