Prediciting Severe Neonatal Hyperbilirubinemia

描述（由申请人提供）：该研究职业发展奖的总体目标是促进罗恩凯伦博士作为一个独立的以患者为导向的临床研究人员的发展。为了实现这一目标，Keren博士制定了一个全面的计划，将导师职位与J. Sanford Schwartz，M.D.，卫生服务研究和临床流行病学方面的正式教学培训，以及原创性研究的表现。这种教学和实践研究经验的结合将使Keren博士能够扩展和加强他在奖学金培训中获得的技能。Keren博士希望利用这一奖项来确立自己在儿科结果和卫生服务研究方面的学术领导者地位，并在预测建模和经济评估方面拥有专业知识。 博士Keren的兴趣是技术评估和公共卫生政策，因为它们与新生儿筛查和疾病预防有关，特别是与严重新生儿高胆红素血症和核黄疸的预测和预防有关。研究申请的具体目标有三个方面。首先，他将开发一个简单实用的临床预测规则，儿科医生和产科医生可以用来预测足月和近足月新生儿出院后发生严重新生儿高胆红素血症的风险。其次，临床预测规则将在一个独立的样本种族和地理上不同的新生儿，从而证明其普遍性。最后，将开发一个成本效益模型，以模拟使用替代预测模型预防极端高胆红素血症和核黄疸的经济成本和临床危害和益处。未来的研究项目和赠款申请将旨在评估这些预测模型的有效性和成本效益，再加上在实际的托儿所设置特定风险的预防服务。 目前的预防服务包括对出院后被指定为严重新生儿高胆红素血症高风险的婴儿进行更密切的门诊随访。然而，可以预防高危婴儿高胆红素血症的药物（例如锡-中卟啉）的即将可用性使得开发有效的预测规则以对婴儿进行风险分层的应用特别及时、相关和重要。

DESCRIPTION (provided by applicant): The overall goal of this research career development award is to promote the development of Dr. Ron Keren as an independent patient-oriented clinical researcher. To achieve this goal, Dr. Keren has framed a comprehensive program that combines a preceptorship with J. Sanford Schwartz, M.D., formal didactic training in health services research and clinical epidemiology, and performance of original research. This combination of didactic and practical research experiences will allow Dr. Keren to expand and strengthen the set of skills he acquired in his fellowship training. Dr. Keren hopes to use this award to establish himself as an academic leader in pediatric outcomes and health services research, with an expertise in predictive modeling and economic evaluation. Dr. Keren's interest is in technology assessment and public health policy as they relate to newborn screening and disease prevention in general, and to the prediction and prevention of severe neonatal hyperbilirubinemia and kernicterus in particular. The specific aims of the research application are three-fold. First, he will develop a simple and practical clinical prediction rule that pediatricians and neonatologists can use to predict term and near-term newborns' risk of developing severe neonatal hyperbilirubinemia after discharge from the hospital. Second, the clinical prediction rule will be validated in an independent sample of ethnically and geographically diverse newborns, thus demonstrating its generalizability. And finally, a cost-effectiveness model will be developed to simulate the economic costs and clinical harms and benefits of using alternative prediction models in the prevention of extreme hyperbilirubinemia and kernicterus. Future research projects and grant submissions will be aimed at evaluating the effectiveness and cost-effectiveness of these prediction models when coupled with risk-specific preventive services in actual nursery settings. Current preventive services consist of closer out-patient follow-up for infants designated as high risk for the development of severe neonatal hyperbilirubinemia after discharge. However, the imminent availability of drugs, such as tin-mesoporphyrin, that can prevent hyperbilirubinemia in high-risk infants makes the application to develop valid prediction rules to risk stratify infants particularly timely, relevant, and important.