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Sickle cell disease in children

Emily Riehm Meier1, Jeffery L Miller

  • 1Molecular Medicine Branch, National Institute of Diabetes and Digestive and Kidney Diseases, National Institutes of Health, Bethesda, MD 20892, USA.

Drugs
|April 24, 2012
PubMed

Insights

Newborn screening for sickle cell disease (SCD) and early interventions like penicillin and hydroxyurea have improved survival and reduced complications in children. Genetic therapies offer future prevention and cures for SCD.

Area of Science:

  • Pediatrics
  • Hematology
  • Genetics

Background:

  • Newborn screening for sickle cell disease (SCD) is now universal in the US.
  • Early identification improves survival by preventing sepsis with prophylactic penicillin.
  • Key complications in pediatric SCD include stroke, painful crises, and acute chest syndrome.

Purpose of the Study:

  • To review clinical manifestations and therapeutic strategies for pediatric SCD.
  • To discuss the evolving role of gene-focused prevention and therapy for SCD.

Main Methods:

  • Review of existing literature on pediatric sickle cell disease management.
  • Analysis of current and emerging therapeutic strategies, including genetic research.

Main Results:

  • Universal newborn screening and early penicillin prophylaxis significantly reduce sepsis-related mortality.
  • Transcranial Doppler screening and chronic transfusions decrease stroke risk from 10% to 1%.
  • Hydroxyurea therapy reduces painful crises, acute chest syndromes, and transfusion needs.

Conclusions:

  • Current management strategies have markedly improved outcomes for children with SCD.
  • Ongoing genetic research holds promise for the ultimate prevention and cure of SCD.
  • Focus is shifting towards long-term prevention and curative gene-based therapies for SCD.

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