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Sickle hemoglobinopathies

P A Lane1

  • 1Colorado Sickle Cell Treatment and Research Center, University of Colorado Health Sciences Center, Denver 80262.

Current Opinion in Pediatrics
|February 1, 1993
PubMed
Summary
This summary is machine-generated.

Neonatal screening and comprehensive care significantly reduce sickle hemoglobinopathy mortality. However, new research offers hope for better management of complications and potential curative therapies.

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Area of Science:

  • Hematology
  • Pediatric Medicine
  • Genetics

Background:

  • Neonatal screening and comprehensive care, including penicillin prophylaxis and immunizations, have greatly decreased infant mortality from sickle hemoglobinopathies.
  • Despite advances in understanding pathophysiology, current treatments inadequately prevent acute noninfectious complications (e.g., pain crises, strokes) and chronic organ damage.

Purpose of the Study:

  • To review recent advancements in understanding sickle hemoglobinopathies.
  • To highlight improvements in managing acute complications and the emergence of potentially curative therapies.

Main Methods:

  • Review of recent publications on sickle hemoglobinopathies.
  • Analysis of current therapeutic approaches and ongoing clinical trials.

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Main Results:

  • Increased knowledge of factors contributing to clinical heterogeneity in sickle hemoglobinopathies.
  • Modest improvements in identifying and treating acute complications.
  • Emerging hope for curative treatments, with ongoing trials for hydroxyurea and bone marrow transplantation.

Conclusions:

  • While neonatal screening and supportive care have improved survival, significant challenges remain in managing disease complications.
  • Recent research offers promising avenues for more effective treatments and potential cures for sickle hemoglobinopathies.