Long-Term Outcomes of NO for Ventilated Premature Babies

早产相关的呼吸衰竭是一个日益严重的公共卫生问题。尽管死亡率随着 围产期护理的进展，这种疾病消耗了大量的医疗资源，并且越来越多地与 有令人担忧的长期发病率、发育迟缓和家庭负担。吸入一氧化氮(INO)，一种选择性 肺血管扩张剂可改善呼吸衰竭足月新生儿的短期预后，可能会受益 早产儿。因此，一项由NHLBI资助的新的随机对照试验(NHLBI INO RCT)将评估 INO对800例妊娠36周后死亡或氧依赖联合终点的影响 早产相关呼吸衰竭的婴儿。 然而，早产儿相关呼吸衰竭与足月儿呼吸衰竭的病因不同。 而iNO可能影响的广泛的长期后果并没有被现有的研究所涵盖 设计。因此，我们建议扩大和加强NHLBI iNO RCT的后续行动。具体来说，我们的目标是 评估使用INO对以下方面的影响：#1.-长期临床和儿童发育结果；#2.-家庭负担， 以及；#3.早产相关呼吸衰竭的医疗费用。在目标4下，我们将使用来自目标1-3的数据 目的：评价静脉注射一氧化氮在早产儿呼吸机治疗中的成本-效果。 我们将通过增加NHLBI iNO RCT数据收集来实现这些目标：i.)对一名患者的生存随访 平均43岁；2.)在1年、2年、3年和4年对诊所进行全面、标准化的随访，以评估临床 结果、儿童发展和家庭负担；三.)每3个月对父母进行一次有组织的电话采访 在第一年和此后每六个月，平均四年评估慢性发病率和出院后 医疗保健用途；iv.)收集初级住院的详细医院账单；以及；v.)全面分析 计划。 这项研究将使我们能够确定iNO疗法在这种情况下的长期后果，帮助临床医生， 家庭和政策制定者，并立即影响到对危重婴儿的护理。通过与NHLBI iNO结合 RCT，我们利用一个重要的机会，以随机的方式收集预期的长期结果数据 时尚。我们的建议将通过更好地了解以下方面来显著提高对RCT的投资回报 从社会角度看iNO疗法的影响。新生儿重症监护在过去的十年里发生了巨大的变化 好几年了。这项研究还将提供有关早产相关的长期结局的当代信息。 现代管理下的呼吸衰竭。最后，我们的数据将允许评估早期 后续结果的替代物，这是这一领域未来研究设计的关键。

Prematurity-associated respiratory failure is a growing public health problem. Although mortality has dropped with advances in perinatal care, this condition consumes considerable healthcare resources and is increasingly associated with worrisome long-term morbidity, developmental delay, and family burden. Inhaled nitric oxide (iNO), a selective pulmonary vasodilator that improves short-term outcomes in term neonates with respiratory failure, may benefit premature infants. Consequently, a new NHLBI-funded randomized controlled trial (NHLBI iNO RCT) will assess the effect of iNO on the combined end-point of mortality or oxygen dependency at 36 weeks post conceptional age in 800 infants with prematurity-associated respiratory failure. However, prematurity-associated respiratory failure has a different etiology from respiratory failure in term infants and the wide array of long-term consequences that may be affected by iNO are not captured under the existing study design. We therefore propose to extend and enhance the follow-up of the NHLBI iNO RCT. Specifically, we aim to assess the effects of INO use on: #1. - long-term clinical and childhood developmental outcomes; #2. - family burden, and; #3. - healthcare costs of prematurity-associated respiratory failure. Under aim #4, we will use data from aims #1-3 to assess the cost-effectiveness of iNO in ventilated premature infants. We will achieve these aims by augmenting the NHLBI iNO RCT data collection with: i.) survival follow-up for an average of 43 years; ii.) comprehensive, standardized follow-up clinic visits at 1,2, 3 and 4V2 years to assess clinical outcomes, childhood development, and family burden; iii.) structured telephone interviews with parents every 3 months in year 1 and every 6 months thereafter for an average of 4¿ years to assess chronic morbidity and post-discharge healthcare use; iv.) collection of detailed hospital bills for the primary hospitalization, and; v.) a comprehensive analysis plan. This study will allow us to determine the long-term consequences of iNO therapy in this condition, aiding clinicians, families, and policymakers and immediately affecting care of critically ill infants. By combining with the NHLBI iNO RCT, we take advantage of an important opportunity to gather prospective long-term outcome data in a randomized fashion. Our proposal will significantly increase the return on investment in the RCT through a greater understanding of the impact of iNO therapy from a societal perspective. Neonatal intensive care has changed dramatically in the last ten years. This study will also provide contemporary information on the long-term outcomes of prematurity-associated respiratory failure following modern management. Finally, our data will allow assessment of the robustness of early proxies for subsequent outcomes, key for future study design in this area.