Comp C-Improving the Care and Outcomes of People Living with Spina Bifida, Component C

描述（由申请人提供）：脊柱裂新生儿和幼儿的泌尿外科管理目前是基于教条和“专家意见”，这是确定“最佳实践”标准的一个重大限制。一般认为，90%以上的脊柱裂新生儿肾功能正常，肾脏外观正常。如果不加注意，大约50%的儿童在5岁时会遇到某种程度的肾脏损害。有必要对脊柱裂新生儿和幼儿的泌尿外科护理进行标准化，使用管理协议，以保护肾功能并控制有害的膀胱功能。建议的泌尿系统方案保护脊柱裂儿童从出生到5岁的肾功能，提供了机会，以发展一个有证据的基础上的模式，为照顾儿童继发于脊柱裂的神经源性膀胱。该方案的预期结果是保持和保护正常的肾功能，同时以具有成本效益的方式利用资源。 该方案遵循患有脊柱裂的chld从出生到生命的前5年，他们最脆弱的生长期，重点放在最初的新生儿评估和预测哪个孩子有肾损伤风险的能力上。处于危险中的儿童开始接受一系列规定的治疗计划，包括间歇性地插入膀胱导管以消除尿液和药物治疗， 有助于扭转膀胱对肾脏的有害影响。在整个方案期间，沿着进行膀胱功能测试以及肾功能和外观评估。数据是在持续的基础上收集的，将以电子方式发送给CDC进行分析。该方案是动态的，如果这些变化明显会改善肾脏健康，则可能会发生变化。 一个成功的结果具有普遍的吸引力，并有可能标准化所有神经性疾病中神经源性膀胱功能障碍儿童的护理，其中脊柱裂占病因学的最大百分比。这不仅对治疗神经性异常膀胱儿童的大型中心非常重要，而且对那些没有足够患者数量的小型中心也非常重要，这些患者数量无法让他们根据自己的数据对管理做出任何合理的预测。

DESCRIPTION (provided by applicant): The urologic management of the newborn and young child with spina bifida is currently based on dogma and "expert opinion", a significant limitation when it comes to determining "best practice" standards. It is accepted that >90% of newborns with spina bifida have normal kidney function and normal appearing kidneys. If unattended; approximately 50% children encounter some degree of kidney compromise by the age of 5 years. There is a need for standardization of the urologic care of the newborn and young child with spina bifida using a management protocol that will protect kidney function and control harmful bladder function. The proposed Urologic Protocol for the Protection of Kidney Function in children with spina bifida from birth through the age of 5 years provides the opportunity to develop an evidenced based model for the care of children with a neurogenic bladder secondary to spina bifida. The desired outcome of the protocol is to preserve and protect normal kidney function while utilizing resources in a cost efficient fashion. The protocol follows the chld with spina bifida from birth through the first 5 years of life, their most vulnerable growth period Emphasis is placed on the initial newborn evaluation and the ability to predict which child is at risk for kidney damage. Children at risk are started on a combination of prescribed treatment plans that include intermittently catheterizing their bladder to eliminate urine and medications to help reverse harmful bladder effects on the kidney. Throughout the duration of the protocol, testing of bladder function is undertaken along with assessment of kidney function and appearance. Data is collected on an ongoing basis that will be sent electronically to the CDC for analysis. The protocol is created to be dynamic and alterations may occur if it becomes apparent those changes would improve kidney health. A successful outcome has universal appeal and the potential to standardize the care of children with neurogenic bladder dysfunction across all neuropathic disorders of which spina bifida makes up the greatest percent based on etiology. This becomes exceedingly important not only for the large centers caring for children with a neurogenically abnormal bladder but also for smaller centers that don't have the patient volume that would allow them to make any reasonable predictions on management based on their own data.