Over the last decade, the most significant advance in hematology has been the isolation, cloning, and large-scale production of hematopoietic growth factors. In vivo, these factors stimulate bone marrow progenitor cells, resulting in increased production of mature peripheral blood cells. Most of the phase III trials of these factors have involved adult patients, although a number of pediatric phase l/ll trials have been reported. To date, these trials have shown that the toxicity and efficacy of these agents in children and adults are similar. Therefore, despite the paucity of pediatric data and lack of US Food and Drug Administration approval for their use in children, these factors are being used extensively in pediatrics, especially at tertiary care centers. In this article, the clinical trials of erythropoietin, granulocyte colony-stimulating factor, granulocyte-macrophage colony-stimulating factor, and interleukin-3 are reviewed.
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