Research Approaches to Improve the Care and Outcomes of People Living with Spina Bifida- Component C

改善脊柱裂患者的护理和结果的研究方法 - 成分 C

• 批准号: 10441079
• 负责人: David B Joseph
• 金额: $ 2.5万
• 依托单位: UNIVERSITY OF ALABAMA AT BIRMINGHAM
• 依托单位国家: 美国
• 财政年份: 2019
• 资助国家: 美国
• 起止时间: 2019-09-01 至 2024-08-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/10441079
• 关键词: Research; Approaches; Improve; Care; Outcomes

Project Summary- Component C 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% 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 hostile bladder function. The proposed Urologic Management to Preserve Initial Renal Function Protocol for Young Children with Spina Bifida (UMPIRE Protocol) from birth through the age of 10 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 previous funding cycle provided the framework for the UMPIRE protocol and described the treatment regimen for the child with spina bifida from birth through the first 5 years of life. Continuing to follow these children for the next 5 years, until age 10, will be critically important in validating the success of the protocol as written. The University of Alabama at Birmingham (UAB) has enrolled 35 patients in the UMPIRE project which represents 100% of patients born with spina bifida at our site. 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 is sent electronically to the CDC for analysis. Changes to the protocol may occur when it becomes apparent 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. In addition to continuing to provide data points on the 35 patients currently enrolled, UAB also intends to continue patient enrollment of all newborns born with spina bifida at our site. Finally, UAB has proposed a project to determine the incidence of bladder hostility in patients enrolled in the National Spina Bifida Patient Registry prior to the beginning of the UMPIRE protocol and compare that with patients enrolled in the UMPIRE.

项目摘要----构成部分C 目前，新生儿和幼儿脊柱裂的泌尿外科治疗是基于 教条和“专家意见”，在确定“最佳实践”标准时是一个重大的限制。是 接受>90%的脊柱裂新生儿具有正常的肾功能和正常的肾脏外观。 如果无人照料，大约50%的人在5岁时会遇到某种程度的肾脏损害。那里 需要对脊柱裂新生儿和幼儿的泌尿科护理进行标准化， 管理协议，将保护肾功能和控制敌对膀胱功能。拟议 保留早期肾功能的小儿脊柱裂泌尿外科治疗方案 （UMPIRE议定书）从出生到10岁提供了机会，以发展一个有证据的- 基于模型的儿童继发于脊柱裂的神经性膀胱的护理。期望的 该方案的结果是保持和保护正常的肾功能，同时以成本利用资源， 高效时尚 上一个供资周期为UMPIRE议定书提供了框架，并描述了 脊柱裂患儿从出生到5岁的治疗方案。继续按照 这些孩子在未来5年，直到10岁，将是至关重要的，在验证的成功， 协议如所写。伯明翰的亚拉巴马大学（UAB）在UMPIRE中招募了35名患者 该项目代表了我们研究中心100%的先天性脊柱裂患者。 在整个方案期间，膀胱功能的测试沿着进行， 评估肾功能和外观。持续收集数据，并发送 以电子方式发送给疾控中心进行分析当方案发生明显变化时，可能会对其进行变更 可以改善肾脏健康 一个成功的结果具有普遍的吸引力，并有可能使儿童护理标准化， 神经源性膀胱功能障碍在所有神经性疾病，其中脊柱裂弥补最大的 根据病因学的百分比。这不仅对大型中心非常重要， 患有神经性异常膀胱的儿童，但也适用于没有患者容量的小型中心 这将使他们能够根据自己的数据对管理做出任何合理的预测。在 除了继续提供目前招募的35名患者的数据点外，UAB还打算继续 我们研究中心所有患有脊柱裂的新生儿的患者入组。最后，UAB提出了一个项目， 确定在国家脊柱裂患者登记处登记的患者中膀胱敌意的发生率 在UMPIRE方案开始之前，并将其与入组UMPIRE的患者进行比较。