Urologic Management to Preserve Renal Function Protocol - Component C

摘要 患有脊柱裂(SB)的新生儿和幼儿的最佳泌尿外科治疗仍未确定。 对脊髓脊膜膨出(MMC)儿童神经性膀胱的早期评估是直观的 对肾损伤高危人群进行尿动力学检测和后续干预的SB形式将是 这是有益的，但几乎没有数据能证明它优于观察。要保留的泌尿系管理 儿童脊柱裂的初步肾功能方案(裁判)研究将密切关注 评估和跟踪患有MMC的新生儿，以保护初始肾功能，并允许这些儿童 成年后，肾脏和膀胱的结局可能达到最好。创建了一个迭代协议来 在这些孩子出生后的头五年里，按照既定的程序对他们进行前瞻性的跟踪。《公爵综合体》 SB诊所被选为首批招募新生儿参加这项研究的九个中心之一 2014年，部分原因是杜克大学SB人群的规模和多样性，部分原因是临床和科学 杜克大学儿科泌尿外科团队的智慧。杜克大学的临床科学家在这方面发挥了重要作用 该方案的实施、初步评估、分析和传播；此外，杜克的泌尿科团队 一直积极参与某人的活动，如脊柱裂协会的专业咨询 这表明我们致力于改善与某人同住的个人及其家人的生活。 至关重要的是，裁判研究应继续扩大，以继续评估杜克大学目前招收的儿童。 裁判分析的最初计划是，裁判礼仪的最初5年将重点放在早期 膀胱功能与尿路感染的发展。第二个五年期间将重点放在 肠和膀胱控制结果，而第三个5年期间将更多地关注肾脏结果和 为泌尿外科手术确定最合适的适应症。目前，没有注册的儿童达到 五年，这意味着第一个五年区块的分析到目前为止还没有完成。续 超过五岁并进入学龄期的数据查明将使裁判研究人员能够 更好地确定议定书在实现积极健康方面的长期益处和可能的风险 患有某人的年幼儿童的结局。我们继续参与裁判研究的主要目的 1)继续根据协议收集以前登记的患者的纵向数据，2) 继续接触所有3个月大或3个月以下患有MMC的婴儿进行登记，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 调查结果。新提出的具体目标包括证明尿动力学分类的可靠性 在裁判研究中使用，确定超声肾脏长度测量是否有助于 对患有SB的幼儿进行纵向随访，并检查研究参与者的尿路感染发生率 确定抗生素预防的做法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.