Prediciting Severe Neonatal Hyperbilirubinemia

描述(申请人提供)：该研究职业发展奖的总体目标是促进Ron Keren博士作为一名独立的、以患者为导向的临床研究人员的发展。为了实现这一目标，科伦博士制定了一个全面的项目，其中包括与J·桑福德·施瓦茨医学博士的指导、卫生服务研究和临床流行病学方面的正式教学培训，以及原创研究的执行。这种教学和实际研究经验的结合将使科伦博士能够扩大和加强他在团契培训中获得的一套技能。克伦希望利用这个奖项，确立自己在儿科成果和医疗服务研究领域的学术领袖地位，并在预测建模和经济评估方面拥有专业知识。 克伦博士对技术评估和公共卫生政策感兴趣，因为它们与新生儿筛查和疾病预防有关，特别是与预测和预防严重的新生儿高胆红素血症和核黄体症有关。申请研究的具体目的有三个方面。首先，他将开发一种简单实用的临床预测规则，儿科医生和新生儿专家可以使用该规则来预测足月和近期新生儿出院后发展为严重新生儿高胆红素血症的风险。第二，临床预测规则将在种族和地理上不同的新生儿的独立样本中进行验证，从而证明其普适性。最后，将开发一个成本-效果模型，以模拟在预防极端高胆红素血症和核黄斑病变中使用不同预测模型的经济成本和临床危害以及收益。未来的研究项目和赠款申请将旨在评估这些预测模型在与实际托儿所环境中针对风险的预防服务相结合时的有效性和成本效益。目前的预防服务包括对被指定为出院后发展为严重新生儿高胆红素血症高危婴儿的更密切的门诊随访。然而，可以预防高危婴儿高胆红素血症的药物即将问世，这使得开发有效的预测规则来对婴儿进行危险分层变得尤为及时、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.