Bayer Pharma U.S LLC, 100 Bayer Boulevard, Whippany, NJ, NJ 07981, USA; International Neonatal Consortium, USA.
Bayer Pharma U.S LLC, 100 Bayer Boulevard, Whippany, NJ, NJ 07981, USA.
Semin Fetal Neonatal Med. 2022 Aug;27(4):101331. doi: 10.1016/j.siny.2022.101331. Epub 2022 Apr 8.
Persistent pulmonary hypertension of the newborn (PPHN) is a complication of term birth, characterized by persistent hypoxemia secondary to failure of normal postnatal reduction in pulmonary vascular resistance, with potential for short- and long-term morbidity and mortality. The primary pharmacologic goal for this condition is reduction of the neonate's elevated pulmonary vascular resistance with inhaled nitric oxide, the only approved treatment option. Various adjunctive, unapproved therapeutics have been trialed with mixed results, likely related to challenges with recruiting the full, intended patient population into clinical studies. Recently, real-world data and subsequent derived evidence have been utilized to improve the efficiency of various pediatric clinical trials. We aim to provide recent perspectives regarding the use of real-world data in the planning and execution of pediatric clinical trials and how this may facilitate more streamlined assessment of future therapeutics for the treatment of PPHN and other neonatal conditions.
新生儿持续性肺动脉高压（PPHN）是足月出生的一种并发症，其特征是由于正常的产后肺血管阻力降低失败而导致持续的低氧血症，具有短期和长期发病率和死亡率的风险。这种情况的主要药物治疗目标是通过吸入一氧化氮降低新生儿的肺血管阻力，这是唯一被批准的治疗选择。各种辅助性的、未经批准的治疗方法已经进行了试验，但结果喜忧参半，这可能与将完整的、预期的患者群体招募到临床试验中存在挑战有关。最近，真实世界的数据和随后得出的证据已被用于提高各种儿科临床试验的效率。我们旨在提供有关在儿科临床试验的规划和实施中使用真实世界数据的最新观点，以及这如何促进对未来治疗 PPHN 和其他新生儿疾病的治疗方法的更精简评估。
