Treatment and prevention of stroke in children with sickle cell disease

Shyamal H Mehta1, Robert J Adams

  • 1Department of Neurology, Medical College of Georgia, 1120 15th Street, Augusta, GA 30912, USA. rjadams@mcg.edu.

Insights

Regular blood transfusions significantly reduce stroke risk in children with sickle cell disease (SCD). Transcranial Doppler (TCD) screening identifies high-risk children, making transfusions a proven stroke prevention strategy.

Area of Science:

  • Neurology
  • Hematology
  • Pediatrics

Background:

  • Stroke is a significant complication in children with sickle cell disease (SCD), ranging from silent infarcts to severe, disabling lesions.
  • Intracranial hemorrhages in SCD can result from aneurysms, moyamoya disease, or venous issues.
  • While acute stroke treatments are limited, evidence supports stroke prevention strategies in this population.

Purpose of the Study:

  • To review the evidence for stroke prevention in children with sickle cell disease.
  • To highlight the effectiveness of transcranial Doppler (TCD) screening and blood transfusions in primary stroke prevention.
  • To discuss current and potential secondary stroke prevention methods.

Main Methods:

  • The Stroke Prevention Trial in Sickle Cell Anemia (STOP) randomized high-risk children (TCD velocity ≥ 200 cm/s) to transfusion or no transfusion.
  • STOP2 evaluated the impact of discontinuing transfusions on stroke risk.
  • Review of existing literature on stroke prevention in SCD.

Main Results:

  • The STOP trial demonstrated a 90% reduction in first-time stroke among children receiving regular blood transfusions.
  • Discontinuing transfusions in STOP2 led to a high rate of abnormal TCD values and recurrent strokes.
  • TCD screening and chronic transfusion (maintaining HbS < 30%) is the only established primary stroke prevention strategy for SCD.

Conclusions:

  • Transcranial Doppler (TCD) screening and maintaining low hemoglobin S levels through chronic transfusion is the sole proven method for primary stroke prevention in children with SCD.
  • Further research is needed for secondary stroke prevention, with hydroxyurea under investigation.
  • Bone marrow transplantation shows potential for stroke prevention but requires more data.

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