Improving outcomes for pulmonary vascular disease

Abstract

Recognizing the importance of improving lung health through lung disease research, the National Heart, Lung, and Blood Institute (NHLBI) convened a workshop of multidisciplinary experts for the following purpose: (1) to review the current scientific knowledge underlying the basis for treatment of adults and children with pulmonary vascular diseases (PVDs); (2) to identify gaps, barriers, and emerging scientific opportunities in translational PVD research and the means to capitalize on these opportunities; (3) to prioritize new research directions that would be expected to affect the clinical course of PVDs; and (4) to make recommendations to the NHLBI on how to fill identified gaps in adult and pediatric PVD clinical research. Workshop participants reviewed experiences from previous PVD clinical trials and ongoing clinical research networks with other lung disorders, including acute respiratory distress syndrome, chronic obstructive lung disease, and idiopathic pulmonary fibrosis, as well. Bioinformatics experts discussed strategies for applying cutting-edge health information technology to clinical studies. Participants in the workshop considered approaches in the following broad concept areas: (1) improved phenotyping to identify potential subjects for appropriate PVD clinical studies; (2) identification of potential new end points for assessing key outcomes and developing better-designed PVD clinical trials; and (3) the establishment of priorities for specific clinical research needed to advance care of patients with various subsets of PVDs from childhood through adulthood. This report provides a summary of the objectives and recommendations to the NHLBI concentrating on clinical research efforts that are needed to better diagnose and treat PVDs.

Figure 1.

Pediatric pulmonary hypertensive vascular disorders often have multiple factors that contribute to the severity of pulmonary vascular disease.

Figure 2.

Pulmonary vascular disease may result from epigenetic and genetic factors, prenatal exposures, or postnatal insults that produce sustained alterations of vascular signaling pathways in children and adults. PVD indicates pulmonary vascular disease; IUGR, intrauterine growth retardation.

Figure 3.

Clinical research designed to understand the longitudinal course of pulmonary vascular disease development will identify disorders and abnormalities contributing to pulmonary vascular dysfunction. Better diagnostic criteria and phenotyping will result, ultimately leading to better therapeutic responses. Understanding the presymptomatic phase of pulmonary vascular disease might also identify interventions to prevent disease onset, especially in the pediatric population.