Comp B-National SB Patient Registry at Vanderbilt Children's Hospital

描述（由申请人提供）：医疗保健的进步延长了患有脊柱裂的婴儿的寿命，脊柱裂是美国最常见的永久性残疾出生缺陷。然而，长期的健康结果是有限的，实践模式各不相同，最佳实践也没有得到很好的界定。美国疾病控制与预防中心已经证明了国家脊柱裂患者登记处（NSBPR）的可行性。范德比尔特Monroe Carell Jr.儿童医院的脊柱裂项目（MCJCHV）每年评估200多名脊柱裂患者。MCJCHV的神经外科、骨科和泌尿外科项目为本研究设定了以下目标：1)接近我们项目中每个符合条件的患者进行登记，2)在5年的研究期间提供关于同意NSBPR患者的健康状况、临床护理和结果的纵向数据，3)评估NSBPR数据以回答假设驱动的问题，特别是关于脊柱裂患者脊髓栓系的多学科识别和管理以及患者从青春期到成年期的健康差异。PI将与临床研究协调员一起确保提交给CDC的数据的质量控制。PI和高级/关键人员在其他国家数据库和联邦资助的赠款中具有生产性研究的证明记录。已发表的研究结果将有助于为脊柱裂的最佳临床实践指南提供信息。

DESCRIPTION (provided by applicant): Advances in medical care have increased longevity for babies born with spina bifida, the most common permanently disabling birth defect in the United States. However, long-term health outcomes are limited, practice patterns vary, and best practices are not well defined. The CDC has demonstrated the feasibility of a National Spina Bifida Patient Registry (NSBPR). The Spina Bifida Program at Monroe Carell Jr. Children's Hospital at Vanderbilt (MCJCHV) evaluates over 200 patients with spina bifida each year. The neurosurgery, orthopedics, and urology programs at MCJCHV have set the following goals for this study: 1) to approach each eligible patient in our program for enrollment, 2) to contribute longitudinal data on health status, clinical care, and outcomes for consented patients to the NSBPR over the five year study period, and 3) to evaluate NSBPR data to answer hypothesis-driven questions specifically about multi-disciplinary identification and management of tethered cord in spina bifida patients and about health disparities as patients transition from adolescence to adulthood. The PI along with the clinical research coordinator will ensure quality control of data submitted to the CDC. The PI and senior/key personnel have a demonstrated record of productive research with other national databases and federally funded grants. Published results from this study will help to inform best practice clinical guidelines for spina bifida.