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

早产儿输血早期学龄随访（TOP 5）DCC

• 批准号: 10247622
• 负责人: Abhik Das
• 金额: $ 100.56万
• 依托单位: RESEARCH TRIANGLE INSTITUTE
• 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/10247622
• 关键词: 5 year old; Academic skills; Age; Age-Months; Anemia; Behavioral; Biometry; Birth Weight; Brain; Caring; Child; Childhood; Clinical; Clinical Management; Clinical Research; Cognitive; Cohort Studies; Collaborations; Companions; Custom; Data; Data Coordinating Center; Data Reporting; Data Set; Development; Emotional; Enrollment; Ensure; Environment; Erythrocyte Transfusion; Event; Exposure to; Extremely Low Birth Weight Infant; Family; Follow-Up Studies; Funding; Gestational Age; Goals; Hemoglobin; Hemoglobin concentration result; Home; Human Resources; Impairment; Infant; Information Dissemination; Informed Consent; Infrastructure; Intensive Care Units; International; Knowledge; Lead; Leadership; Learning Disabilities; Letters; Life; Logistics; Longterm Follow-up; Low Birth Weight Infant; Manuals; Mental disorders; Monitor; Morbidity - disease rate; Motor; 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; Neurologic; Neurological outcome; Newborn Infant; Outcome; Outcome Study; Outcomes Research; Parents; Patients; Performance; Phase; Population; Premature Infant; Principal Investigator; Process; Productivity; Protocols documentation; Randomized; Randomized Controlled Trials; Recording of previous events; Records; Reporting; Research; Research Infrastructure; Research Personnel; Resources; Running; Safety; Sample Size; School-Age Population; Schools; Statistical Data Interpretation; Structure; Survival Rate; System; Time; Training; Transfusion; United States National Institutes of Health; Update; Visit; academic preparation; base; clinical center; clinical practice; cognitive function; cognitive skill; cohort; data management; data quality; design; electronic data capture system; evidence base; experience; flexibility; follow-up; functional outcomes; improved; infant outcome; meetings; neurodevelopment; neuronal circuitry; payment; premature; randomized trial; skills; success; tool

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.

项目概要 高达 95% 的早产儿在重症监护室接受红细胞 (RBC) 输注，但 目前尚不清楚更严格的输血还是更自由的输血是否会导致最佳的大脑发育。这 早产儿输血 (TOP) 试验是一项由 NHLBI 资助并得到以下机构支持的多中心研究 NICHD 新生儿研究网络 (NRN)。 TOP 试验的主要目标是评估生存率和 极低出生体重 (ELBW) 22-26 个月校正年龄神经发育障碍的发生率 随机分配至自由或限制性红细胞输血阈值的婴儿。审判开始了 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。 虽然运动和认知功能的主要缺陷可能在 22-26 个月大时被发现，但这些婴儿 年龄太小，无法评估认知、行为和协调技能，这些技能如果受损，可能会导致问题 具有家庭或学校环境、条件下的学术技能、运动表现或适应性功能 这种情况在这一人群中更为普遍，并给儿童及其家庭带来了巨大的发病率。 由于最佳输血管理是新生儿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个不同组的婴儿的功能性神经发育结果 TOP 试验中 5 岁校正年龄的输血阈值。 TOP 5 研究临床协调中心 (CCC) 由联合 PI 博士 Peg Nopoulos 领导，他拥有十年的研究经验 研究接受自由或限制性输血的早产儿结果的历史，以及萨拉博士 DeMauro 拥有与以下机构合作进行多中心结果研究的丰富经验 NICHD NRN 和 RTI International 的数据协调中心 (DCC)。 DCC PI 博士 Abhik Das 还 领导 NICHD NRN 的 DCC。 NRN 在学龄成果研究和 与 NHLBI 富有成效的合作历史。因此，TOP 5 研究经过精心设计，旨在 利用现有成功的研究基础设施、关系和资源，以减少 冗余并确保成功。 TOP 5 研究的结果将提供以下证据： 新生儿输血方法（宽松或限制）可最大限度地减少对脆弱神经元的损害 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。