Early Detection of Endotheliopathy Post-Transplant
试验速览
- 阶段
- 不适用
- 状态
- 终止
- 入组人数
- 32
- 试验地点
- 1
- 主要终点
- Proportion of pediatric patients undergoing HCT who experience development of endothelial-related organ dysfunction syndromes
研究概览
简要总结
The goal of this research study is to learn more about possible risk factors that might be associated with side effects from stem cell transplants in people between the ages of 0 to 26 years old. Specifically, this study is looking at complications that arise from injury to the endothelium, a small layer of cells lining the blood vessels and heart. These complications can affect the heart, lungs, liver, kidneys and intestines and increase risk of severe illness needing care in the intensive care unit.
详细描述
Primary Objectives:
- To evaluate the proportion of pediatric patients undergoing Hematopoietic cell transplantation (HCT) who develop critical illness (defined by admission to the pediatric intensive care unit at St. Jude for reasons other than observation after procedure) within 100 days of such therapies.
- To evaluate the proportion of pediatric patients undergoing HCT who experience development of endothelial-related organ dysfunction syndromes.
Secondary Objectives
- To report the baseline (pre-conditioning and post-conditioning but pre-cellular therapy) levels of specified circulating biomarkers, vascular reactivity as measured by reactive hyperemia index (RHI), and to describe baseline clot structure in patients age 0-26 years of age planned to undergo HCT.
- To compare the levels of circulating biomarkers between the cohort of patients that experience critical illness (defined by admission to the pediatric intensive care unit at St. Jude for reasons other than observation after procedure) any time during the first 100 days of HCT following cellular infusion and the cohort of patients that do not ever experience critical illness in the first 100 days of HCT following cellular infusion.
- To evaluate the time to development of any complications including development of endotheliopathies, development of critical illness (defined by admission to the pediatric intensive care unit at St. Jude for reasons other than observation after procedure), development of abnormal vascular reactivity by RHI, development of abnormal clot structure, and time to peak (or trough, if applicable) level of circulating biomarkers.
The study will require a minimum of 1 blood draw before transplant and 6 blood draws throughout the first 100 days following transplant. If participants are admitted to the intensive care unit or are diagnosed with specific complications additional blood draws will be done. Peripheral arterial tonometry (PAT) testing will occur 1-2 times during the first 100 days in several older patients who can tolerate the procedure by sitting still for the duration of the testing period.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Prospective
入排标准
- 年龄范围
- — 至 26 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Age 0-26 years planned to undergo allogeneic or autologous HCT for any indication with any preparative regimen planned.
排除标准
- •Less than 10 kg at time of consent or any child in which blood volume required for scheduled blood draws would pose more than minimal risk.
- •Lack of agreement from primary HCT physician.
- •Inability or unwillingness of research participant and/or legal guardian/representative to give written informed consent.
结局指标
主要结局
Proportion of pediatric patients undergoing HCT who experience development of endothelial-related organ dysfunction syndromes
时间窗: Up to 100 days after transplant (plus or minus 3 days)
The proportion will be estimated using one-sample binomial estimates and various confidence intervals.
Proportion of pediatric patients undergoing Hematopoietic cell transplantation (HCT) who develop critical illness
时间窗: Up to 100 days after transplant (plus or minus 3 days)
The proportion of pediatric patients undergoing HCT who develop critical illness (defined by admission to the pediatric intensive care unit at St. Jude for reasons other than observation after procedure) will be estimated using one-sample binomial estimates and various confidence intervals.
次要结局
- Difference in circulating biomarkers(From Baseline blood draws up to 100 days post ICU admission)
- Difference in clot structure(From Baseline blood draws up to 100 days post ICU admission)
- Baseline clot structure(Baseline (before preparative transplant regimen starts) and Day 0 (on day of transplant, before therapy takes place))
- Baseline levels of circulating biomarkers(Baseline (before preparative transplant regimen starts) and Day 0 (on day of transplant, before therapy takes place))
- Baseline vascular reactivity(Baseline (before preparative transplant regimen starts) and Day 0 (on day of transplant, before therapy takes place))
- Difference in vascular reactivity(From Baseline blood draws up to 100 days post ICU admission)
- Time to development of any complications or critical illness(Up to 100 days after transplant (plus or minus 3 days))