Comp B-Natl SB Patient Registry and Urologic Management of Young Children with SB- Duke Project

描述（申请人提供）：超过166，000名美国人患有脊柱裂（SB），这是一种先天性神经缺陷，可影响胎儿，成长中的儿童，国家SB患者登记处（NSBPR）于2009年由疾病控制和预防中心（CDC）发起，建立一个全国性的SB诊所网络，通过一个共同的登记处连接起来，以提供1）全国SB患者健康状况的更清晰的现状，2）更好地描述美国SB患者的人口统计数据，3）评估全国SB诊所单独和集体提供的护理，4）回答假设驱动的问题，以评估临床干预对SB患者健康和生活质量的影响，5）确定未来合作研究的机会; 6）可能为SB患者制定国家护理标准。自2011年以来，杜克大学一直是NSBPR的数据收集网站，并在头两年从其SB诊所招募了225名患者。鉴于我们的SB诊所规模庞大，包括相对较高比例的成年患者，以及我们在促进NSBPR整体目标相关活动中的参与，我们认为我们继续参与NSBPR非常重要，并可能促进其成功。该项目建议在我们的研究中心进一步扩大NSBPR的入组人数，通过继续向我们的患者管理新的、扩展的问卷来增强迄今为止在NSBPR中收集的信息，继续与我们在CDC的同事合作，使用增强的数据来回答假设驱动的问题，合并关于人口统计学和泌尿外科管理的简单数据，以便在国家层面上向泌尿外科界介绍，并启动额外的研究，调查SB患者泌尿外科手术的具体结果，包括泌尿系统疾病、患者独立性和最终的肾功能。

DESCRIPTION (provided by applicant): More than 166,000 Americans are living with Spina Bifida (SB), a congenital neurologic defect that can affect multiple organ systems in the fetus, growing child, and adult and is the most common permanently disability birth defect in the U.S. The National SB Patient Registry (NSBPR) was initiated in 2009 by the Centers for Disease Control and Prevention (CDC) to create a national network of SB clinics linked through a common registry to provide 1) a clearer current picture of the health of SB patients around the country, 2) better describe the demographics of Americans living with SB, 3) assess the care being delivered at SB clinics both individually and collectively across the country, 4) answer hypothesis-driven questions to assess the impact of clinical interventions upon the health and quality of life of individuals with SB, 5) identify opportunities for future collaborative research and 6) potentially develop national standards of care for individuals with SB. Duke University has been a site of data collection for the NSBPR since 2011, and enrolled 225 patients from its SB clinic in the first two years. Given the large size of our SB clinic, including a relatively lare proportion of adult patients, as well as our proven participation in activities related to furtherig the aims of the NSBPR as a whole, we believe that our continued participation in the NSBPR is important and likely to enhance its success. This project proposes to further expand enrollment in the NSBPR at our site, enhance the information collected in the NSBPR thus far by continuing to administer new, expanded questionnaires to our patients, continue to work with our colleagues at CDC to use the enhanced data to answer hypothesis-driven questions, coalesce simple data on demographics and urologic management for presentation to the urology community on a national level, and initiate additional studies investigating specific outcomes of urologic surgery in SB patients including urinary continence, patient independence, and ultimately renal function.