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Enhancing Insights into Pulmonary Vascular Disease through a Precision Medicine Approach. A Joint NHLBI-Cardiovascular Medical Research and Education Fund Workshop Report

American journal of respiratory and critical care medicine, 2017-06, Vol.195 (12), p.1661-1670 [Peer Reviewed Journal]

Copyright American Thoracic Society Jun 15, 2017 ;Copyright © 2017 by the American Thoracic Society 2017 ;ISSN: 1073-449X ;EISSN: 1535-4970 ;DOI: 10.1164/rccm.201701-0150WS ;PMID: 28430547

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Title:
Enhancing Insights into Pulmonary Vascular Disease through a Precision Medicine Approach. A Joint NHLBI-Cardiovascular Medical Research and Education Fund Workshop Report
Author: Newman, John H ; Rich, Stuart ; Abman, Steven H ; Alexander, John H ; Barnard, John ; Beck, Gerald J ; Benza, Raymond L ; Bull, Todd M ; Chan, Stephen Y ; Chun, Hyung J ; Doogan, Declan ; Dupuis, Jocelyn ; Erzurum, Serpil C ; Frantz, Robert P ; Geraci, Mark ; Gillies, Hunter ; Gladwin, Mark ; Gray, Michael P ; Hemnes, Anna R ; Herbst, Roy S ; Hernandez, Adrian F ; Hill, Nicholas S ; Horn, Evelyn M ; Hunter, Kendall ; Jing, Zhi-Cheng ; Johns, Roger ; Kaul, Sanjay ; Kawut, Steven M ; Lahm, Tim ; Leopold, Jane A ; Lewis, Greg D ; Mathai, Stephen C ; McLaughlin, Vallerie V ; Michelakis, Evangelos D ; Nathan, Steven D ; Nichols, William ; Page, Grier ; Rabinovitch, Marlene ; Rich, Jonathan ; Rischard, Franz ; Rounds, Sharon ; Shah, Sanjiv J ; Tapson, Victor F ; Lowy, Naomi ; Stockbridge, Norman ; Weinmann, Gail ; Xiao, Lei
Subjects: Biomarkers ; Cancer therapies ; Clinical trials ; Drugs ; Education ; Heart failure ; Humans ; Hypertension, Pulmonary - therapy ; Lung diseases ; National Heart, Lung, and Blood Institute (U.S.) ; NHLBI Workshop ; Patients ; Pharmaceutical industry ; Precision medicine ; Precision Medicine - methods ; Pulmonary hypertension ; United States ; Working groups
Is Part Of: American journal of respiratory and critical care medicine, 2017-06, Vol.195 (12), p.1661-1670
Description: 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.
Publisher: United States: American Thoracic Society
Language: English
Identifier: ISSN: 1073-449X
EISSN: 1535-4970
DOI: 10.1164/rccm.201701-0150WS
PMID: 28430547
Source: GFMER Free Medical Journals
MEDLINE
Alma/SFX Local Collection
ProQuest Central

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Enhancing Insights into Pulmonary Vascular Disease through a Precision Medicine Approach. A Joint NHLBI-Cardiovascular Medical Research and Education Fund Workshop Report

Copyright American Thoracic Society Jun 15, 2017 ;Copyright © 2017 by the American Thoracic Society 2017 ;ISSN: 1073-449X ;EISSN: 1535-4970 ;DOI: 10.1164/rccm.201701-0150WS ;PMID: 28430547

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