Urologic Management to Preserve Initial Renal Function Protocol for Young Children with Spina Bifida (UMPIRE) at Texas Children's Hospital/Baylor College of Medicine

项目摘要 脊柱裂(SB)是儿童中最常见的永久性致残出生缺陷。 超过15万人与SB及其对包括尿路在内的多个器官系统的长期衰弱作用一起生活 一条小路。对于SB新生儿的泌尿外科治疗，保留上尿路从而避免 通过适当的膀胱管理永久丧失肾功能一直是儿科医生的一个关键管理目标 泌尿科医生和SB医疗保健提供者。 患有SB的新生儿通常接受观察方案，包括连续尿路成像和 尿流动力学，以及清洁间歇插管(CIC)和抗胆碱能药物等治疗选择 药物。通过膀胱循环促进逼尿肌松弛和发育，确保膀胱 引流以努力保护上尿路和防止肾积水2然而，这种做法的结果是 治疗的记录很少，特别是那些被认为肾损害风险较低的患者，而且 对于系列成像/测试的时间以及CIC和 在新生儿SB的治疗中引入抗胆碱能药物。此外，这些项目的前瞻性、纵向数据 患者不存在肾功能以及生活质量指标(例如尿/粪 大小便失禁)，使得评估任何管理方案的有效性都变得困难。 该项目的长期目标是改善儿童SB患者的临床管理。我们的目标是 继续实施和评估泌尿外科管理以保留初始肾功能(裁判) 美国疾病控制与预防中心制定的脊柱裂儿童治疗方案，最终导致标准化 新生儿和婴幼儿SB保留尿路有效护理的管理方案 至少在生命的头五年及以后的肾功能。我们的中心假设是这个标准化的 管理方案将建立有效的临床管理，保存长期的肾功能，促进 有效利用卫生保健资源，降低当前治疗算法的发病率。该计划的基本原理 建议的研究是，研究验证了其他临床领域的标准化管理方案，如膀胱癌 纤维化和先天性心脏病改善了临床结果和医疗保健的成本效益 22，23德克萨斯儿童医院/贝勒医学院一直是裁判协议的领先地点 2015通过追求以下具体目标来检验中心假设并更接近本提案的目标 继续作为疾病预防控制中心登记点参加，该登记点将监测和评估有SB的幼儿裁判员议定书。 由于我们机构庞大而独特的人口，国家登记的登记对象将包括产前关闭。 患者和不同种族的患者，包括具有西班牙裔背景的患者。

Project Summary Spina bifida (SB) is the most common permanently disabling birth defect in children.1 In the United States alone, more than 150,000 people live with SB and its long-term debilitating effects on multiple organ systems including the urinary tract. For the urologic management of newborns with SB, preservation of the upper urinary tracts and thus avoiding permanent loss of renal function via appropriate bladder management has been a key management goal for pediatric urologists and SB healthcare providers. Newborns with SB are generally placed on an observation protocol that includes serial urinary tract imaging and urodynamics, as well as treatment options such as clean intermittent catheterization (CIC) and anticholinergic medications. This promotes detrusor muscle relaxation and development by bladder cycling and ensures bladder drainage in efforts to protect the upper urinary tract and prevent hydronephrosis.2 However, the outcomes of this management are poorly documented, especially in patients who are presumed at low risk for renal damage, and there is a lack of consensus on the timing of serial imaging/tests and the timing of when interventions such as CIC and anticholinergics are introduced in SB management of the newborn. Moreover, prospective, longitudinal data for these patients does not exist with respect to renal function, as well as, quality of life measures (e.g. urinary/fecal incontinence), making evaluation of the efficacy of any management protocol difficult. The long-term goal of this project is to improve the clinical management of pediatric patients with SB. The objective is to continue implementation and evaluation of the Urologic Management to Preserve Initial Renal Function (UMPIRE) Protocol for Young Children with Spina Bifida that was developed at the CDC, ultimately leading to a standardized management protocol of efficient and effective urinary tract care in newborns and young children with SB that preserves renal function for at least the first five years of life and beyond. Our central hypothesis is that this standardized management protocol will establish effective clinical management that preserves long-term renal function, promote efficient use of health care resources, and reduce the morbidity of current treatment algorithms. The rationale for the proposed research is that research validated standardized management protocols in other clinical areas such as cystic fibrosis and congenital heart conditions have improved clinical outcomes and cost effectiveness of healthcare delivery.22,23 Texas Children's Hospital/Baylor College of Medicine has been a leading site for the UMPIRE protocol since 2015 testing the central hypothesis and coming closer to the objective of this proposal by pursuing the specific aim of continued participation as a CDC registry site that will monitor and evaluate the UMPIRE Protocol for young children with SB. Due to the large and unique population at our institution, enrollees to the national registry will include prenatal closure patients and ethnically diverse patients including those with Hispanic backgrounds.