Transfusion of Prematurity Early School Age Follow-up (TOP 5) CCC

早产儿输血早期学龄随访（TOP 5）CCC

• 批准号: 10247613
• 负责人: Amy Lynn Conrad
• 金额: $ 53.98万
• 依托单位: UNIVERSITY OF IOWA
• 依托单位国家: 美国
• 财政年份: 2018
• 资助国家: 美国
• 起止时间: 2018-09-20 至 2025-08-31
• 项目状态: 未结题
• 来源: https://reporter.nih.gov/project-details/10247613
• 关键词: 5 year old; Academic skills; Age; Age-Months; Assessment tool; Behavioral; Brain; Bronchopulmonary Dysplasia; Budgets; Cessation of life; Child; Child Development; Classification; Clinical; Clinical Management; Cognition; Cognitive; Collaborations; Data Coordinating Center; Development; Emotional; Enrollment; Ensure; Environment; Erythrocyte Transfusion; Exposure to; Extremely Low Birth Weight Infant; Family; Funding; Future; Home; Hydrocortisone; Impairment; Infant; Intensive Care Units; International; Iowa; Knowledge; Lead; Measures; Methods; Morbidity - disease rate; Motor; Motor Skills; Movement; Multicenter Neonatal Research Network; Multicenter Studies; National Heart, Lung, and Blood Institute; National Institute of Child Health and Human Development; Neonatal; Neonatology; Network Infrastructure; Neurodevelopmental Impairment; Neurological outcome; Outcome; Outcome Measure; Outcome Study; Outcomes Research; Packed Red Blood Cell Transfusion; Pediatric Hospitals; Performance; Philadelphia; Population; Premature Infant; Randomized; Readiness; Recording of previous events; Research Infrastructure; Research Personnel; Resources; Sample Size; School-Age Population; Schools; Survival Rate; System; Testing; Time; Transfusion; Universities; Work; academic preparation; cognitive function; cognitive skill; cohort; design; experience; follow-up; functional disability; functional outcomes; improved; infant outcome; innovation; neuronal circuitry; phonology; premature; primary outcome; randomized trial; respiratory; skills; success

Project Summary Up to 95% of premature infants undergo red blood cell (RBC) transfusion while in the intensive care unit, yet it is unknown whether more restrictive or more liberal transfusions will lead to optimal brain development. The Transfusion of Prematures (TOP) Trial is a multi-center study funded by the NHLBI and supported by the NICHD Neonatal Research Network (NRN). The primary objective of the TOP Trial is to assess survival and rates of neurodevelopmental impairment at 22-26 months corrected age in extremely low birth weight (ELBW) infants that are randomized to either liberal or restrictive RBC transfusion thresholds. The trial began enrollment in December 2012 and reached the target sample size of 1,824 infants on time in April 2017. Although major deficits in motor and cognitive function may be detected at 22-26 months of age, these infants are too young to assess cognitive, behavioral, and coordination skills that, if impaired, can lead to problems with academic skills, motor performance or adaptive functioning in home or school environments, conditions that are far more prevalent in this population and create substantial morbidity for the children and their families. Because optimal transfusion management is a critical knowledge gap in neonatology, the currently proposed TOP 5 Study will assess functional neurodevelopmental outcomes of infants randomized to two different transfusion thresholds in the TOP Trial at 5 years corrected age. The TOP 5 Study Clinical Coordinating Center (CCC) is led by Co-PIs Dr. Peg Nopoulos, who has a ten-year history of studying the outcomes of premature infants exposed to liberal or restrictive transfusion, and Dr. Sara DeMauro, who has extensive experience conducting multicenter outcomes studies in collaboration with the NICHD NRN and the Data Coordinating Center (DCC) at RTI International. The DCC PI Dr. Abhik Das also leads the DCC for the NICHD NRN. The NRN has a superb track record in school-age outcomes research and history of productive collaboration with NHLBI. Thus, the TOP 5 Study has been thoughtfully designed to leverage existing successful research infrastructure, relationships, and resources in order to reduce redundancy and ensure success. The results of the TOP 5 study will provide evidence about which approach to neonatal transfusion (liberal or restrictive) minimizes damage to vulnerable neuronal circuits and, in turn, which transfusion strategy will improve both short and long-term outcomes for these vulnerable premature infants.

项目摘要 高达95%的早产儿在重症监护室接受红细胞（RBC）输注， 更严格的输血和更宽松的输血是否会导致最佳的大脑发育尚不清楚。的 早产儿输血（TOP）试验是一项多中心研究，由NHLBI资助， NICHD新生儿研究网络。TOP试验的主要目的是评估生存率， 极低出生体重儿（ELBW）22-26个月矫正年龄时神经发育障碍的发生率 随机分配至自由或限制RBC输血阈值的婴儿。审判开始 2012年12月入组，并于2017年4月按时达到1，824名婴儿的目标样本量。 虽然在22-26个月大时可能会检测到运动和认知功能的主要缺陷，但这些婴儿 年龄太小，无法评估认知、行为和协调技能，如果这些技能受损，可能会导致问题。 在家庭或学校环境中具有学术技能、运动表现或适应功能， 这些疾病在这一人群中更为普遍，并给儿童及其家庭造成了严重的发病率。 由于最佳输血管理是血液学中的一个关键知识缺口，目前提出的 TOP 5研究将评估随机分配到两种不同的婴儿的功能性神经发育结果 TOP试验中5岁校正年龄时的输血阈值。 排名前5的研究临床协调中心（CCC）由Co-PI Peg Nopoulos博士领导，他有十年的 研究早产儿暴露于自由或限制性输血的结果的历史，以及Sara博士 DeMauro，他在与美国医学会合作开展多中心结局研究方面拥有丰富的经验， NICHD NRN和RTI International的数据协调中心（DCC）。DCC PI Abhik Das博士还 领导NICHD NRN的DCC。NRN在学龄期成果研究方面有着出色的记录， 与NHLBI富有成效的合作历史。因此，TOP 5研究经过精心设计， 利用现有的成功的研究基础设施、关系和资源， 冗余，确保成功。TOP 5研究的结果将提供证据， 新生儿输血的方法（自由或限制）最大限度地减少了对脆弱神经元的损伤 回路，反过来，哪种输血策略将改善短期和长期结果， 这些脆弱的早产儿