Comp C-National Spina Bifida Patient Registry/Urological Component

Comp C-国家脊柱裂患者登记处/泌尿科部分

• 批准号: 9319022
• 负责人: PAULA RUTH PETERSON
• 金额: $ 2.05万
• 依托单位: IHC HEALTH SERVICES, INC.
• 依托单位国家: 美国
• 财政年份: 2014
• 资助国家: 美国
• 起止时间: 2014-09-01 至 2019-08-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/9319022
• 关键词: Comp; National; Spina; Bifida; Patient

DESCRIPTION (provided by applicant): Spina bifida (SB) is the most common permanently disabling birth defect in the United States. There exists a broad spectrum in the degree to which this condition impairs each individual patient. In addition, SB involves multiple organ systems. As a result, the care of such patients is complex, requires multiple specialists and must be individualized to each patient's degree of impairment within each organ system. This has made it difficult to create evidence-based algorithms for management of spina bifida patients with mostly retrospective and incomplete data to rely on. Spina bifida can inhibit proper bladder function due to loss of normal bladder innervation. This can result in high bladder pressures, which over time can lead to hydronephrosis, vesicoureteral reflux, incontinence, urinary tract infections and renal insufficiency. The primary goal of urologic management in patients with SB is preservation of renal function. Secondary goals include prevention of urinary tract infection and achieving acceptable urinary continence. Practice patterns in the management of newborns and small children with SB vary across different institutions and regions. There are currently limited prospective data on protocols to manage these patients with clear outcomes related to renal function. The primary goal of this study is to implement and evaluate a protocol for the urologic management and follow- up of infants and young children with SB across 10 different institutions. Measures of renal function at five years of age will serve as the primary outcome for the study. Secondary outcomes will include cost- effectiveness of the protocol and frequency of urinary tract infections. Results of this prospective study will be used to compare with outcomes in renal function and urinary tract infection at other institutions involved in the recently create National Spina Bifida Patient Registry. This proposal supports the overall mission of the Centers for Disease Control and Prevention by helping protect this vulnerable population and establishing standards of care that will promote improved health among patients with SB.

描述（由申请人提供）：脊柱裂（SB）是美国最常见的永久致残性出生缺陷。这种情况对每个病人的损害程度有很大的范围。此外，SB涉及多个器官系统。因此，这类患者的护理是复杂的，需要多名专家，并且必须针对每个患者在每个器官系统内的损伤程度进行个性化。这使得它很难创建基于证据的算法来管理脊柱裂患者，主要是回顾性和不完整的数据依赖。脊柱裂可以抑制正常膀胱神经支配的损失，由于膀胱功能正常。这可能导致膀胱压力高，随着时间的推移，可能导致肾积水，膀胱输尿管反流，尿失禁，尿路感染和肾功能不全。SB患者的泌尿外科管理的主要目标是保护肾功能。次要目标包括预防尿路感染和达到可接受的尿路感染。不同机构和地区的新生儿和幼儿SB管理实践模式各不相同。目前关于管理这些患者的方案的前瞻性数据有限，具有与肾功能相关的明确结局。本研究的主要目的是实施和评价一项方案，用于10个不同机构的婴幼儿SB的泌尿外科管理和随访。5岁时的肾功能指标将作为以下研究的主要结局： 书房次要结果将包括方案的成本效益和尿路感染的频率。这项前瞻性研究的结果将用于与最近创建的国家脊柱裂患者登记处的其他机构的肾功能和尿路感染结果进行比较。该提案支持疾病控制和预防中心的总体使命，帮助保护这一弱势群体，并建立护理标准，促进SB患者的健康改善。