National Spina Bifida Patient Registry and Urologic Management of Young Children with Spina Bifida - Component B

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ABSTRACT Spina bifida (SB) is the most common permanently disability birth defect and affects more than 166,000 Americans. Incomplete closure of the neural tube during fetal life results in impairments to multiple organ systems including the nervous, musculoskeletal, urinary, and gastrointestinal systems. Affected individuals can have a range of negative health outcomes such as alterations in neurocognitive development, mobility, renal function, urinary and fecal continence, skin integrity, reproduction, and overall quality of life. The National Spina Bifida Patient Registry (NSBPR) was developed by the Centers for Disease Control (CDC) to provide 1) a clearer current picture of the health of SB patients across the US, 2) better describe the demographics of Americans with SB, 3) assess the care being delivered at SB clinics both individually and collectively around the US, 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) develop national standards of care for individuals with SB. In the first seven years of participation in the NSBPR, the research team at Duke has enrolled and collected longitudinal data on more than 460 individuals with SB towards these goals. Duke clinician-scientists have been leaders in refinement and data analysis for the NSBPR, resulting in numerous publications and presentations. This research has produced significant insights that result in better health outcomes for individuals with SB. The continued participation of the Duke SB clinic and their clinician-scientists in the NSBPR is needed to help achieve the laudable and obtainable goals set forth by the CDC over the next five years. Specifically, the aims of the Duke clinician-scientists include: 1) continued collection of longitudinal data on currently enrolled patients, 2) continued recruitment of new patients into the registry, 3) continued refinement of definitions of urinary and fecal continence, 4) longitudinal analysis of continence in individual patients to better evaluate efficacy of treatment modalities including bowel management and bladder neck surgical procedures, 5) assess variations in surveillance of renal function in patients over time, and 6) help develop and execute a smaller collaborative study of genetic determinants of SB.