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Pediatric Pulmonary Strategic Planning
Lung diseases are the fourth leading cause of death and disability in the United States. Historically, much focus has been on the treatment of lung diseases once symptoms are manifest and disease is well established. More recently, it has been recognized that many diseases start early in life, even in utero. Lung development is rapid in the fetus; lungs must be mature enough to support air breathing at birth, or respiratory difficulties will ensue. Evidence now suggests that in addition to genetic factors, pre- and postnatal environmental exposures exert specific long-term effects on lung structure and function which persist and adversely affect lung function and respiratory health into adulthood. The mechanisms of these early influences on lung health are not well understood. Recognizing the relationship of early life events on lung health and disease, the National Heart, Lung, and Blood Institute (NHLBI) convened a Working Group of extramural experts, at a Pediatric Pulmonary Strategic Planning Workshop on July 9-10, 2008 in order to develop a strategic plan. These experts made recommendations to NHLBI about gaps in current knowledge, and priority setting for new research directions that would capitalize on scientific opportunities.
This pediatric plan will synergize with the NHLBI Strategic Plan. The working group was charged with identifying methods to facilitate translation of basic research findings into practice to better diagnose, treat, and prevent pulmonary diseases in children. The priority areas identified for research in pediatric pulmonary diseases included: 1) epigenetic and environmental influences on lung development that program pediatric lung diseases, 2) injury, regeneration, and repair in the developing lung, 3) pulmonary vascular disease in children, 4) development and adaptation of ventilatory responses to postnatal life, 5) non-atopic wheezing: aberrant large airway development or injury? 6) strategies to improve assessment, diagnosis, and treatment of pediatric respiratory diseases, and 7) predictive and personalized medicine for children.
- Determine whether in utero and early postnatal environmental exposures or nutritional factors cause epigenetic alterations in the lung
- Use animal models to determine whether altering epigenetic influences (e.g. reverting chromatin state) with pharmacotherapy in early life can alter the incidence or progression of lung disease
- Identify the roles of genes, gene networks and the programming of the processes regulating lung injury and repair during development.
- Identify distinct populations of bone marrow-derived or intrinsic lung progenitor cells that may serve as biomarkers for disease, mediate the pathogenesis of lung disorders or provide novel therapeutic approaches to childhood respiratory illnesses.
- Elucidate mechanisms of lung angiogenesis and vasculogenesis
- Define the natural history, epidemiology and course of pediatric pulmonary vascular disease (PVD) in diverse diseases
- Determine the impact of early interventions for pulmonary artery hypertension (PAH) on long-term outcomes
- Elucidate the biology of upper airway growth and pathology aiming to characterize multiple disease entities associated with upper airway dysfunction in children.
- Develop improved physiologic measurements, imaging of airway structures and computational modeling of wheeze generation to understand the multiple mechanisms that may contribute to non-atopic airway phenotype .
- Improve reliable diagnostic tests of food aspiration with or without gastro-oesophageal reflux to assess the prevalence of these conditions in pediatric populations and their role in atypical forms of wheezing in early life.
- Develop non-invasive collection methods that can be integrated with genomic and environmental data to identify specific phenotypes within pediatric respiratory disorders
- Elucidate specific proteomic and/or cellular signatures that will provide developmental époque specific biomarkers of the pediatric respiratory disease phenotypes
- Steve H. Abman, M.D., University of Colorado School of Medicine
- Victor Chernick M.D. (Professor Emeritus), University of Manitoba
- Alan H. Jobe, M.D., Ph.D., Cincinnati Children's Hospital
- Frank J. Accurso, M.D., University of Colorado
- Robyn Barst, M.D. (Professor Emeritus), Columbia University
- Mario Castro, M.D., M.P.H., Washington University
- F. Sessions Cole, M.D., Washington University
- Gary Cutting, M.D., Johns Hopkins University
- Estelle Gauda, M.D., John Hopkins University
- James E. Gern, M.D., University of Wisconsin-Madison
- David Gozal, M.D., University of Louisville
- Gabriel G. Haddad, M.D., Rady Children's Hospital-San Diego
- James S. Hagood, M.D., University of Alabama-Birmingham
- Aaron Hamvas, M.D., Washington University
- Carolyn Kercsmar, M.D., Cincinnati Children's Hospital Medical Center
- Fernando D. Martinez, M.D., University of Arizona College of Medicine
- Nanduri Prabhakar, Ph.D., University of Chicago
- J .Usha Raj, M.D., University of Illinois at Chicago
- Maria Ramirez, Ph.D., Boston University
- Greg Redding, M.D., Children's Hospital and Regional Medical Center, Seattle
- W. Gerald Teague, M.D., Emory University of School of Medicine
- Robert Tepper M.D., Ph.D., Indiana University School of Medicine
- Jose Venegas, Ph.D., Massachusetts General Hospital
- Jeffrey Whitsett, M.D., Cincinnati Children's Hospital Medical Center
- Robert E. Wood, M.D., Ph.D., Cincinnati Children's Hospital Medical Center
- Rosalind J. Wright, M.D., Brigham & Women's Hospital
- Carol J. Blaisdell, M.D., Division of Lung Diseases
- Dorothy B. Gail, Ph.D., Division of Lung Diseases
- Gregory J. Kato, M.D. , Division of Intramural Research
- James P. Kiley, Ph.D., Division of Lung Diseases
- Gail G. Weinmann, M.D., Division of Lung Diseases