Data Fusion: A Sustainable, Scalable, Open Source Registry Advancing PVD Research

数据融合：可持续、可扩展、开源注册中心推进 PVD ​​研究

• 批准号: 9059170
• 负责人: Steven Herbert Abman
• 金额: $ 217.86万
• 依托单位: UNIVERSITY OF COLORADO DENVER
• 依托单位国家: 美国
• 财政年份: 2014
• 资助国家: 美国
• 起止时间: 2014-07-01 至 2018-04-30
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/9059170
• 关键词: Academic Medical Centers; Address; Adopted; Adult; Age; American; Award; Bioinformatics; Biometry; Bronchopulmonary Dysplasia; Cardiac; Cardiology; Child; Childhood; Clinical; Clinical Research; Clinical Sciences; Clinical and Translational Science Awards; Code; Combined Modality Therapy; Communities; Comorbidity; Data; Data Analyses; Data Collection; Data Provenance; Data Set; Data Sources; Databases; Disease; Economic Burden; Educational workshop; Electronic Health Record; Endothelin Receptor Antagonist; Enrollment; Evaluation; Family; Feeds; Funding; Goals; Health; Home environment; Individual; Informatics; Institutional Review Boards; Interview; Investigation; Knowledge; Learning; Life; Link; Lung; Measures; Medical center; Medicine; Methods; Morbidity - disease rate; National Heart, Lung, and Blood Institute; Natural History; Newborn Infant; Ontology; Outcome; Patient Outcomes Assessments; Patients; Pediatric Hospitals; Pharmacologic Substance; Pharmacotherapy; Population Research; Privacy; Pulmonary Hypertension; Pulmonology; Registries; Research; Research Design; Research Infrastructure; Rheumatology; Running; Safety; Science; Site; Societies; Surrogate Endpoint; System; Technology; Testing; Translational Research; United States National Institutes of Health; United States National Library of Medicine; Update; Vascular Diseases; Vertebral column; abstracting; base; biocomputing; cohort; comparative effectiveness; data registry; design; disease phenotype; health information technology; informatics infrastructure; inhibitor/antagonist; mortality; multidisciplinary; novel; novel therapeutics; open data; open source; operation; programs; prospective; response; scale up; social; treatment response

DESCRIPTION (provided by applicant): Pediatric pulmonary vascular diseases (PVD), including pediatric pulmonary hypertension (PH), are high morbidity and mortality conditions, yet treatment options are limited. Despite new drug therapies, survival of patients with severe PH remains poor. Although most clinical studies have emphasized disease in adults, pediatric PVD can be devastating, contributing substantially to poor outcomes in newborns, infants and children that are strikingly different from those encountered in adults. Unfortunately, very few studies have addressed the safety and efficacy of PVD therapies in children. Current approaches to PVD are limited by small numbers of patients with PH and the association of PH with a variety of pediatric disorders and subspecialties at any individual center; by few established, multidisciplinary programs in pediatric PH; and by limited interactions among existing PVD programs. Multi-center investigations are necessary for sufficiently powered interventional studies, but study designs have been restricted by lack of knowledge as to disease phenotypes, clinical course, outcomes, and age-appropriate endpoints. Our objective, therefore, is the establishment and operation of a Bioinformatics Clinical Coordinating Center (BCCC) for advancing the understanding and treatment of PVD in children, comprised of national leaders in pediatric pulmonary and cardiac medicine, informatics, and biostatistics as well as registry-based and electronic health records (EHR) clinical research. We leverage and extend the existing Pediatric Pulmonary Hypertension Network (PPHnet) for large-scale registry and EHR-based research, using a well tested and widely adopted open-source data warehouse technology infrastructure (i2b2/SHRINE), developed through the NIH National Centers for Biocomputing and Clinical and Translational Science Award (CTSA) Programs. The goal of this proposal is three-fold: (1) establish a sustainable, open source, reusable registry infrastructure across 8 PPHnet sites to capture multi-sourced EHR, "traditional" prospective patient-based data, and patient reported outcomes (PROs) across a large, prospective, pediatric pulmonary hypertension (PH) observational cohort; (2) enroll >500 registry subjects and capture data on >750 EHR subjects across 8 PPHnet sites, linking EHR with registry data at 3 sites; (3) address three classes of unanswered questions crucial for the characterization and management of PH, comparing the information value of registry vs. EHR vs. fused data across registry/EHR/PROs, in the domains of spectrum of PH comorbidities, PH indicators and endpoints of morbidity and mortality, and response to therapies in PH.

描述（由申请人提供）：小儿肺血管疾病（PVD），包括小儿肺动脉高压（PH），是高发病率和死亡率状况，但治疗方案受到限制。尽管有新的药物疗法，但严重pH患者的存活仍然很差。尽管大多数临床研究都强调了成年人的疾病，但小儿PVD可能是毁灭性的，这大大导致了与新生儿，婴儿和儿童在与成年人遇到的疾病截然不同的结果。不幸的是，很少有研究解决了儿童PVD疗法的安全性和功效。当前对PVD的方法受到少数pH患者的限制，并且在任何个体中心，pH值与各种儿科疾病和亚科疾病的关联；很少有小儿pH中建立的多学科计划；以及现有PVD计划之间的互动有限。多中心研究对于足够动力的介入研究是必要的，但是由于缺乏有关疾病表型，临床过程，结果和适合年龄的终点的知识，研究设计受到限制。因此，我们的目标是生物信息学临床协调中心（BCCC）的建立和运作，以促进儿童对PVD的理解和治疗，包括儿童肺和心脏医学，信息统计学以及生物统计学以及基于注册表的基于注册和基于注册和电子健康记录（EHR）临床研究的国家领导者。我们利用经过良好测试且广泛采用的开放源源数据仓库技术基础架构（I2B2/Shrine）来利用并扩展现有的小儿肺动脉高压网络（PPHNET）进行大规模注册表和基于EHR的研究，该研究是通过NIH国家生物计算和临时和翻译科学奖（CTSA）（CTSA）开发的。该提案的目的是三重：（1）建立一个可持续的，开源的，可重复使用的注册表基础架构，跨8个PPHNET站点捕获多种源自的EHR，“传统的”前瞻性患者数据，并在大型，前瞻性的，儿科的肺血压（pH）观察量（PROS）中报告的患者报告（PROS）； （2）注册> 500名注册主题，并在8个PPHNET站点上捕获> 750个EHR受试者的数据，将EHR与3个站点的注册表数据联系起来； （3）解决三类未解决的问题对于pH的表征和管理至关重要，将注册表与EHR与跨注册表/EHR/PROS的信息值进行比较，在pH合并症，pH指标和终点的pH合并症和死亡率和死亡率的范围内，以及对PH疗法的响应。