Comp B-National Spina Bifida Patient Registry

描述（由申请人提供）：本项目的目的是：1）继续收集洛杉矶儿童医院（CHLA）脊柱裂患者的数据，用于国家脊柱裂患者登记处（NSBPR），以更好地了解人群的需求，提高患者和家庭的护理质量，并确定未来研究的领域，以及2）在特定的感兴趣领域制定和实施研究计划，以促进对登记数据的分析。脊柱裂（SB）发生率为每10，000例活产1.9例， 美国目前有7万人患有这种疾病。SB是由妊娠28天神经管闭合不全和脊膜突出引起的，包括脊髓脊膜膨出、脂肪脊膜膨出和脊膜膨出。在脊髓脊膜膨出中，典型的损伤包括脑积水、基亚里II畸形、导致失禁的神经源性肠和/或膀胱、下肢肌无力和感觉缺失以及认知功能障碍。这些损伤会对日常生活产生重大影响，并会干扰学校、工作和其他社群活动。鉴于SB可能存在的无数症状，这种情况可能对生活质量产生重大影响。通过研究这一人群的健康状况，特别是那些去专门的脊柱裂诊所的儿童和青少年，可以确定干预措施和策略，以改善获得全面的，循证的，具有成本效益的护理，并最终提高生活质量的个人与条件。该项目允许识别产生以下成果的创新： 最佳成果，提供了一种分享最佳做法的手段，并作为多地点研究的平台。登记网络收集的数据为研究人员和临床医生提供了一个强大的数据库，以调查人口的健康状况。这项研究工作产生的结果已经并将继续为临床干预提供证据，以改善脊柱裂患者的护理和生活质量。治疗和结果可以通过诊所，人口和地理区域进行比较。本项目的目标包括所有合格患者的100%入组，以便入组登记研究、收集和录入数据、参加所有要求的会议、与其他研究中心合作、识别研究和质量改进机会以及传播获得的新知识。

DESCRIPTION (provided by applicant): The aims of this project are: 1) to continue to collect data about patients with Spina bifida at Children's Hospital Los Angeles (CHLA) for the National Spina Bifida Patient Registry (NSBPR), to better understand the needs of the population, improve the quality of care for patients and families, and identify areas for future research, and 2) to develop and implement a research plan in a specific area of interest to contribute to the analysis of Registry data. Spina bifida (SB) occurs in 1.9 per 10,000 live births with an estimated 70,000 people in the United States currently living with the condition. SB results from incomplete closure of the neural tube and protrusion of the spinal membrane at 28 days gestation, and includes myelomeningocele, lipomeningocele and meningocele. In myelomeningocele, typical impairments include hydrocephalus, Chiari II malformation, neurogenic bowel and/or bladder resulting in incontinence, muscle weakness and lack of sensation in lower extremities, and cognitive dysfunction. These impairments can have significant impact on day-to-day living and can interfere with school, work and other community activities. Given the myriad symptoms that can be present in SB, the condition can have a significant impact on quality of life. By studying the health of this population, specifically those children and youth who attend specialized Spina bifida clinics, interventions and strategies can be identified to improve access to comprehensive, evidence based, cost effective care and ultimately, to improve the quality of life of individuals living with the condition. This project allows for the identification of innovations that yield the best outcomes, provides a means to share best practices and serves as a platform for multisite research. Data collected by the Registry network provides researchers and clinicians with a robust database to investigate the health status of the population. The findings generated by this research effort have and will continue to generate evidence for clinical interventions to improve the care and quality of life for individuals with Spina bifida. Treatments and outcomes can be compared by clinic, population and geographic region. Objectives of this project include enrollment of 100% of all eligible patients will be approached for enrollment into the Registry, collection and entry of data, participation in all required meetings, collaboration with other site, identification of research and quality improvement opportunities, and dissemination of new knowledge gained.