2/2 Hydrocortisone for BPD Respiratory and Developmental Outcomes Study (HYBRiD Outcomes Study): Data Coordinating Center

2/2 氢化可的松用于 BPD 呼吸和发育结果研究（HYBRiD 结果研究）：数据协调中心

• 批准号: 10886194
• 负责人: Abhik Das
• 金额: $ 40.77万
• 依托单位: RESEARCH TRIANGLE INSTITUTE
• 依托单位国家: 美国
• 财政年份: 2017
• 资助国家: 美国
• 起止时间: 2017-09-01 至 2024-08-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/10886194
• 关键词: 5 year old; Affect; Age; Biometry; Bronchopulmonary Dysplasia; Caring; Child; Clinical; Clinical Research; Clinical Trials; Cognitive; Cohort Studies; Collaborations; Companions; Consensus; Counseling; Data; Data Coordinating Center; Data Reporting; Data Set; Development; Education; Enrollment; Ensure; Evaluation; Event; Exposure to; Failure; Follow-Up Studies; Future; Goals; Human Resources; Hydrocortisone; Impairment; Infant; Information Dissemination; Informed Consent; Infrastructure; Institution; Intervention Studies; Journals; Knowledge; Leadership; Letters; Longterm Follow-up; Low Birth Weight Infant; Lung diseases; Manuals; Measures; Monitor; Multicenter Neonatal Research Network; National Heart, Lung, and Blood Institute; National Institute of Child Health and Human Development; Neonatal; Neonatal Intensive Care Units; Network Infrastructure; Newborn Infant; Oscillometry; Outcome; Outcome Study; Parents; Participant; Patients; Peer Review; Phase; Population; Premature Infant; Process; Productivity; Protocols documentation; Publications; Qualifying; Recording of previous events; Records; Reporting; Research; Research Personnel; Resources; Respiratory Failure; Running; Safety; Sample Size; Sampling Studies; School-Age Population; Schools; Services; Severities; Statistical Data Interpretation; Structure; System; Testing; Training; United States National Institutes of Health; Update; Visit; Walking; Work; clinical center; clinical practice; cohort; data cleaning; data management; data quality; economic outcome; electronic data; evidence base; exercise capacity; experience; extreme prematurity; flexibility; follow-up; functional disability; health economics; high risk; improved; improved outcome; infancy; meetings; motor impairment; neonate; novel; payment; placebo controlled trial; prevent; primary outcome; programs; respiratory; tool

Project Summary Bronchopulmonary dysplasia (BPD) affects up to half of extremely preterm infants, and is associated with significant adverse respiratory, developmental, educational, and health economic outcomes. The NICHD Neonatal Research Network (NRN) Hydrocortisone for BPD Trial has enrolled 652 of a planned total of 800 preterm infants at high risk for BPD in a placebo controlled trial of hydrocortisone (HC). The proposed “Hydrocortisone for BPD Respiratory and Developmental (HYBRiD) Outcomes Study” builds on this ongoing clinical trial to characterize the functional respiratory and developmental outcomes of this established trial population at 5 years corrected age, or early school age. Functional assessments, which cannot be performed before early school age, provide parents and schools with a realistic picture of a child's strengths and challenges in every day scenarios, so that appropriate services can be provided to prevent school failure. This proposed trial follow-up takes advantage of (1) a large, well-described, trial population of children who had severe neonatal lung disease and are being followed until 2 years corrected age; (2) the support of the NRN infrastructure, (3) the NRN's superb track records in school age trial follow-up and collaboration with NHLBI, and (4) the long-standing successful relationship between the NRN Data Coordinating Center (DCC) at RTI and the Clinical Coordinating Center (CCC) and NRN investigators. The CCC and DCC both have extensive expertise and experience in the conduct and follow-up of large neonatal clinical trials. The HYBRiD Outcomes Study will use novel functional assessments of early school age developmental and respiratory outcomes to assess the impact of both HC exposure and severity of lung disease. The primary outcome of functional impairment is defined as any one of four outcomes that can be measured directly in the child: cognitive delay, motor impairment, academic delay, or poor functional exercise capacity as measured by the 6-minute walk test (6MWT). The HYBRiD Outcome Study specific aims are, within the NRN HC for BPD Trial at 5 years corrected age, to evaluate: (1) the impact of HC exposure on rates of functional impairment; and (2) the relationship between severity of BPD (based on the categorical NIH consensus definition of BPD) and functional impairment. The HYBRiD Outcome Study's functional respiratory outcomes, which are novel in this population, will be validated with impulse oscillometry in the participants at a subset of centers. Supported unanimously by the NRN, this study will address several critical knowledge gaps about the early school age outcomes of children with neonatal respiratory failure, based on both the severity of BPD and exposure to HC, as well as the relationships between respiratory and developmental outcomes. These results will have immediate impact on counseling of parents in the neonatal intensive care unit and management of neonates in the unit and after discharge. In addition, these data will be essential for the development of future intervention studies aimed to improve the long-term outcomes of preterm infants with respiratory failure.

项目摘要 支气管肺发育不良（BPD）影响多达一半的极早产儿， 具有显著的不利的呼吸、发育、教育和健康经济后果。NICHD 新生儿研究网络（NRN）氢化可的松用于BPD试验已招募计划总数800人中的652人 氢化可的松（HC）安慰剂对照试验中BPD高危早产儿。拟议 “氢化可的松对BPD呼吸和发育（HYBRiD）结局的研究”建立在正在进行的 临床试验，以表征功能性呼吸和发育的结果，这一建立 5岁矫正年龄或学龄早期的试验人群。功能评估，不能 在学龄前进行，为家长和学校提供一个孩子的优势的现实画面 和挑战，以便能够提供适当的服务，防止学业失败。 这项拟议的试验随访利用了（1）大量描述良好的儿童试验人群 患有严重新生儿肺部疾病并正在随访至2岁矫正年龄的患者;（2） NRN基础设施，（3）NRN在学龄试验后续行动和与 NHLBI，以及（4）NRN数据协调中心（DCC）与 RTI和临床协调中心（CCC）和NRN研究人员。CCC和DCC都有 在开展和跟进大型新生儿临床试验方面拥有丰富的专业知识和经验。 HYBRiD结局研究将使用新的学龄早期发育功能评估， 和呼吸结果，以评估HC暴露和肺部疾病严重程度的影响。主 功能障碍的结局定义为可以直接在 儿童：认知延迟、运动障碍、学业延迟或功能性运动能力差，通过以下指标测量 6分钟步行试验（6 MWT）。HYBRiD结局研究的具体目标是，在BPD的NRN HC内 在5岁矫正年龄时进行试验，以评价：（1）HC暴露对功能损害发生率的影响; 和（2）BPD严重程度之间的关系（基于BPD的分类NIH共识定义） 和功能性损伤。HYBRiD结局研究的功能性呼吸结局，这是新的， 这一人群将在一个中心子集的参与者中用脉冲多普勒测量法进行验证。 在NRN的一致支持下，这项研究将解决有关 根据BPD的严重程度和 暴露于HC，以及呼吸和发育结果之间的关系。这些结果 将对新生儿重症监护室的父母咨询和新生儿监护室的管理产生直接影响。 新生儿在单位和出院后。此外，这些数据对未来的发展至关重要。 干预研究旨在改善患有呼吸衰竭的早产儿的长期结局。