Mentorship of Early-Career Clinicians in Bronchopulmonary Dysplasia Drug Therapies

患有支气管肺发育不良(BPD)的早产儿通常死亡，幸存者患有终生疾病。 BPD是最常见的早产儿发病率，每年影响18,000名婴儿。美国国家卫生研究院将BPD定义为 在36周调整年龄时接受外源性氧气或气道正压治疗。BPD的成本是 包括社会和经济两方面，并以受损的儿童健康和生活质量、家庭压力和 经济困难，以及增加的医疗成本。因为BPD的后果是灾难性的， 新生儿科医生经常使用未经证实疗效的药物，试图预防和治疗BPD。 不幸的是，目前还没有FDA批准的预防或治疗BPD的药物。发展安全和安全 治疗BPD的有效药物是改善公共健康的理想机会，为BPD提供了发展的平台 受训者在试验设计、法规要求和严格的量化方法方面的专业知识 应用于自己的独立研究事业。 研究人员马修·M·劳恩博士，医学博士，公共卫生硕士，是一名执业的新生儿专家，公共卫生硕士学位。 流行病学和临床药理学培训。Laughon博士已经获得了学员发展的权限 北卡罗来纳大学教堂山分校的项目，包括新生儿-围产期 医学院、吉林斯公共卫生学院和伊谢曼药学院。劳恩医生还有一个 与世界上最大的学术研究组织杜克临床研究所建立了长期的学术合作伙伴关系 研究所。通过这些计划，劳恩博士将指导北卡罗来纳大学和杜克大学的实习生，并提供 学员获得流行病学、临床药理学或 药物流行病学。初级临床医生研究人员将遵循一个合理的过程，包括设计 一个原创的假设驱动的研究项目，完成教学研究，参与一对一和 小组辅导，定期评估和反馈。学员将专注于以下方面的准备和实施 新生儿肺部疾病的早期药物试验，主要是BPD。这些预期的可行性的证据是 由在量化、以患者为导向的研究方面取得成功的现任和前任受训人员提供。 确定早产儿BPD药物的PK和安全性是一项迫切的、尚未得到满足的公共卫生需求。 研究BPD的药物是解决BPD研究差距问题的理想机会 药物治疗早产儿是因为：1)患有bpd的早产儿会终生患病；2)没有 已被证明可以预防或治疗BPD的药物；3)新生儿科医生通常在 未进行适当剂量和安全性研究的早产儿；以及4)预防死亡率和发病率 与早产相关的疾病会带来终生的好处。拟议的学员主导的研究项目将使用 10种常用药物的机会性研究设计，将为学员提供发展ALL的平台 早产儿的临床研究技能和合理的药物开发方法。

Premature infants with bronchopulmonary dysplasia (BPD) often die and survivors have life-long morbidities. BPD is the most common morbidity of prematurity, affecting >18,000 infants per year. The NIH defines BPD as the receipt of exogenous oxygen or positive airway pressure at 36 weeks adjusted age. The costs of BPD are both social and economic and are measured in impaired childhood health and quality of life, family stress and economic hardship, and increased healthcare costs. Because the consequences of BPD are catastrophic, neonatologists frequently use drugs without proven efficacy in an attempt to prevent and treat BPD. Unfortunately, there are no FDA approved drugs for the prevention or treatment of BPD. Developing safe and effective drugs for BPD is an ideal opportunity to improve public health and provides a platform to develop trainees’ expertise in trial design, regulatory requirements, and rigorous quantitative methods that will be applied to their own independent research career. The investigator, Dr. Matthew M. Laughon, MD, MPH is a practicing neonatologist with an MPH in epidemiology and training in clinical pharmacology. Dr. Laughon has secured access for trainee development to The University of North Carolina at Chapel Hill programs including the Division of Neonatal-Perinatal Medicine, the Gillings School of Public Health, and the Eshelman School of Pharmacy. Dr. Laughon also has a longstanding academic partnership with the world’s largest academic research organization, the Duke Clinical Research Institute. Through these programs, Dr. Laughon will mentor trainees at UNC and Duke and provide a pathway for trainees to secure advanced training in epidemiology, clinical pharmacology, or pharmacoepidemiology. Beginning clinician investigators will follow a logical progression including designing an original hypothesis driven research project, completing didactic studies, participation in one on one and group mentoring, and regular evaluation and feedback. Trainees will focus on the preparation and conduct of early-phase drug trials in neonatal lung disease, primarily BPD. Proof of the feasibility of these expectations is provided by current and former trainees who have achieved success in quantitative, patient-oriented research. Determining the PK and safety of drugs for BPD in premature infants is an urgent, unmet public health need. Investigating drugs for BPD represents an ideal opportunity to approach the problem of the research gap of drugs in premature infants because: 1) Premature infants with BPD have life-long morbidities; 2) There are no drugs that have been proven to prevent or treat BPD; 3) Neonatologists are commonly using drugs in premature infants without appropriate dosing and safety studies; and 4) Preventing mortality and morbidities associated with premature birth has life-long benefits. The proposed trainee-led research project will use an opportunistic study design of 10 commonly used drugs that will provide a platform for trainee to develop all aspects of clinical research skills and a rational approach to drug development in premature infants.