Urologic Management to Preserve Renal Function Protocol - Component C

摘要 脊柱裂（SB）新生儿和幼儿的最佳泌尿外科管理仍不明确。 早期评估脊髓脊膜膨出（MMC）患儿的神经性膀胱是直观的 通过尿动力学检查和随后的干预，对肾损伤高危人群进行SB检查， 有益，但很少有数据证明优于观察。泌尿系统管理，以保护 脊柱裂幼儿的初始肾功能方案（UMPIRE）研究旨在密切 评估和随访MMC新生儿，以保留初始肾功能，并允许这些儿童 以最好的肾脏和膀胱结果达到成年。创建了一个迭代方案， 在这些孩子出生后的前五年，按照一套方案进行前瞻性随访。杜克综合学校 SB诊所被选为首批九个中心之一，将新生儿纳入启动的研究 2014年，部分原因是杜克SB人群的规模和多样性，部分原因是临床和科学 他是杜克大学小儿泌尿科团队的一名专家。杜克大学的临床科学家们在 本方案的实施、初步评价、分析和传播;此外，杜克泌尿科团队 一直积极参与SB活动，如脊柱裂协会的专业咨询 理事会，表明我们致力于改善与SB生活的个人及其家庭的生活。 至关重要的是，UMPIRE研究应扩展到继续评估杜克目前招募的儿童。 UMPIRE分析的最初计划是，UMPIRE方案的前5年将侧重于早期 膀胱功能和尿路感染的发展。第二个五年期将侧重于 肠和膀胱的预后，而第三个5年期将更多地关注肾脏的预后， 确定泌尿外科手术的最佳适应症。目前，没有一名入学儿童达到 5岁，这意味着第一个5年区块的分析迄今为止尚未完成。继续 五岁以上和学龄期的数据确定将使UMPIRE研究人员能够 更好地确定议定书对实现积极健康的长期益处和可能风险 我们继续参与UMPIRE研究的主要目的是 1）根据方案继续收集既往入组患者的纵向数据，2） 继续接触所有3个月或以下的MMC婴儿进行入组，以及3）继续 我们的临床科学家在完善数据收集、数据分析和传播方面的领导作用 的发现。新提出的具体目标包括证明尿动力学分类的可靠性 在UMPIRE研究中使用，确定超声肾长度测量是否有助于 对患有SB的幼儿进行纵向随访，并检查研究参与者中UTI的发病率， 确定抗生素预防的实践是否应该扩大或缩小。

ABSTRACT The optimal urologic management of newborns and young children with spina bifida (SB) remains undefined. It is intuitive that early assessment of the neuropathic bladder in children with the myelomeningocele (MMC) form of SB by urodynamic testing and subsequent intervention for those at high risk for renal injury would be beneficial, but little data exist to prove superiority over observation. The Urologic Management to Preserve Initial Renal Function Protocol for Young Children with Spina Bifida (UMPIRE) study was conceived to closely evaluate and follow newborns with MMC in order to preserve initial renal function and allow these children to reach adulthood with the best possible renal and bladder outcomes. An iterative protocol was created to prospectively follow these children for the first five years of life on a set protocol. The Duke Comprehensive SB clinic was selected as one of the inaugural nine centers to enroll newborns into the study that was initiated in 2014 due in part to the size and diversity of Duke’s SB population and in part due to the clinical and scientific acumen of the pediatric urology team at Duke. Clinical-scientists at Duke have been instrumental in the implementation, initial evaluation, analysis, and dissemination of this protocol; in addition, Duke’s urology team has been heavily involved in SB activities, such as the Spina Bifida Association’s Professional Advisory Council, demonstrating our commitment to improving the lives of individuals living with SB and of their families. It is critical that the UMPIRE study be extended to continue to assess the currently enrolled children at Duke. The initial plan for UMPIRE analyses was that the initial 5 years of the UMPIRE protocol would focus on early bladder function and development of urinary tract infections. The second 5-year period will be focused on bowel and bladder continence outcomes, while the third 5-year period will focus more on renal outcomes and defining the most appropriate indications for urologic surgeries. Currently, no enrolled children have reached five years of age, meaning that the analyses of the first 5-year block are thus far not yet complete. Continued data ascertainment beyond five years of age and into school-aged years will allow UMPIRE researchers to better determine the long-term benefits and possible risks of the protocol towards achieving positive health outcomes for young children with SB. The primary aims for our continued participation in the UMPIRE study are 1) to continue to collect longitudinal data on previous enrolled patients according to the protocol, 2) continue to approach all infants with MMC who are 3 months old or younger for enrollment, and 3) continue the leadership role of our clinical-scientists in the refinement of data collection, analysis of data, and dissemination of findings. Specific newly proposed aims include demonstrating the reliability of the urodynamic categorization used in the UMPIRE study, determining if sonographic renal length measurements are helpful in the longitudinal follow up of young children with SB, and examining the incidence of UTI in study participants to determine if the practice of antibiotic prophylaxis should be expanded or contracted.