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

描述(由申请人提供)：医疗护理的进步增加了出生时患有脊柱裂的婴儿的寿命，脊柱裂是美国最常见的永久性致残出生缺陷。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定义。疾控中心已经证明了建立全国脊柱裂患者登记系统(NSBPR)的可行性。小门罗·卡雷尔的脊柱裂项目。范德比尔特儿童医院(MCJCHV)每年评估200多名脊柱裂患者。MCJCHV的神经外科、整形外科和泌尿外科项目为这项研究设定了以下目标：1)接近我们计划中每个符合条件的患者进行登记，2)在五年的研究期间向NSBPR提供关于健康状况、临床护理和同意患者的结果的纵向数据，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的多学科鉴定和脊髓栓系的管理，以及关于患者从青春期向成年期过渡时的健康差异。PI将与临床研究协调员一起确保提交给疾控中心的数据的质量控制。私营部门和高级/关键人员在利用其他国家数据库和联邦政府资助的赠款进行卓有成效的研究方面有良好的记录。这项研究发表的结果将有助于为脊柱裂的最佳实践提供临床指南。

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.