Comp C-National Spina Bifida Patient Registry/Urological Component

描述(申请人提供)：脊柱裂(SB)是美国最常见的永久性致残出生缺陷。这种情况对每个患者的损害程度是很广泛的。此外，SB还涉及多个器官系统。因此，这类患者的护理是复杂的，需要多名专家，并且必须根据每个患者在每个器官系统内的损害程度进行个体化。这使得创建基于证据的算法来管理脊柱裂患者变得困难，这些患者大多具有追溯性和不完整的数据可依赖。由于失去正常的膀胱神经支配，脊柱裂可抑制正常的膀胱功能。这会导致膀胱压力升高，随着时间的推移，可能会导致肾积水、膀胱输尿管返流、尿失禁、尿路感染和肾功能不全。SB患者的泌尿外科治疗的首要目标是保护肾功能。次要目标包括预防尿路感染和实现可接受的尿控。不同机构和地区在管理新生儿和患有SB的儿童方面的做法模式各不相同。目前关于治疗这些患者的方案的前瞻性数据有限，与肾功能相关的明确结果。这项研究的主要目标是实施和评估一项针对10个不同机构的患有SB的婴儿和幼儿的泌尿外科管理和随访方案。五岁时的肾功能测量将作为以下主要结果 这项研究。次要结果将包括该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全国脊柱裂患者登记所涉及的其他机构在肾功能和尿路感染方面的结果进行比较。这项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，以促进SB患者的健康改善。

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.