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

描述(申请人提供)：脊柱裂(SB)是一种先天性神经缺陷，可影响胎儿、成长中的儿童和成人的多个器官系统。SB是在怀孕第一个月结束时神经管闭合不全以及脊膜和神经突出所致。脊髓脊膜膨出是最严重的脊髓脊膜膨出，脊髓和神经穿过脊髓缺损处，暴露在表面。这会导致广泛的损伤，影响许多身体系统。特别是在泌尿科，从出生到脊髓脊膜膨出闭合，中心之间的护理差异就被认识到了。众所周知，90%以上的儿童脊髓脊膜膨出有神经源性膀胱，如果不治疗，神经源性膀胱的后遗症可能会随着时间的推移而导致肾功能衰竭。婴儿的泌尿外科护理可能会主动识别并开始早期治疗，或者可能更具观察性，只有在肾脏开始受损时才开始治疗。全国SB患者登记(NSBPR)是由疾病控制和预防中心(CDC)于2008年发起的，目前已从全国19个SB诊所收集了4000多名患者的数据。这一试点项目展示了多个SB中心合作登记SB患者并收集相关患者数据的能力，以改善对SB患者的集体护理。(2)登记的下一个合乎逻辑的步骤是检查 应用多中心协作数据收集模式在SB患者护理中的关键问题。疾控中心最近招募了儿科泌尿外科和肾病学专家来制定一项针对脊髓脊膜膨出新生儿的前瞻性标准化泌尿外科管理方案(UPPF)，该方案将在出生后的头五年内进行。美国各地受影响的新生儿数量太少，无法在前瞻性治疗和观察性治疗之间进行随机、前瞻性对照试验，但对标准化方案的定期评估应该允许确定干预措施的必要性和规律性，如成像、尿动力学测试和实验室测试，以保护成长中儿童的肾功能。本项目建议测试在杜克大学实施UPPF的可行性，评估接受UPPF的儿童在出生后五年内肾脏和膀胱功能的长期泌尿系统结果，并确定在这五年内跟踪UPPF的所有组成部分的必要性，以最大限度地实现保存肾功能的目标 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.