Sickle Cell Disease Workshop: Clinical Priorities and Clinical Trials

DoubleTree Hotel & Executive Meeting Center
8120 Wisconsin Avenue
Bethesda, Maryland 20814

October 22-24, 2008

A sickle cell patient receiving treatment.

In March, 2008, NHLBI announced a recommitment to research in sickle cell disease. With advice from stakeholders in the lay and professional communities, the NHLBI Advisory Council reviewed the current research portfolio and scientific opportunities, and recommended an aggressive program to bring new technologies and approaches to improve the lives of persons with sickle cell disease. NHLBI is committed to expanding the scope of inquiry in this disease, to include investigators in relevant disciplines that have not previously been involved in sickle cell disease research. This expansion in scientific reach will need to be matched by a parallel effort to extend clinical research opportunities to a much wider spectrum of subjects and investigators than previously.

With limited resources, it will be vital to set priorities for the next generation of clinical trials. Workshop attendees will address two interrelated questions:

  • What are the most important phase II and phase III clinical studies that need to be conducted in areas that have been identified as high priorities for sickle cell disease?
  • What is the best organization structure for these clinical trials that will ensure the maximum participation by subjects and investigators?

The clinical research priorities discussions will be initiated by eight sub-committees, each of which will be dedicated to a high-priority area of research, as identified by the NHLBI Advisory Council. These Sub-committees will be composed of investigators from outside and inside the sickle cell community. Recommendations developed by the Sub-committees during meetings prior to the Workshop will be presented.

The clinical trials discussion will draw on the expertise of senior investigators in oncology, HIV disease and asthma.   These senior investigators will present their experience with regard to recruitment of clinical sites, prioritization of clinical protocols, and their track records for study completion and publication.

Breakout sessions and question-and-answer periods will allow attendees to present their views.

Attendance will be limited to 200 individuals.

Workshop Co-chairs

Kwaku Ohene-Frempong, MD, Professor of Pediatrics, University of Pennsylvania School of Medicine

Barbara Alving, M.D., Director, National Center for Research Resources

Confirmed Speakers

  • Alan Schechter, MD, Chief, Molecular Biology and Genetics Section, NIDDK, NIH
  • Barbara Alving, M.D., Director, National Center for Research Resources
  • Clinton Joiner, M.D., Ph.D., Cincinnati Children's Hospital Medical Center
  • David Dilts, PhD, MBA, Professor of Operations Research, Owen Graduate School of Management, Co-Director, Center for Management Research in Healthcare, Vanderbilt University
  • Gail Weinmann, M.D. NHLBI Division of Lung Diseases
  • George Dover, MD, Professor and Chair, Department of Pediatrics, Johns Hopkins Medical Center
  • James Neaton, PhD, Professor of Biostatistics, University of Minnesota, Principal Investigator, International Network for Strategic Initiatives in Global HIV Trials (INSIGHT).(NIAID)
  • Johnson Haynes, MD, Professor of Medicine, Division of Pulmonary and Critical Care Medicine, University of Southern Alabama Medical Center
  • Jon Scheinmann, MD, Professor of Pediatrics, Chief, Pediatric Nephrology, University of Kansas Medical Center
  • Judith Manola, M.S., Dana-Farber Cancer Institute
  • Kathryn Hassell, M.D., Colorado Sickle Cell Center and NHLBI Division of Blood Diseases and Resources
  • Mark Gladwin, MD, Professor of Medicine, Chief, Division of Pulmonary, Allergy, and Critical Care Medicine, University of Pittsburgh
  • Mark Walters, MD, Director, Blood and Bone Marrow Transplantation Program, Children's Hospital Oakland Research Institute
  • Robert Adams, MD, Professor of Neuroscience, Director, Stroke Center, Medical University of South Carolina
  • Roy L. Silverstein, MD, Professor of Molecular Medicine, Chair, Department of Cell Biology, The Cleveland Clinic Foundation
  • Russell Ware, MD, PhD, Chair, Department of Hematology, St. Jude Children's Research Hospital
  • Scott Miller, MD, Professor of Clinical Pediatrics, SUNY-Downstate Medical Center
  • Wally Smith, MD, Professor of Medicine, Chair, Division of Quality Health Care, Virginia Commonwealth University
  • Willarda Edwards, MD, MBA, President, Sickle Cell Disease Association of America

Organizing Committee

  • Susan Shurin, MD, Deputy Director, NHLBI, Acting Division Director, Division of Blood Diseases and Resources.
  • Harvey Luksenburg, MD, Medical Officer, Division of Blood Diseases and Resources, NHLBI
For more information, please contact:


Harvey Luksenburg, MD


Coordinator: Ms. Wanda Ware

Sponsored by:

Department of Health and Human Services
National Institutes of Health
National Heart, Lung, and Blood Institute
Division of Blood Diseases and Resources
Blood Diseases Program
6701 Rockledge Drive, MSC 7950
Bethesda, MD 20892-7950
Phone: 301-435-0050     Fax: 301-480-1046