Improving the Urologic Care and Outcomes of Young Children with Spina Bifida in Wisconsin

改善威斯康星州脊柱裂幼儿的泌尿科护理和结果

• 批准号: 10220761
• 负责人: Elizabeth B Roth
• 金额: $ 2.5万
• 依托单位: CHILDREN'S HOSPITAL OF WISCONSIN
• 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/10220761
• 关键词: Improving; Urologic; Care; Outcomes; Young

PROJECT SUMMARY/ABSTRACT In 2014, the Urologic Management to Preserve Initial Renal Function Protocol (UMPIRE Protocol) was introduced arising from a collaborative, evidence-based effort from experts in urological and renal management of children with spina bifida. This application represents an effort to continue and expand upon the ongoing UMPIRE Protocol assessments within the National Spina Bifida Patient Registry. This protocol offers a prime opportunity to validate and standardize care thresholds for infants and young children with spina bifida. Furthermore, work is needed to expand the protocol beyond the first 5 years of life. This application proposes to build upon existing data collection within the UMPIRE Protocol. The Spina Bifida Clinic at the Children’s Hospital of Wisconsin (CHW) would be an ideal collaborator for the next 5-year grant cycle. The clinic sees on average at least 11 new infants per year with spina bifida. Further, the Division of Pediatric Urology at CHW has been actively engaged in participant recruitment and prospective data capture for other urological diseases, including bladder exstrophy, setting a high standard for approach and recruitment. Therefore, the infrastructure and volume at CHW will ensure a robust participant enrollment effort throughout the study period. As an active member of the UMPIRE Protocol, we would propose multiple avenues of further study. First, by retrospectively validating the urodynamic risk thresholds defined in the first iteration of the protocol and prospectively assessing renal outcomes based on these risk thresholds, we will assess the clinical impact of the current UMPIRE Protocol through the first 5 years of implementation. Second, we will assess quality of life in participants on protocol, focusing caregiver burden for those parents and caregivers of infants and young children with spina bifida. These assessments are currently underutilized in the spina bifida population and will provide insight into the burden, including protocol burden, for these families. These will be important additional outcome measures when assessing utility and potential modifiable elements of the protocol. Finally, using the information gathered in the above summarized research approach, we will identify opportunities for improvements within the protocol. These improvements will include (a) potential additions or modifications to improve clinical outcomes in high risk children; (b) potential subtractions or omissions to the protocol to minimize caregiver burden and cost, when such studies would be felt to lack additive value; and (c) extend the protocol to children aged 6–10, with focus on continence outcomes and surgical procedures in addition to the aforementioned renal outcomes.

项目总结/摘要 2014年，泌尿科管理以保留初始肾功能方案（UMPIRE方案） 介绍了来自泌尿和肾脏管理专家的协作，循证努力 脊柱裂的儿童。这项申请代表了一项努力，继续和扩大正在进行的 国家脊柱裂患者登记系统中的UMPIRE方案评估。该协议提供了一个主要的 有机会验证和标准化患有脊柱裂的婴幼儿的护理阈值。 此外，还需要开展工作，将该议定书扩大到生命的头5年。本申请提出 以UMPIRE协议内现有的数据收集为基础。儿童脊柱裂诊所 威斯康星州医院（CHW）将是下一个5年资助周期的理想合作者。诊所看到 平均每年至少有11名新生儿患有脊柱裂。此外，CHW儿科泌尿科 积极参与其他泌尿外科的参与者招募和前瞻性数据采集 该项目为治疗包括膀胱外翻在内的各种疾病制定了高标准的方法和招募标准。因此 CHW的基础设施和数量将确保在整个研究期间进行稳健的受试者招募工作。 作为《UMPIRE议定书》的积极成员，我们将提出进一步研究的多种途径。一是通过 回顾性验证方案第一次迭代中定义的尿动力学风险阈值， 基于这些风险阈值前瞻性评估肾脏结局，我们将评估 目前的UMPIRE议定书通过第一个5年的实施。其次，我们将评估生活质量 在方案参与者中，将照顾者负担重点放在婴幼儿的父母和照顾者身上， 患有脊柱裂的儿童。这些评估目前在脊柱裂人群中未得到充分利用， 深入了解这些家庭的负担，包括方案负担。这些将是重要的补充 评估方案的效用和潜在可修改要素时的结果测量。最后利用 在上述总结的研究方法中收集的信息，我们将确定机会， 协议内的改进。这些改进将包括：（a）可能增加或修改 改善高危儿童的临床结局;（B）方案的潜在删减或遗漏， 在认为这些研究缺乏附加价值时，尽量减少照顾者的负担和费用;以及（c） 6-10岁儿童的治疗方案，重点是除了 上述肾脏结果。