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: 8918273
• 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/8918273
• 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性出生缺陷。在这种情况下损害每个患者的程度上存在广泛的范围。此外，SB涉及多个器官系统。结果，此类患者的护理很复杂，需要多个专家，并且必须在每个器官系统内对每个患者的损害程度进行个性化。这使得难以创建基于证据的算法来管理脊柱裂裂，这些患者主要是回顾性和不完整的数据。由于正常膀胱神经神经缺失，脊柱裂可以抑制适当的膀胱功能。这可能导致高膀胱压力，随着时间的流逝，这会导致肾结通，囊泡分流，尿失禁，尿路感染和肾脏不足。 SB患者泌尿科管理的主要目标是保存肾功能。次要目标包括预防泌尿道感染和实现可接受的尿道。在不同机构和地区的新生儿和小孩的管理中，实践模式各不相同。目前，关于管理这些患者的方案的预期数据有限，与肾功能相关的明确结果。这项研究的主要目标是在10个不同机构中实施和评估泌尿外科管理方案和跟进SB的婴儿和幼儿。五岁时肾功能的度量将成为 研究。次要结果将包括尿路感染方案和频率的成本效益。这项前瞻性研究的结果将用于与最近创建的国家脊柱裂片患者注册中的其他机构的肾功能和尿路感染的结果进行比较。该提案通过帮助保护这一脆弱人群并建立护理标准来支持疾病控制和预防中心的整体使命，从而促进SB患者的健康状况。