Enhancing Insights into Pulmonary Vascular Disease through a Precision Medicine Approach A Joint NHLBI-Cardiovascular Medical Research and Education Fund Workshop Report
Enhancing Insights into Pulmonary Vascular Disease through a Precision Medicine Approach A Joint NHLBI-Cardiovascular Medical Research and Education Fund Workshop Report
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DOI：
10.1164/rccm.201701-0150ws
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发表时间：
2017-06-15
影响因子：
24.7
通讯作者：
Xiao, Lei
中科院分区：
文献类型：
--
作者：
Newman, John H.;Rich, Stuart;Xiao, Lei
NHLBI肺部疾病部门和心血管医学教育和研究基金举办了一次研讨会，讨论如何利用最近启动的“通过肺血管疾病表型组学重新定义肺动脉高压”（PVDOMICS）计划的预期科学成果，开发治疗肺血管疾病的新方法。PVDOMICS是一个合作的、方案驱动的网络，用于分析所有肺动脉高压患者人群，以确定新型肺血管疾病（PVD）表型。利益相关者，包括基础、翻译和临床研究者;临床医生;患者倡导组织;监管机构;和制药行业专家，共同讨论了精准医学在PVD临床试验中的应用。根据NHLBI战略愿景目标，提出了讨论研究优先事项的建议，包括：（1）涉及所有利益相关者的国家努力，应寻求将所有资助项目的生物样本和生物数据协调到基于网络的存储库，以便信息可以与其他研究项目共享和关联。NHLBI赞助的项目包括PVDOMICS、肺动脉高压突破计划、PAH国家生物样本和数据库以及国家精准医学计划。(2)一个工作组负责制定PVD的主临床试验方案，将精准医学原则应用于未来的临床试验。具体功能包括：（a）采用适应性和创新性统计设计，采用纳入生物标志物指导富集策略的小型临床试验;（B）开发反映明确定义和临床意义变化的较新终点。(3)在成像、血流动力学、细胞、基因组和代谢测试中开发更新的系统变量，这将有助于精确识别PVD的个体和共同特征，并作为治疗干预的新表型的基础。
The Division of Lung Diseases of the NHLBI and the Cardiovascular Medical Education and Research Fund held a workshop to discuss how to leverage the anticipated scientific output from the recently launched "Redefining Pulmonary Hypertension through Pulmonary Vascular Disease Phenomics" (PVDOMICS) program to develop newer approaches to pulmonary vascular disease. PVDOMICS is a collaborative, protocol-driven network to analyze all patient populations with pulmonary hypertension to define novel pulmonary vascular disease (PVD) phenotypes. Stakeholders, including basic, translational, and clinical investigators; clinicians; patient advocacy organizations; regulatory agencies; and pharmaceutical industry experts, joined to discuss the application of precision medicine to PVD clinical trials. Recommendations were generated for discussion of research priorities in line with NHLBI Strategic Vision Goals that include: (1) A national effort, involving all the stakeholders, should seek to coordinate biosamples and biodata from all funded programs to a web-based repository so that information can be shared and correlated with other research projects. Example programs sponsored by NHLBI include PVDOMICS, Pulmonary Hypertension Breakthrough Initiative, the National Biological Sample and Data Repository for PAH, and the National Precision Medicine Initiative. (2) A task force to develop a master clinical trials protocol for PVD to apply precision medicine principles to future clinical trials. Specific features include: (a) adoption of smaller clinical trials that incorporate biomarker-guided enrichment strategies, using adaptive and innovative statistical designs; and (b) development of newer endpoints that reflect well-defined and clinically meaningful changes. (3) Development of updated and systematic variables in imaging, hemodynamic, cellular, genomic, and metabolic tests that will help precisely identify individual and shared features of PVD and serve as the basis of novel phenotypes for therapeutic interventions.