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: 10165348
• 负责人: Sara Bonamo DeMauro
• 金额: $ 26.64万
• 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 2024-08-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/10165348
• 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; Questionnaires; 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; clinical center; 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 trial; 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.

项目概要 支气管肺发育不良 (BPD) 影响多达一半的极度早产儿，并且与 对呼吸系统、发育、教育和健康经济产生重大不利影响。 新生儿研究网络 (NRN) 氢化可的松治疗 BPD 试验已招募 652 名，计划总数 800 名 在一项氢化可的松 (HC) 安慰剂对照试验中，早产儿患有 BPD 高风险。 “氢化可的松治疗 BPD 呼吸和发育 (HYBRiD) 结果研究”建立在这一正在进行的基础上 临床试验来表征该已建立的功能性呼吸和发育结果 5 岁校正年龄或早期学龄的试验人群，无法进行功能评估。 在学龄前进行，为家长和学校提供有关孩子优势的真实情况 以及日常场景中的挑战，以便提供适当的服务以防止学业失败。 这项拟议的试验后续行动利用了 (1) 大量、描述良好的儿童试验人群 患有严重新生儿肺病并随访至校正年龄 2 岁的人； (2) 的支持； NRN 基础设施，(3) NRN 在学龄试验后续行动和与以下机构合作方面的出色记录 NHLBI，以及 (4) NRN 数据协调中心 (DCC) 之间长期的成功关系 RTI 和临床协调中心 (CCC) 以及 NRN 研究人员 CCC 和 DCC 都有。 在大型新生儿临床试验的进行和随访方面拥有丰富的专业知识和经验。 HYBRiD 结果研究将使用针对学龄早期发育的新颖功能评估 和呼吸系统结果，以评估 HC 暴露和肺部疾病严重程度的影响。 功能障碍的结果被定义为可以直接在系统中测量的四种结果中的任何一种 儿童：认知迟缓、运动障碍、学业迟缓或功能锻炼能力差 6 分钟步行测试 (6MWT) 的 HYBRiD 结果研究的具体目标是在 BPD 的 NRN HC 内。 5 岁校正年龄试验旨在评估：(1) HC 暴露对功能损伤发生率的影响； (2) BPD 严重程度之间的关系（基于 NIH BPD 的分类共识定义） HYBRiD 结果研究的功能性呼吸结果在以下方面是新颖的。 该人群将通过脉冲振荡法在部分中心的参与者中进行验证。 这项研究得到了 NRN 的一致支持，将解决有关 新生儿呼吸衰竭儿童的早期学龄结局，基于 BPD 的严重程度和 HC 暴露以及呼吸和发育结果之间的关系。 将对新生儿重症监护病房父母的咨询和管理产生直接影响 此外，这些数据对于新生儿在病房内和出院后的发展至关重要。 干预研究旨在改善呼吸衰竭早产儿的长期结局。