Neurological Diseases Research in High Risk Newborns

描述(由申请人提供)：这项建议旨在帮助Nehal Parikh博士，一位具有公认的研究技能的新生儿专家，成为对高危婴儿围产期脑损伤进行方法上强有力的临床研究的领导者。我们提出了一个全面的5年职业发展计划，包括：1)两项关于极低出生体重儿脑损伤的重要临床研究：a)一项前瞻性队列研究，对足月或近期进行定量(体积)磁共振成像(MRI)以预测18个月神经感觉损害(这项研究评估了定量MRI作为诊断测试的特性，比定性MRI和常规临床预测指标的增量预测价值，以及总脑容量和局部脑体积的预测价值)；B)对依赖呼吸机的婴儿(常规使用大剂量地塞米松治疗)进行随机试验，以评估小剂量氢化可的松治疗是否能防止此类婴儿常见的局部和总皮质体积减少。这些研究将传授神经诊断测试、神经保护策略和高危婴儿预后评估方面的研究技能。这些发现将引起人们的极大兴趣，并为K23资金结束时为R01资金铺平道路；2)密集的指导。 主要导师是在脑损伤研究方面经验丰富的新生儿学家和流行病学家(Jon Tyson，医学博士，公共卫生硕士)，NICHD新生儿研究网络的PI，NHLBI临床研究课程的PI，临床研究和循证医学中心主任，以及一位敬业和成功的导师。在上述研究中，Parikh博士还将在神经学(医学博士Lan Butler)、发展心理学和定量核磁共振(医学博士Robert Lasky)以及新生儿学和研究人员培训(医学博士、公共卫生硕士，临床研究硕士项目主任)方面找到杰出的合作导师。泰森中心和儿科将支持这些研究；3)临床研究硕士学位(由Parikh博士于9月9日入学)。2003年)。课程(36学分)包括研究伦理学、高级生物统计学和高级流行病学。他的论文将成为上述研究的主要出版物。机构优势包括美国最大的高危婴儿人口之一，强大的围产期研究计划(NICHD母婴和新生儿网络仅有的8个中心之一)，支持临床研究的新生儿ICU(去年在网络试验中随机抽取的婴儿数量最多)，以及运作良好的后续计划。拟议的计划将为Parikh博士提供研究经验、专门的指导和必要的课程，以成为围产期脑损伤临床研究的领导者。

DESCRIPTION (provided by applicant): This proposal is designed to prepare Dr. Nehal Parikh, a neonatologist with demonstrated research skills, to become a leader in performing methodologically strong clinical studies of perinatal brain injury in high-risk infants. We propose a comprehensive 5-year career development program involving: 1) Two important clinical studies of brain injury in extremely low birth weight infants: A) A prospective cohort study of quantitative (volumetric) magnetic resonance imaging (MRI) performed at or near term to predict neurosensory impairments at >= 18 months (a study evaluating the properties of quantitative MRI as a diagnostic test, the incremental predictive value over qualitative MRI and routine clinical predictors, and the predictive value of total and regional brain volumes); B) A randomized trial among ventilator-dependent infants (who are conventionally treated with high-dose dexamethasone) to assess whether low-dose hydrocortisone therapy prevents the reduced regional and total cortical volumes common among such infants. These studies will impart research skills in neurodiagnostic tests, neuroprotective strategies, and prognostic assessments for high-risk infants. The findings will be of great interest and pave the way for R01 funding by the end of K23 funding; 2) Intensive mentorship. The primary mentor is a neonatologist and epidemiologist (Jon Tyson, MD, MPH), who is experienced in studies of brain injury, a PI in the NICHD Neonatal Research Network, a PI in the NHLBI Clinical Research Curriculum, Director of the Center for Clinical Research and Evidence Based Medicine, and a dedicated and successful mentor. For the above studies, Dr. Parikh will also have prominent co-mentors in neurology (lan Butler, MD), developmental psychology and quantitative MRI (Robert Lasky, PhD), and neonatology and investigator training (Kathleen Kennedy, MD, MPH, Director, Master's Degree Program in Clinical Research). Dr. Tyson's Center and the Pediatrics Department will support these studies; 3) Master's Degree in Clinical Research (matriculated by Dr. Parikh in Sept. 2003). Courses (36 credit hrs) include Research Ethics, Advanced Biostatistics, and Advanced Epidemiology. His thesis will be a major publication from the above research. Institutional strengths include one of the largest populations of high-risk infants in the US, a strong perinatal research program (1 of only 8 centers in both the NICHD Maternal-Fetal and Neonatal Networks), a neonatal ICU supportive of clinical research (with the largest number of infants randomized in Network trials last year), and a well functioning follow up program. The proposed program will provide Dr. Parikh the research experience, dedicated mentorship, and necessary coursework to become a leader in clinical research addressing perinatal brain injury.