Enhancing Insights into Pulmonary Vascular Disease (PVD) Through a Precision Medicine Approach. A Joint NHLBI-CMREF Workshop Report.
Am J Respir Crit Care Med. 2017 Apr 21;:
Authors: Newman JH, Rich S, Abman SH, Alexander JH, Barnard J, Beck GJ, Benza RL, Bull TM, Chan SY, Chun HJ, Doogan D, Dupuis J, Erzurum SC, Frantz RP, Geraci M, Gillies H, Gladwin M, Gray MP, Hemnes AR, Herbst RS, Hernandez AF, Hill NS, Horn EM, Hunter K, Jing ZC, Johns R, Kaul S, Kawut SM, Lahm T, Leopold JA, Lewis GD, Mathai SC, McLaughlin VV, Michelakis ED, Nathan SD, Nichols W, Page G, Rabinovitch M, Rich J, Rischard F, Rounds S, Shah SJ, Tapson VF, Lowy N, Stockbridge N, Weinmann G, Xiao L
The Division of Lung Diseases of the National Heart, Lung and Blood Institute, (NHLBI) and the Cardiovascular Medical Education and Research Fund (CMREF), held a workshop to discuss to leverage of the anticipated scientific output from the recently launched "Redefining Pulmonary Hypertension (PH) through Pulmonary Vascular Disease Phenomics" (PVDOMICS) program. PVDOMICS is a protocol driven network to analyze PH patient populations to define novel pulmonary vascular disease (PVD) phenotypes. Basic, translational and clinical investigators, patient advocacy organizations, regulatory agencies, and pharmaceutical industry experts discussed the application of precision medicine to PVD clinical trials. Discussion of priorities in line with NHLBI Strategic Vision Goals included: 1. A national effort to coordinate bio-samples and bio-data from all funded programs to a web based repository so that information can be shared and correlated with other research projects. Example NHLBI programs include PVDOMICS, Pulmonary Hypertension Breakthrough Initiative (PHBI), the National Biological Sample and Data Repository for PAH, and the National Precision Medicine Initiative. 2. A taskforce 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 strategies, using adaptive and innovative statistical designs. 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.
PMID: 28430547 [PubMed - as supplied by publisher]