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

项目总结 支气管肺发育不良(BPD)影响多达一半的极早产儿，并与 对呼吸道、发育、教育和卫生经济产生严重不利影响。NICHD 新生儿研究网络(NRN)用于BPD的氢化可的松试验已经招募了652人，计划总共800人 氢化可的松(HC)安慰剂对照试验中BPD高危早产儿。建议数 “氢化可的松对BPD呼吸和发育(混合)结果的研究”建立在这项正在进行的研究基础上 临床试验，以表征这一已建立的功能性呼吸和发育结果 5岁校正年龄或早期入学年龄的试验人群。功能评估，这不能 在学龄前表演，为家长和学校提供一个关于孩子长处的真实图景 以及日常情景中的挑战，以便提供适当的服务，防止学校失败。 这项拟议的试验后续行动利用了(1)大量描述良好的试验儿童 患有严重新生儿肺部疾病并被追踪至2岁矫正年龄； NRN基础设施，(3)NRN在学龄期试验后续行动和与 NHLBI，以及(4)NRN数据协调中心(DCC)之间的长期成功关系 RTI和临床协调中心(CCC)和NRN调查人员。CCC和DCC都有 在进行和跟踪大型新生儿临床试验方面有丰富的专业知识和经验。 混合结果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。初级阶段 功能损害的结果被定义为四种结果中的任何一种，可以直接在 儿童：认知迟缓、运动障碍、学习迟缓或功能锻炼能力差 6分钟步行试验(6MWT)。混合结果研究的具体目标是，在BPD的NRN HC中 在校正年龄为5岁时进行试验，以评估：(1)接触HC对功能损害率的影响； 和(2)BPD严重程度之间的关系(基于NIH对BPD的分类共识定义) 和功能障碍。混合结果研究的功能性呼吸结果，这在 这一群体将在中心的子集的参与者中用脉冲振荡法进行验证。 在NRN的一致支持下，这项研究将解决关于 新生儿呼吸衰竭儿童的早期学龄期结局，基于BPD的严重程度和 接触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病房中父母的咨询和 新生儿在病房内和出院后。此外，这些数据对未来的发展将是必不可少的 干预研究旨在改善早产儿呼吸衰竭的长期结局。

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.