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Am J Respir Crit Care Med. 2017 Jun 15;195(12):1661-1670. doi: 10.1164/rccm.201701-0150WS.

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

Author information

1
1 Division of Allergy, Pulmonary, and Critical Care Medicine, Vanderbilt Medical Center, Nashville, Tennessee.
2
2 Division of Cardiology, Department of Medicine, Northwestern University, Chicago, Illinois.
3
3 Pediatric Heart and Lung Center, University of Colorado, Aurora, Colorado.
4
4 Division of Cardiology and.
5
5 Department of Quantitative Health Sciences.
6
6 Department of Cardiovascular Disease, Allegheny Health Network, Pittsburgh, Pennsylvania.
7
7 Division of Pulmonary and Critical Care Medicine and.
8
8 Division of Cardiology, Department of Medicine, University of Pittsburgh, Pittsburgh, Pennsylvania.
9
9 Division of Cardiovascular Medicine and.
10
10 Independent Consultant, Palm Coast, Florida.
11
11 Department of Medicine, University of Montreal, Montreal, Quebec, Canada.
12
12 Department of Pathobiology, and.
13
13 Department of Medicine, Cleveland Clinic, Cleveland, Ohio.
14
14 Cardiovascular Diseases, Mayo Clinic, Rochester, Minnesota.
15
15 Division of Pulmonary and Critical Care Medicine, Department of Medicine, Indiana University, Indianapolis, Indiana.
16
16 Independent Consultant and Pharmaceutical Physician, Half Moon Bay, California.
17
17 Pulmonary Hypertension Association, Silver Spring, Maryland.
18
18 Division of Medical Oncology, Department of Medicine, Yale University, New Haven, Connecticut.
19
19 Duke Clinical Research Institute, Duke University, Durham, North Carolina.
20
20 Division of Pulmonary, Critical Care, and Sleep Medicine, Tufts University, Boston, Massachusetts.
21
21 Division of Cardiology, Cornell University, New York, New York.
22
22 College of Engineering and Applied Science, University of Colorado, Denver, Colorado.
23
23 FuWai Hospital, Peking Union Medical College and Chinese Academy of Medical Sciences, Beijing, China.
24
24 Department of Anesthesiology and Critical Care and.
25
25 Division of Cardiology and.
26
26 Department of Medicine, Perelman School of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania.
27
27 Division of Cardiology, Department of Medicine, Brigham and Women's Hospital, Boston, Massachusetts.
28
28 Division of Cardiology, Massachusetts General Hospital, Boston, Massachusetts.
29
29 Division of Pulmonary and Critical Care Medicine, Johns Hopkins University, Baltimore, Maryland.
30
30 Division of Cardiology, Department of Medicine, University of Michigan, Ann Arbor, Michigan.
31
31 University of Alberta, Edmonton, Alberta, Canada.
32
32 Advanced Lung Disease Program, Inova Fairfax Hospital, Falls Church, Virginia.
33
33 Department of Human Genetics, Cincinnati Children's Hospital, Cincinnati, Ohio.
34
34 RTI International, Atlanta, Georgia.
35
35 Division of Pediatric Cardiology, Department of Pediatrics, Stanford University, Stanford, California.
36
36 Division of Cardiology, University of Arizona, Tucson, Arizona.
37
37 Department of Medicine and Laboratory Medicine, Brown University, Providence, Rhode Island.
38
38 Division of Pulmonary and Critical Care Medicine, Cedars-Sinai Medical Center, Los Angeles, California.
39
39 Division of Cardiovascular and Renal Products, Food and Drug Administration, Office of Drug Evaluation I, Office of New Drugs, Food and Drug Administration Silver Spring, Maryland; and.
40
40 Division of Lung Diseases, NHLBI, National Institutes of Health, Bethesda, Maryland.

Abstract

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.

KEYWORDS:

Pulmonary Vascular Disease Phenomics; master protocol; precision medicine; pulmonary hypertension; pulmonary vascular disease

PMID:
28430547
PMCID:
PMC5476915
DOI:
10.1164/rccm.201701-0150WS
[Indexed for MEDLINE]
Free PMC Article

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