1/2 Hydrocortisone for BPD Respiratory and Developmental Outcomes Study (HYBRiD Outcomes Study): Clinical Coordinating Center

1/2 氢化可的松治疗 BPD 呼吸和发育结果研究（HYBRiD 结果研究）：临床协调中心

• 批准号: 9370603
• 负责人: Sara Bonamo DeMauro
• 金额: $ 31.94万
• 依托单位: CHILDREN'S HOSP OF PHILADELPHIA
• 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 2018-08-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/9370603
• 关键词: 5 year old; Address; Affect; Age; Behavior; Bronchopulmonary Dysplasia; Budgets; Categories; Child; Clinical; Clinical Trials; Cognitive; Collaborations; Consensus; Counseling; Data; Data Coordinating Center; Development; Developmental Coordination Disorders; Enrollment; Ensure; Evaluation; Exposure to; Failure; Follow-Up Studies; Future; Health; Home environment; Hybrids; Hydrocortisone; Infant; Intelligence quotient; Intervention Studies; Knowledge; Learning; Life; Lung; Lung diseases; Measures; 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; Oscillometry; Outcome; Outcome Measure; Outcome Study; Parents; Participant; Phenotype; Placebos; Population; Premature Infant; Quality of life; Recording of previous events; Records; Regimen; Research; Research Personnel; Respiratory Failure; School-Age Population; Schools; Services; Severities; Spirometry; Testing; Time; United States National Institutes of Health; Voting; Walking; base; cohort; economic outcome; exercise capacity; experience; follow-up; functional disability; functional outcomes; health economics; high risk; improved; improved outcome; infancy; instrument; motor impairment; neonate; novel; peer; placebo controlled study; prevent; primary outcome; respiratory; secondary outcome; skills

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）影响多达一半的早产儿，并且与之相关 具有严重的不良呼吸，发育，教育和健康经济成果。 Nichd BPD试验的新生儿研究网络（NRN）氢化可的松已经招募了计划总计800 在安慰剂对照试验的氢化可的松（HC）试验中，BPD高风险的早产婴儿。提议 “用于BPD呼吸和发育（混合）结果研究的氢化可的松研究”是基于这一持续的 临床试验以表征该已建立的功能性呼吸和发育结果 校正年龄或早期年龄的5岁时试用人群。功能评估，这是不可能的 在学龄前之前表演，为父母和学校提供有关孩子长处的现实情况 并在每天的情况下挑战，以便提供适当的服务以防止学校失败。 该拟议的试验随访利用了（1）大量，描述的，审判的儿童人群 患有严重的新生儿肺部疾病，一直遵循2岁的年龄； （2）支持 NRN基础架构，（3）NRN在学校年龄审判后续攻击和合作中的出色记录 NHLBI和（4）NRN数据协调中心（DCC）的长期成功关系 RTI和临床协调中心（CCC）和NRN研究人员。 CCC和DCC都有 大型新生儿临床试验的行为和随访方面的广泛专业知识和经验。 混合结果研究将使用早期年龄发展的新型功能评估 和呼吸结局，以评估HC暴露和肺部疾病严重程度的影响。主要 功能障碍的结果被定义为可以直接测量的四个结果中的任何一个 儿童：认知延迟，运动障碍，学术延迟或功能运动能力不佳 6分钟的步行测试（6MWT）。混合结果研究的特定目的是在NRN HC内用于BPD 在5岁时进行的试验评估：（1）HC暴露对功能障碍率的影响； （2）BPD的严重程度之间的关系（基于BPD的分类NIH共识定义） 和功能障碍。混合结果研究的功能性呼吸结局，这是新颖的 该人群将通过一部分中心的参与者的脉冲振荡来验证。 这项研究在NRN一致支持下，将解决有关的几个关键知识差距 基于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的咨询立即影响 单位中的新生儿和出院后。此外，这些数据对于未来的发展至关重要 干预研究旨在改善早产儿的呼吸衰竭的长期结局。