NEWS & EVENTS
NHLBI Workshop On Sarcoidosis - “Leveraging Scientific Advancements to Understand Sarcoidosis Variability and Improve Outcomes”
August 31 to September 1, 2015
The National Heart, Lung and Blood Institute
Description

Sarcoidosis is an enigmatic disease characterized pathologically by granulomatous inflammation in the lung, heart, brain, eyes, skin, liver and other organs.  It remains unclear  why patients’ disease manifestations, severity and long-term prognoses vary so widely among the individuals affected.  It also remains unclear why sarcoidosis’ prevalence and mortality are significantly higher among African-Americans and women. As such, sarcoidosis has become a disease of health disparities. The National Heart, Lung and Blood Institute (NHLBI) held a two-day workshop with the stakeholders of this disease to discuss how to leverage recent scientific advances to better understand sarcoidosis pathogenesis and to identify and help patients at-risk for severe sarcoidosis.  The participants of the workshop included many experts outside of cardiology and pulmonary medicine since a more coordinated and collaborative approach by all stakeholders is needed to accelerate research in this disease and improve sarcoidosis patients’ outcomes.

Recap

Recommendations

Establish a cohort of rigorously phenotyped sarcoidosis patients with longitudinal follow-up data and serial biospecimens collected using standardized methodologies.This resource can be used

Standardize definitions of disease phenotypes, organ-specific measures of dysfunction, and templates for collection of data/quality of life measures to facilitate clinical management and future clinical studies

Establish standard of care for different phenotypes of sarcoidosis patients based on the best available evidence and then fill in the knowledge gaps with future clinical studies/trials. Devise new outreach strategies to communities with high prevalance and/or high burden of sarcoidosis patients and increase early referrals of at-risk patients to Centers of Excellence for advanced management and clinical trials.

Develop innovative approaches including both prevention and therapeutic interventions to reduce the burden and severity of disease among those most greatly affected.

Prioritize clinical trials that evaluate interventions for life-threatening complications of sarcoidosis and consider new trial strategies such as “randomized withdrawal design” for trials addressing rarer sarcoidosis complications such as neurosarcoidosis. Biospecimens from these sarcoidosis subjects in these trials should also be collected for future biomarkers/pathogenesis studies.

Apply “omics” and systems biology research to improve the molecular characterization of sarcoidosis disease phenotypes.

Develop animal models that recapitulate features and mechanisms found in chronic sarcoidosis.

Integrate and use healthcare electronic medical records systems to 1) assess sarcoidosis disease burden in the U.S. longitudinally; 2) determine the disease trajectories among patients with different disease phenotypes; 3) analyze the impact of pain, fatigue, depression, and cognitive dysfunction and other disabilities on patients and 4) determine the contribution of treatment complications, aging, and comorbid conditions to long-term morbidity and mortality in sarcoidosis.

Workshop Co-Chairs

  • Lisa A. Maier, M.D., University of Colorado
  • William J. Martin, II M.D., The Ohio State University

Workshop Participants

  • Bob Baughman, M.D., University of Cincinnati Medical Center
  • Edward S. Chen, M.D., Johns Hopkins School of Medicine
  • Ronald G. Collman, M.D., University of Pennsylvania School of Medicine
  • Yvette C. Cozier, Sc.D., Boston University School of Public Health
  • Elliott D. Crouser, M.D., The Ohio State University
  • Daniel Culver, D.O., Cleveland Clinics
  • Wonder Puryear Drake, M.D.,  Vanderbilt University Medical Center
  • Tasha Fingerlin, Ph.D., National Jewish Health
  • Andrew P. Fontenot, M.D., University of Colorado
  • Alicia K. Gerke, M.D., University of Iowa
  • Nabeel Hamzeh, M.D., National Jewish Health
  • Michael C. Iannuzzi, M.D., SUNY Downstate Medical Center
  • Marc Judson, M.D., Albany Medical College
  • Naftali Kaminski, M.D., Yale School of Medicine
  • Laura Koth, M.D., UCSF School of Medicine
  • David R. Moller, M.D., Johns Hopkins School of Medicine
  • Courtney Gray Montgomery, Ph.D., Oklahoma Medical Research Foundation
  • Patrick Nana-Sinkam, M.D., The Ohio State University
  • Elizabeth Ofili, M.D., Morehouse School of Medicine
  • Carlos Pardo-Villamizar, M.D., Johns Hopkins School of Medicine
  • Paula Yvette Polite, City of Memphis
  • Ganesh Raghu, M.D. Professor of Medicine
  • Walter Royal III, M.D., University of Maryland Medical Center
  • Benjamin A. Rybicki, Ph.D., Henry Ford Hospital
  • William Sauer, M.D., University of Colorado
  • Leslie Serchuck, M.D., Foundation for Sarcoidosis Research
  • Ginger Spitzer, Foundation for Sarcoidosis Research
  • Barney J. Stern, M.D., University of Maryland School of Medicine
  • Nadera Sweiss, M.D., University of Illinois College of Medicine at Chicago
  • Gloria Elaine B. Westney, M.D.,  Morehouse School of Medicine
  • Ivana Yang, Ph.D., University of Colorado

NHLBI/NIH Staff

  • Lisa Begg, Dr.P.H., R.N., Office of Research on Women’s Health
  • Lis Caler, Ph.D. Division of Lung Diseases, NHLBI
  • Sandra Colombini-Hatch, M.D., Ph.D., Division of Lung Diseases, NHLBI
  • Michael Engelgaus, M.D., CTRIS, NHLBI
  • Jerry Eu, M.D., Division of Lung Diseases, NHLBI
  • Joseph Fontana, M.D., Intramural Program,
  • Katrina Gwinn, M.D., NINDS
  • Nancy MacGarvey, M.D., Division of Lung Diseases, NHLBI
  • Weiniu Gan, Ph.D., Division of Lung Diseases, NHLBI
  • Ivan Navarro, Ph.D., Division of Lung Diseases, NHLBI
  • James P. Kiley, Ph.D., Division of Lung Diseases, NHLBI
  • Walter Koroshetz, M.D., NINDS
  • George Mensah, M.D, CTRIS, NHLBI
  • Laura Moen, Ph.D., NAIMS
  • Timothy M. Moore, M.D., Ph.D., Division of Lung Diseases, NHLBI
  • Lisa Postow, Ph.D., Division of Lung Diseases, NHLBI
  • Lora Reineck, M.D., Division of Lung Diseases, NHLBI
  • Xenia Tigno, Ph.D., Division of Lung Diseases, NHLBI
  • Lisa Viviano, R.N., Division of Lung Diseases, NHLBI
  • Gail Weinmann, M.D., Division of Lung Diseases, NHLBI
  • Thomas A. Wynn, Ph.D., NIAID

Related Materials

Compendium of Best Practices for Data Coordinating Centers