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

项目摘要/摘要 2014年，《保留初始肾功能的泌尿外科管理议定书》(裁判议定书) 来自泌尿外科和肾脏管理专家的基于证据的协作努力 患有脊柱裂的儿童。此应用程序代表了继续和扩展正在进行的 国家脊柱裂患者登记处内的裁判协议评估。该协议提供了一个素数 有机会验证和标准化对患有脊柱裂的婴幼儿的护理阈值。 此外，还需要开展工作，将议定书扩展到生命的头5年以外。此应用程序建议 在《裁判议定书》现有数据收集的基础上再接再厉。儿童脊柱裂诊所 威斯康星医院(CHW)将是下一个5年赠款周期的理想合作者。诊所正在看病。 平均每年至少有11名患有脊柱裂的新生儿。此外，CHW的儿科泌尿科 一直积极参与其他泌尿外科的参与者招募和预期数据采集 包括膀胱外翻在内的各种疾病，为招生和招募设定了高标准。因此， CHW的基础设施和数量将确保在整个研究期间强有力的参与者登记工作。 作为《裁判议定书》的积极成员，我们将提出进一步研究的多种途径。首先，通过 回顾验证在方案的第一次迭代中定义的尿动力学风险阈值和 根据这些风险阈值前瞻性评估肾脏预后，我们将评估 目前的裁判员协议通过了头5年的实施。第二，我们将评估生活质量 在参与者的礼仪上，关注照顾者的负担，这些父母和婴幼儿的照顾者 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，并将 洞察这些家庭的负担，包括礼仪负担。这些将是重要的额外 评估议定书的效用和潜在的可修改内容时的结果衡量标准。最后，使用 通过以上总结的研究方法收集的信息，我们将寻找机会 协议中的改进。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)可能减去或遗漏 最大限度地减少照顾者的负担和费用，当这种研究被认为缺乏附加值的时候；和(C)扩大 给6-10岁儿童的方案，除了注意控制结果和外科手术外 上述肾脏结果。

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.