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National Institutes of Health Consensus Development Conference Statement: Hydroxyurea Treatment for Sickle Cell Disease
- Otis W. Brawley, MD;
- Llewellyn J. Cornelius, PhD, LCSW;
- Linda R. Edwards, MD;
- Vanessa Northington Gamble, MD, PhD;
- Bettye L. Green, RN;
- Charles Inturrisi, PhD;
- Andra H. James, MD, MPH;
- Danielle Laraque, MD;
- Magda Mendez, MD;
- Carolyn J. Montoya, RN, MSN, CPNP;
- Brad H. Pollock, MPH, PhD;
- Lawrence Robinson, MD, MPH;
- Aaron P. Scholnik, MD; and
- Melissa Schori, MD, MBA*
- From Emory University and the American Cancer Society, Atlanta, Georgia; University of Maryland School of Social Work, Baltimore, Maryland; College of Medicine, University of Florida, Jacksonville, Jacksonville, Florida; The George Washington University, Washington, DC; Saint Joseph Regional Medical Center and African-American Women in Touch, South Bend, Indiana; Weill Medical College of Cornell University and Mount Sinai School of Medicine, New York, and Lincoln Medical and Mental Health Center, Bronx, New York; Women's Hemostasis and Thrombosis Clinic and Duke University Medical Center, Durham, North Carolina; National Association of Pediatric Nurse Practitioners and College of Nursing, University of New Mexico, Albuquerque, New Mexico; School of Medicine, University of Texas Health Science Center at San Antonio, San Antonio, Texas; Philadelphia Department of Public Health, Philadelphia, Pennsylvania; Upper Peninsula Hematology/Oncology Associates and Cancer Research Office, Marquette General Health System, Marquette, Michigan; and Princeton Healthcare System, Princeton, New Jersey.
National Institutes of Health consensus and state-of-the- science statements are prepared by independent panels of health professionals and public representatives on the basis of 1) the results of a systematic literature review prepared under contract with the Agency for Healthcare Research and Quality (AHRQ), 2) presentations by investigators working in areas relevant to the conference questions during a 2-day public session, 3) questions and statements from conference attendees during open discussion periods that are part of the public session, and 4) closed deliberations by the panel during the remainder of the second day and morning of the third. This statement is an independent report of the panel and is not a policy statement of the National Institutes of Health or the U.S. government. The statement reflects the panel's assessment of medical knowledge available at the time the statement was written. Thus, it provides a “snapshot in time” of the state of knowledge on the conference topic. When reading the statement, keep in mind that new knowledge is inevitably accumulating through medical research.
Sickle cell disease is an inherited blood disorder that affects 50 000 to 100 000 people in the United States. It is estimated that 2000 babies are born with sickle cell disease in the United States each year. Sickle cell disease was the first disease for which a specific molecular defect in a gene was identified, and it is the most common genetic disease identified as part of the Newborn Screening Program in the United States. The condition is chronic and lifelong, and it is associated with a decreased lifespan. Sickle cell disease is most common in people whose families come from Africa, South or Central America, Caribbean islands, Mediterranean countries (such as Turkey, Greece, and Italy), India, and Saudi Arabia.
Sickle cell disease occurs when an infant inherits the …
