Management of sickle cell disease: Summary of the 2014 evidence-based report by expert panel members

Barbara P. Yawn, George R. Buchanan, Araba N. Afenyi-Annan, Samir K. Ballas, Kathryn L. Hassell, Andra H. James, Lanetta B Jordan, Sophie M. Lanzkron, Richard Lottenberg, William J. Savage, Paula J. Tanabe, Russell E. Ware, M. Hassan Murad, Jonathan C. Goldsmith, Eduardo Ortiz, Robinson Fulwood, Ann Horton, Joylene John-Sowah

Research output: Contribution to journalArticle

516 Citations (Scopus)

Abstract

IMPORTANCE: Sickle 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. OBJECTIVE: To support and expand the number of health professionals able and willing to provide care for persons with SCD. EVIDENCE REVIEW: Databases 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. FINDINGS: Strong 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 RELEVANCE: Hydroxyurea 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.

Original languageEnglish
Pages (from-to)1033-1048
Number of pages16
JournalJAMA - Journal of the American Medical Association
Volume312
Issue number10
DOIs
StatePublished - Jan 1 2014

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Sickle Cell Anemia
Hydroxyurea
Therapeutics
Acute Chest Syndrome
Databases
Chelation Therapy
Enzyme Therapy
Sickle Hemoglobin
Inborn Genetic Diseases
Iron Overload
Light Coagulation
Spirometry
Angiotensin-Converting Enzyme Inhibitors
Pulmonary Hypertension
MEDLINE
Chronic Pain
Blood Transfusion
Penicillins
Opioid Analgesics
Observational Studies

ASJC Scopus subject areas

  • Medicine(all)

Cite this

Yawn, B. P., Buchanan, G. R., Afenyi-Annan, A. N., Ballas, S. K., Hassell, K. L., James, A. H., ... John-Sowah, J. (2014). Management of sickle cell disease: Summary of the 2014 evidence-based report by expert panel members. JAMA - Journal of the American Medical Association, 312(10), 1033-1048. https://doi.org/10.1001/jama.2014.10517

Management of sickle cell disease : Summary of the 2014 evidence-based report by expert panel members. / Yawn, Barbara P.; Buchanan, George R.; Afenyi-Annan, Araba N.; Ballas, Samir K.; Hassell, Kathryn L.; James, Andra H.; Jordan, Lanetta B; Lanzkron, Sophie M.; Lottenberg, Richard; Savage, William J.; Tanabe, Paula J.; Ware, Russell E.; Murad, M. Hassan; Goldsmith, Jonathan C.; Ortiz, Eduardo; Fulwood, Robinson; Horton, Ann; John-Sowah, Joylene.

In: JAMA - Journal of the American Medical Association, Vol. 312, No. 10, 01.01.2014, p. 1033-1048.

Research output: Contribution to journalArticle

Yawn, BP, Buchanan, GR, Afenyi-Annan, AN, Ballas, SK, Hassell, KL, James, AH, Jordan, LB, Lanzkron, SM, Lottenberg, R, Savage, WJ, Tanabe, PJ, Ware, RE, Murad, MH, Goldsmith, JC, Ortiz, E, Fulwood, R, Horton, A & John-Sowah, J 2014, 'Management of sickle cell disease: Summary of the 2014 evidence-based report by expert panel members', JAMA - Journal of the American Medical Association, vol. 312, no. 10, pp. 1033-1048. https://doi.org/10.1001/jama.2014.10517
Yawn, Barbara P. ; Buchanan, George R. ; Afenyi-Annan, Araba N. ; Ballas, Samir K. ; Hassell, Kathryn L. ; James, Andra H. ; Jordan, Lanetta B ; Lanzkron, Sophie M. ; Lottenberg, Richard ; Savage, William J. ; Tanabe, Paula J. ; Ware, Russell E. ; Murad, M. Hassan ; Goldsmith, Jonathan C. ; Ortiz, Eduardo ; Fulwood, Robinson ; Horton, Ann ; John-Sowah, Joylene. / Management of sickle cell disease : Summary of the 2014 evidence-based report by expert panel members. In: JAMA - Journal of the American Medical Association. 2014 ; Vol. 312, No. 10. pp. 1033-1048.
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author = "Yawn, {Barbara P.} and Buchanan, {George R.} and Afenyi-Annan, {Araba N.} and Ballas, {Samir K.} and Hassell, {Kathryn L.} and James, {Andra H.} and Jordan, {Lanetta B} and Lanzkron, {Sophie M.} and Richard Lottenberg and Savage, {William J.} and Tanabe, {Paula J.} and Ware, {Russell E.} and Murad, {M. Hassan} and Goldsmith, {Jonathan C.} and Eduardo Ortiz and Robinson Fulwood and Ann Horton and Joylene John-Sowah",
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AU - Yawn, Barbara P.

AU - Buchanan, George R.

AU - Afenyi-Annan, Araba N.

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AU - Hassell, Kathryn L.

AU - James, Andra H.

AU - Jordan, Lanetta B

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AU - Savage, William J.

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N2 - IMPORTANCE: Sickle 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. OBJECTIVE: To support and expand the number of health professionals able and willing to provide care for persons with SCD. EVIDENCE REVIEW: Databases 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. FINDINGS: Strong 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 RELEVANCE: Hydroxyurea 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.

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