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Trials in sickle cell disease

Fenella J Kirkham1, Norma B Lerner, Michael Noetzel

  • 1Institute of Child Health (University of London), London, England. F.Kirkham@ich.ucl.ac.uk

Pediatric Neurology
|June 13, 2006
PubMed

Insights

Children with sickle cell disease face neurological risks like stroke. Transcranial Doppler screening and transfusions can prevent strokes in high-risk children, with ongoing trials exploring further preventative strategies.

Area of Science:

  • Neurology
  • Pediatrics
  • Hematology

Background:

  • Children with sickle cell disease are susceptible to severe neurological complications, including stroke, cognitive decline, and seizures.
  • Early detection and intervention are crucial for preventing long-term neurological damage.

Purpose of the Study:

  • To evaluate the effectiveness of transcranial Doppler (TCD) screening in identifying children with sickle cell disease at high risk for stroke.
  • To assess the efficacy of chronic blood transfusions in preventing overt clinical strokes in these children.
  • To explore new preventative strategies, including TCD velocity monitoring, aspirin, and respiratory support.

Main Methods:

  • The Stroke Prevention Trial in Sickle Cell Anemia (STOP) utilized TCD to identify high-risk children.
  • Children with critically high TCD velocities (>200 cm/sec) received chronic transfusions.
  • Ongoing trials (STOP II, Silent Infarct Transfusion) investigate discontinuing transfusions, the impact of transfusions on silent cerebral infarcts, and pilot studies for aspirin and respiratory support.

Main Results:

  • The STOP trial demonstrated a significant reduction in overt strokes among high-risk children receiving chronic transfusions.
  • Ongoing research aims to determine the safety of discontinuing transfusions and the benefits of transfusions for silent infarcts.

Conclusions:

  • Transcranial Doppler screening and chronic transfusions are effective in preventing strokes in children with sickle cell disease.
  • Further research is ongoing to optimize preventative strategies and improve long-term neurological outcomes in this population.

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