Comp C-National Spina Bifida Patient Registry/Urological Component

描述（由申请人提供）：脊柱裂（SB）是美国最常见的永久性残疾出生缺陷。这种情况对每个患者的损害程度存在广泛的范围。此外，SB涉及多个器官系统。因此，对这些病人的护理是复杂的，需要多位专家，并且必须根据每个病人在每个器官系统中的损伤程度进行个体化治疗。这使得很难建立以证据为基础的算法来管理脊柱裂患者，这些算法主要是回顾性的和不完整的数据。由于膀胱神经支配的丧失，脊柱裂可抑制膀胱的正常功能。这可能导致膀胱高压，久而久之会导致肾积水、膀胱输尿管反流、尿失禁、尿路感染和肾功能不全。SB患者泌尿外科治疗的主要目标是保存肾功能。次要目标包括预防尿路感染和实现可接受的尿失禁。不同的机构和地区对新生儿和患有SB的幼儿的管理实践模式各不相同。目前，关于治疗这些与肾功能有明确结果的患者的方案的前瞻性数据有限。本研究的主要目的是在10个不同的机构中实施和评估一套婴幼儿SB的泌尿系统管理和随访方案。5岁时的肾功能测量将作为治疗的主要结果

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.