Hydrocephalus Clinical Research Network: a consortium for pediatric hydrocephalus

描述(由申请人提供)：本申请涉及广泛的挑战领域05比较有效性研究和特定挑战主题05-NS-101临床神经科学比较有效性研究联盟建设。长期目标是将当前的研究范式从单中心回顾性病例系列报告转变为使用现代电子数据捕获系统进行前瞻性多中心方案驱动的重点研究，并建立网络基础设施作为开展儿童脑积水比较有效性研究的持久资源。方法将是：a.建立一个由成员机构和研究人员组成的协作小组，并配备专门的研究支持人员；b.在经验丰富的数据协调中心的支持下；c.实施核心数据项目，该项目将详细描述患者群体及其管理的特征；d.在儿科脑积水的特定领域进行重点研究。早期进展感兴趣的各方于2006年最后一个夏天开始开会，并与犹他州数据协调中心建立了关系，制定了脑积水临床研究网络(HCRN)委员会结构和治理文件，并制定了初步研究方案。这些是：1.核心数据项目，旨在描述患有脑积水的儿童及其管理的详细情况。在头10个月里，它已经积累了605名患者。2.一项减少分流感染的方案，在头17个月中已进行了1162例手术。初步分析表明，遵守该方案可降低感染率。3.评估出血性脑积水治疗的方案。已经在整个网络中进行了一项回溯性的初步研究，并计划制定一项前瞻性方案，以比较两种治疗出血性脑积水的外科技术。4.评估超声引导下分流置入的方案已经制定，并在所有中心获得了IRB的批准，并刚刚开始对患者进行评估。该网络的两年目标是完成这些项目，并根据他们的发现，开发R01应用程序，以便在2011年进行预期试验。儿科脑积水是一种非常常见的问题，2003年有近40,000人入院，40,000多天住院，住院费用为2亿美元。除了极少数例外，临床研究一直是在单一中心进行的，具有回溯性数据，这不允许对治疗效果得出强有力的结论。这项提议是建立一个协调网络，以确定脑积水研究中最迫切需要的领域，并进行前瞻性研究，以解决这些问题。在合作的头18个月中，该网络制定了一个核心数据项目和三项重点研究，这些研究将产生规划未来预期试验所需的试点数据。

DESCRIPTION (provided by applicant): This application addresses broad challenge area 05 Comparative Effectiveness Research and specific challenge topic 05-NS-101 Consortia Building for Comparative Effectiveness Research in Clinical Neuroscience. The long-term objective is to shift the current research paradigm from single center retrospective case series reports to prospective multicenter protocol driven focused studies using modern electronic data capture systems and to establish a network infrastructure as a lasting resource for the conduct of comparative effectiveness research in pediatric hydrocephalus. The approach will be to: a. Establish a collaboration group of member institutions and investigators with dedicated research support personnel, b. supported by an experienced data coordinating center, c. conduct a core data project, which will characterize the patient population and their management in detail, d. conduct focused studies in specific areas of pediatric hydrocephalus. Early Progress Interested parties began meeting in the last summer of 2006 and developed a relationship with the Utah Data Coordinating Center, developed the Hydrocephalus Clinical Research Network (HCRN) committee structure and governance documents and developed the initial study protocols. These are: 1. The Core Data Project, which is designed to characterize children with hydrocephalus and their management in detail. It has accrued 605 patients in the first 10 months. 2. A protocol to reduce shunt infections, which has accrued 1,162 procedures in the first 17 months. Preliminary analysis suggests decreased infection rates with adherence to the protocol. 3. A protocol to evaluate the management of post-hemorrhagic hydrocephalus. A retrospective pilot study has been conducted across the Network and a prospective protocol is planned to compare two surgical techniques for the management of post- hemorrhagic hydrocephalus. 4. A protocol to evaluate ultrasound guided shunt placement has been written, obtained IRB approval at all centers and has just started patient accrual. The two year goal for the Network is to complete these projects and based on their findings, developed R01 applications for prospective trials in 2011. Pediatric hydrocephalus is a very common problem resulting in almost 40,000 hospital admissions, more than 400,000 hospital days and $2,000,000,000 in hospital charges in 2003. With very few exceptions, clinical research has been conducted at a single center with retrospective data, which do not allow strong conclusions about treatment efficacy. This proposal is to establish a coordinated network to identify the areas of most urgent need in hydrocephalus research and conduct prospective studies to solve these problems. In the first 18 months of collaboration, the Network has developed a Core Data Project and three focused studies, which will generate the pilot data needed to plan prospective trials in the future.