Comp C-Urologic Management to Preserve Renal Function Protocol - Duke Project

描述（由申请人提供）：脊柱裂（SB）是一种先天性神经系统缺陷，可影响胎儿、生长期儿童和成人的多个器官系统。SB是由妊娠第一个月结束时神经管闭合不完全和脊髓膜和神经突出引起的。脊髓脊膜膨出是SB的最严重形式，其中脊髓和神经穿过脊髓缺损并暴露在表面上。这会导致影响许多身体系统的广泛损伤。特别是在泌尿外科，从出生和脊髓脊膜膨出闭合开始就认识到中心之间的护理差异。众所周知，超过90%的脊髓脊膜膨出患儿患有神经源性膀胱，如果不及时治疗，神经源性膀胱的后遗症可能导致肾功能衰竭。婴儿的泌尿科护理可以是积极主动的，以识别和开始早期治疗“肾盂”膀胱，或可能是更具观察性的，只有当肾脏开始受到损害时才开始治疗。国家SB患者登记处（NSBPR）于2008年由疾病控制和预防中心（CDC）发起，现在已经收集了来自全国19个SB诊所的4000多名患者的数据。该试点项目证明了多个SB中心协作招募SB患者并收集相关患者数据的能力，以共同改善SB患者的护理。(2)注册表的下一个逻辑步骤是检查 通过应用这种多中心协作数据收集模型，在SB患者的护理中发现关键问题。疾病预防控制中心最近招募了儿科泌尿学和肾脏学专家，制定了一项在生命的前五年内对患有脊髓脊膜膨出的新生儿进行前瞻性标准化泌尿管理的方案（保护肾功能的泌尿系统方案- UPPF）。美国受影响的新生儿数量太少，无法在主动和观察管理之间进行随机、前瞻性对照试验，但对标准化方案的定期评估应允许确定干预的必要性和规律性，如成像、尿动力学检查和实验室检查，以保护生长中儿童的肾功能。本项目旨在测试在杜克实施UPPF的可行性，评估UPPF后5年内儿童肾脏和膀胱功能的长期泌尿学结局，并确定在5年内遵循UPPF所有组成部分的必要性，以最佳方式实现保护肾功能的目标。 健康结果。

DESCRIPTION (provided by applicant): Spina Bifida (SB) is a congenital neurologic defect that can affect multiple organ systems in the fetus, growing child, and adult. SB results from incomplete neural tube closure and protrusion of spinal membranes and nerves by the end of the first month of pregnancy. Myelomeningocele is the most severe form of SB in which the spinal cord and nerves come through the spinal defect and are exposed on the surface. This results in a wide range of impairments that affect many body systems. In urology specifically, variation in care between centers is recognized beginning at birth and myelomeningocele closure. It is well known that over 90% of children with myelomeningocele have neurogenic bladder, and if untreated, the sequelae of neurogenic bladder may result in renal failure over time. Urologic care in infants may be proactive to identify and initiate early treatment of "hostil" bladders or may be more observational with initiation of therapy only when kidneys begin to suffer insult. The National SB Patient Registry (NSBPR) was initiated in 2008 by the Centers for Disease Control and Prevention (CDC) and now has collected data on over 4000 patients from 19 SB clinics across the nation. This pilot project has demonstrated the ability of multiple SB centers to work collaboratively to enroll SB patient and collect pertinent patient data in an effor to improve care of SB patients collectively.(2) The next logical step of the registry is to examine critical issues in the care of SB patients by application of this model of multi-center collaboratie data collection. The CDC recently enlisted experts in pediatric urology and nephrology to develop a protocol for prospective standardized urologic management of newborns with myelomeningocele (Urologic Protocol for the Protection of Renal Function - UPPF) to be conducted over the first five years of life. The number of affected newborns across the U.S. is too small to make feasible a randomized, prospective controlled trial between proactive and observational management, but regular evaluation of a standardized protocol should allow determinations to be made about the necessity and regularity of interventions, such as imaging, urodynamic testing, and laboratory tests, to preserve renal function in the growing child. This project proposes to test the feasibility of implementation of the UPPF at Duke, assess the long term urologic outcomes of renal and bladder function in children following the UPPF over the first five years of life, and determine the necessity of following all components of the UPPF during the five years to optimally achieve the goal of preservation of renal function as a positive health outcome.