Clinical Core

项目摘要 临床核心：摘要 临床核心（CC）支持帕克-里夫斯基亚里和脊髓空洞症中心（PRCSC）的主要参与者 筛选、入组和知情同意过程以及临床数据、神经行为 评估（NIH）和患者报告的结局指标（PROM），包括健康相关质量 生命质量（HRQOL）工具。这将包括通过临床试验招募参与研究的CM患者。 在华盛顿大学/圣路易斯儿童医院/巴恩斯犹太医院的做法，但也通过 多中心Park-Reeves基亚里遗传学小组。 气候变化委员会将维护5个关键数据集，以支持所有四个拟议P01的目标和科学举措 项目：1）PRCSC多中心注册研究; 2）PRCSC的42中心PCORI支持的随机临床试验 后颅窝减压加或不加硬脑膜成形术治疗基亚里I型Chiari畸形合并脊髓空洞症 (the PFD/D试验）; 3）基亚里合并症数据库; 4）Park-Reeves基亚里遗传学数据库;和5）P01 前瞻性队列1（9-18岁）和队列2（≥19岁）。所有的数据都将被加密，匿名， 使用现有的标准操作程序上传和存储。通过这些和相关的严格 过程，CC将支持PRCSC的首要使命，即使用先进的、高质量的 遗传学、放射学和临床数据，并确定CM对大脑发育、神经系统和神经系统的影响。 结果和PROM。 协调委员会的职能是： 1.招募并维持PRCSC所有方面的研究队列。 2.协调所有项目和核心的所有临床随访、神经行为评估和PROM。 3.向所有PRCSC核心和项目提供临床数据、发育评估和PROM， 促进他们的科学倡议。 4.进行长期、纵向临床随访，进行神经行为评估和胎膜早破， hrQOL评分，以确定CM和SM的疾病自然史和治疗持久性。 5.管理所有PRCSC范围内的临床数据库的工作流程，包括表格设计，数据录入， 质量控制，以及将分析的临床数据集分发给研究者、项目和核心。 6.让CM社区参与PRCSC研究并支持其研究重点。 7.通过培养早期调查员来支持PRCSC的使命，使他们发展成为独立的， 有效的CM科学家

PROJECT SUMMARY Clinical Core: Abstract The Clinical Core (CC) supports the Park-Reeves Chiari & Syringomyelia Center (PRCSC) by leading participant screening, enrollment, and consenting processes as well as the acquisition of clinical data, neurobehavioral assessments (NIH Toolbox), and patient-reported outcome measures (PROM), including health-related quality of life (hrQOL) instruments. This will include patients with CM recruited for participation through the clinical practices at Washington University/St. Louis Children’s Hospital/Barnes-Jewish Hospital but also through the multicenter Park-Reeves Chiari Genetics Group. The CC will maintain the 5 key datasets to support the aims and scientific initiatives all four proposed P01 Projects: 1) PRCSC multicenter registry; 2) PRCSC’s 42-center PCORI-supported randomized clinical trial Posterior Fossa Decompression With or Without Duraplasty for Chiari Type I Malformation with Syringomyelia (the PFD/D Trial); 3) Chiari Comorbidites database; 4) Park-Reeves Chiari Genetics database; and 5) P01 Prospective Cohort 1 (aged 9-18 years) and Cohort 2 (aged ≥19 years). All data will be coded, anonymized, uploaded, and stored using existing Standing Operating Procedures. Through these and related rigorous processes, the CC will support the PRCSC’s overarching mission to redefine CM using leading-edge, high quality genetic, radiological, and clinical data, and to determine the effect of CM on brain development, neurological outcomes, and PROM. The functions of the CC are to: 1. Recruit and maintain the study cohorts for all aspects of PRCSC. 2. Coordinate all clinical follow-up, neurobehavioral assessments, and PROM across all Projects and Cores. 3. Provide clinical data, developmental assessments, and PROM to all PRCSC Cores and Projects to facilitate their scientific initiatives. 4. Conduct long-term, longitudinal clinical follow-up with neurobehavioral assessment, and PROM and hrQOL scoring to determine disease natural history and treatment durability in CM and SM. 5. Manage the workflow of all PRCSC-wide clinical databases, including the design of forms, data entry, quality control, and the distribution of analyzed clinical data sets to investigators, Projects, and Cores. 6. Engage the CM community to contribute to PRCSC studies and support its research priorities. 7. Support the PRCSC mission by nurturing early-stage investigators so that they develop into independent, effective CM scientists.