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Sickle Cell Disease in Children

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Abstract

Early identification of infants with sickle cell disease (SCD) by newborn screening, now universal in all 50 states in the US, has improved survival, mainly by preventing overwhelming sepsis with the early use of prophylactic penicillin. Routine transcranial Doppler screening with the institution of chronic transfusion decreases the risk of stroke from 10% to 1% in paediatric SCD patients. Hydroxyurea decreases the number and frequency of painful crises, acute chest syndromes and number of blood transfusions in children with SCD. Genetic research continues to be driven toward the prevention and ultimate cure of SCD before adulthood. This review focuses on clinical manifestations and therapeutic strategies for paediatric SCD as well as the evolving topic of gene-focused prevention and therapy.

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Article 15 March 2018

Gregory J. Kato, Frédéric B. Piel, … Elliott P. Vichinsky

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Acknowledgements

The Intramural Research Program of the National Institute of Diabetes and Digestive and Kidney Diseases supported this work. The authors thank Y. Terry Lee and Colleen Byrnes for their assistance in the preparation of this manuscript.

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  1. Molecular Medicine Branch, National Institute of Diabetes and Digestive and Kidney Diseases, National Institutes of Health, 10 Center Drive, Building 10, Room 9N311, Bethesda, Maryland, MD 20892, USA

    Emily Riehm Meier &  Jeffery L. Miller

  2. Center for Cancer and Blood Disorders, Children’s National Medical Center, Washington, D.C., USA

    Emily Riehm Meier

  3. Department of Pediatrics, The George Washington University Medical Center, Washington, DC, USA

    Emily Riehm Meier

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  1. Emily Riehm Meier
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Meier, E.R., Miller, J.L. Sickle Cell Disease in Children. Drugs 72, 895–906 (2012). https://doi.org/10.2165/11632890-000000000-00000

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