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Management of sickle cell disease: summary of the 2014 evidence-based report by expert panel members.
IMPORTANCESickle cell disease (SCD) is a life-threatening genetic disorder affecting nearly 100,000 individuals in the United States and is associated with many acute and chronic complications requiring immediate medical attention. Two disease-modifying therapies, hydroxyurea and long-term blood transfusions, are available but underused.
OBJECTIVETo support and expand the number of health professionals able and willing to provide care for persons with SCD.
EVIDENCE REVIEWDatabases of MEDLINE (including in-process and other nonindexed citations), EMBASE, Cochrane Database of Systematic Reviews, Cochrane Central Register of Controlled Trials, CINAHL, TOXLINE, and Scopus were searched using prespecified search terms and keywords to identify randomized clinical trials, nonrandomized intervention studies, and observational studies. Literature searches of English-language publications from 1980 with updates through April 1, 2014, addressed key questions developed by the expert panel members and methodologists.
FINDINGSStrong recommendations for preventive services include daily oral prophylactic penicillin up to the age of 5 years, annual transcranial Doppler examinations from the ages of 2 to 16 years in those with sickle cell anemia, and long-term transfusion therapy to prevent stroke in those children with abnormal transcranial Doppler velocity (≥200 cm/s). Strong recommendations addressing acute complications include rapid initiation of opioids for treatment of severe pain associated with a vasoocclusive crisis, and use of incentive spirometry in patients hospitalized for a vasoocclusive crisis. Strong recommendations for chronic complications include use of analgesics and physical therapy for treatment of avascular necrosis, and use of angiotensin-converting enzyme inhibitor therapy for microalbuminuria in adults with SCD. Strong recommendations for children and adults with proliferative sickle cell retinopathy include referral to expert specialists for consideration of laser photocoagulation and for echocardiography to evaluate signs of pulmonary hypertension. Hydroxyurea therapy is strongly recommended for adults with 3 or more severe vasoocclusive crises during any 12-month period, with SCD pain or chronic anemia interfering with daily activities, or with severe or recurrent episodes of acute chest syndrome. A recommendation of moderate strength suggests offering treatment with hydroxyurea without regard to the presence of symptoms for infants, children, and adolescents. In persons with sickle cell anemia, preoperative transfusion therapy to increase hemoglobin levels to 10 g/dL is strongly recommended with a moderate strength recommendation to maintain sickle hemoglobin levels of less than 30% prior to the next transfusion during long-term transfusion therapy. A strong recommendation to assess iron overload is accompanied by a moderate strength recommendation to begin iron chelation therapy when indicated.
CONCLUSIONS AND RELEVANCEHydroxyurea and transfusion therapy are strongly recommended for many individuals with SCD. Many other recommendations are based on quality of evidence that is less than high due to the paucity of clinical trials regarding screening, management, and monitoring for individuals with SCD.
Olmsted Medical Center, Rochester, Minnesota.,
University of Texas Southwestern Medical Center, Dallas.,
University of North Carolina, Chapel Hill.,
Thomas Jefferson University, Cardeza Foundation, Philadelphia, Pennsylvania.,
University of Colorado, Denver.,
University of Virginia, Charlottesville.,
Foundation for Sickle Cell Disease Research, University of Miami, Miller School of Medicine, Miami, Florida.,
Johns Hopkins School of Medicine, Baltimore, Maryland.,
University of Florida, Gainesville.,
Brigham and Women's Hospital and Harvard Medical School, Boston, Massachusetts.,
Duke University, Schools of Nursing and Medicine, Durham, North Carolina.,
Cincinnati Children's Hospital Medical Center, Cincinatti, Ohio.,
Mayo Clinic College of Medicine, Rochester, Minnesota.,
National Heart, Lung, and Blood Institute, Bethesda, Maryland16Dr Goldsmith is now with the Rare Diseases Program, Office of New Drugs, US Food and Drug Administration, Bethesda, Maryland.,
National Heart, Lung, and Blood Institute, Bethesda, Maryland17Dr Ortiz is now a private senior consultant in Bethesda, Maryland.,
National Heart, Lung, and Blood Institute, Bethesda, Maryland18Dr Fulwood is now retired.,
American Institutes for Research, Silver Spring, Maryland.
National Heart, Lung, and Blood Institute, Bethesda, Maryland.
Anemia, Sickle Cell
Consensus Development Conferences as Topic
Physical Therapy Modalities
Practice Guidelines as Topic
Pub Type(s)Journal Article
Research Support, N.I.H., Extramural