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

德克萨斯儿童医院/贝勒医学院针对脊柱裂幼儿 (UMPIRE) 进行的泌尿外科管理以保留其初始肾功能方案

• 批准号: 10650196
• 负责人: Susan Jarosz
• 金额: $ 2.5万
• 依托单位: BAYLOR COLLEGE OF MEDICINE
• 依托单位国家: 美国
• 财政年份: 2019
• 资助国家: 美国
• 起止时间: 2019-09-01 至 2024-08-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/10650196
• 关键词: Urologic; Management; Preserve; Initial; Renal

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.

项目摘要 脊柱裂（SB）是儿童中最常见的永久致残性出生缺陷。1仅在美国， 超过150，000人患有SB及其对多个器官系统（包括泌尿系统）的长期衰弱影响。 道。对于新生儿SB的泌尿外科管理，保留上尿路，从而避免 通过适当的膀胱管理永久性丧失肾功能一直是儿科患者的关键管理目标， 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， 尿动力学，以及治疗选择，如清洁间歇性导管插入术（CIC）和抗胆碱能药物 药物治疗这通过膀胱循环促进逼尿肌松弛和发育，并确保膀胱 引流以保护上尿路和预防肾积水。2然而， 管理记录不足，特别是在假定肾损害风险较低的患者中， 缺乏对系列成像/测试的时间以及诸如CIC和 在新生儿SB管理中引入抗胆碱能药物。此外，前瞻性的纵向数据，这些 患者不存在肾功能以及生活质量指标（例如尿/粪便 失禁），使得难以评价任何管理方案的功效。 该项目的长期目标是改善儿童SB患者的临床管理。 继续实施和评价保留初始肾功能的泌尿系统管理（UMPIRE） 在疾病预防控制中心制定的脊柱裂幼儿方案，最终导致了标准化的 新生儿和婴幼儿SB的高效和有效的尿路护理管理方案， 至少在生命的前五年及以后的肾功能。我们的中心假设是， 管理方案将建立有效的临床管理，保护长期肾功能，促进 有效利用卫生保健资源，并降低当前治疗算法的发病率。的理由 拟议的研究是，研究验证了其他临床领域的标准化管理方案， 纤维化和先天性心脏病改善了医疗保健的临床结果和成本效益 22，23德克萨斯儿童医院/贝勒医学院一直是UMPIRE协议的领先网站， 2015年，通过追求以下具体目标，检验中心假设并接近本提案的目标： 继续参与CDC注册网站，将监测和评估SB幼儿的UMPIRE方案。 由于在我们的机构的庞大和独特的人口，登记到国家登记处将包括产前关闭 患者和种族多样的患者，包括西班牙裔背景的患者。