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.

项目摘要 高达95%的早产儿在重症监护病房接受红细胞(RBC)输血，但它 目前尚不清楚更严格的输血或更自由的输血是否会导致最佳的大脑发育。这个 早产儿输血试验(TOP)是一项多中心研究，由NHLBI资助，由 新生儿研究网络(NRN)。TOP试验的主要目标是评估存活率和 极低出生体重(ELBW)校正年龄22-26个月神经发育损害率 将婴儿随机分为自由或限制性红细胞输注阈值。审判开始了 2012年12月注册，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个月龄时被检测到，但这些婴儿 年龄太小，不能评估认知、行为和协调能力，如果这些技能受损，可能会导致问题 具有学习技能、运动能力或适应家庭或学校环境、条件 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新生儿科的一个关键知识缺口，目前提出的 TOP 5研究将评估随机分为两组的婴儿的功能神经发育结果 在5岁校正年龄的顶级试验中的输血阈值。 排名前五的研究临床协调中心(CCC)由联合PIS Peg Nopoulos博士领导，他有十年的 研究接受自由或限制性输血的早产儿结局的历史和萨拉博士 DeMauro，他拥有与 NICHD NRN和RTI国际数据协调中心(DCC)。DCC派Abhik Das博士也 领导NICHD NRN的DCC。NRN在学龄期成果研究和 与NHLBI进行富有成效的协作的历史。因此，排名前5位的研究被精心设计为 利用现有成功的研究基础设施、关系和资源，以减少 冗余，确保成功。排名前五的研究结果将提供证据，说明 新生儿输血方法(自由或限制性)可将对脆弱神经元的损害降至最低 循环，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。