Stimulation of myelopoiesis in patients with aplastic anemia by recombinant human granulocyte-macrophage colony-stimulating factor

S. Vadhan-Raj, S. Buescher, Hal Broxmeyer, A. LeMaistre, J. Lepe-Zuniga, G. Ventura, S. Jeha, L. J. Horwitz, J. M. Trujillo, S. Gillis, W. N. Hittelman, J. U. Gutterman

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Abstract

Aplastic anemia is a syndrome in which pancytopenia occurs in the presence of hypocellularity of the bonemarrow. To assess the biologic activities of recombinant human granulocyte-macrophage colony-stimulating factor (GM-CSF) in aplastic anemia, we gave GM-CSF (60 to 500 μg per square meter of body-surface area) to 10 patients with moderate or severe disease, by continuous intravenous infusion daily for two weeks, and repeated the treatment after a two-week rest period. The treatment increased the white-cell count (1.6- to 10-fold) in all patients, primarily because of an increase in the numbers of neutrophils (1.5- to 20-fold), eosinophils (12- to >70-fold), and monocytes (2- to 32-fold). Rates of hydrogen peroxide production in purified granulocyte fractions increased during GM-CSF treatment. Increases in bone marrow cellularity, myeloid precursor cells, and myeloid:erythroid cell ratios accompanied the white-cell response. Despite the in vivo response of the white cells, the concentration of colony-forming cells remained the same. Measureable concentrations of interleukin-2 (2 to 15 units per milliliter) were found in the serum of 8 patients, and high levels of erythropoietin (81 to 1200 IU per liter) were found in 10 patients. The predominant side effects were constitutional symptoms. These results indicate that recombinant human GM-CSF is effective in stimulating myelopoiesis in patients with severe aplastic anemia and may benefit some patients in whom the disorder is refractory to standard forms of therapy.

Original languageEnglish (US)
Pages (from-to)1628-1634
Number of pages7
JournalNew England Journal of Medicine
Volume319
Issue number25
StatePublished - 1988

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Myelopoiesis
Aplastic Anemia
Granulocyte-Macrophage Colony-Stimulating Factor
Myeloid Cells
Erythroid Cells
Pancytopenia
Body Surface Area
Therapeutics
Erythropoietin
Granulocytes
Eosinophils
Intravenous Infusions
Human Activities
Hydrogen Peroxide
Interleukin-2
Monocytes
Neutrophils
Cell Count
Bone Marrow
Serum

ASJC Scopus subject areas

  • Medicine(all)

Cite this

Vadhan-Raj, S., Buescher, S., Broxmeyer, H., LeMaistre, A., Lepe-Zuniga, J., Ventura, G., ... Gutterman, J. U. (1988). Stimulation of myelopoiesis in patients with aplastic anemia by recombinant human granulocyte-macrophage colony-stimulating factor. New England Journal of Medicine, 319(25), 1628-1634.

Stimulation of myelopoiesis in patients with aplastic anemia by recombinant human granulocyte-macrophage colony-stimulating factor. / Vadhan-Raj, S.; Buescher, S.; Broxmeyer, Hal; LeMaistre, A.; Lepe-Zuniga, J.; Ventura, G.; Jeha, S.; Horwitz, L. J.; Trujillo, J. M.; Gillis, S.; Hittelman, W. N.; Gutterman, J. U.

In: New England Journal of Medicine, Vol. 319, No. 25, 1988, p. 1628-1634.

Research output: Contribution to journalArticle

Vadhan-Raj, S, Buescher, S, Broxmeyer, H, LeMaistre, A, Lepe-Zuniga, J, Ventura, G, Jeha, S, Horwitz, LJ, Trujillo, JM, Gillis, S, Hittelman, WN & Gutterman, JU 1988, 'Stimulation of myelopoiesis in patients with aplastic anemia by recombinant human granulocyte-macrophage colony-stimulating factor', New England Journal of Medicine, vol. 319, no. 25, pp. 1628-1634.
Vadhan-Raj, S. ; Buescher, S. ; Broxmeyer, Hal ; LeMaistre, A. ; Lepe-Zuniga, J. ; Ventura, G. ; Jeha, S. ; Horwitz, L. J. ; Trujillo, J. M. ; Gillis, S. ; Hittelman, W. N. ; Gutterman, J. U. / Stimulation of myelopoiesis in patients with aplastic anemia by recombinant human granulocyte-macrophage colony-stimulating factor. In: New England Journal of Medicine. 1988 ; Vol. 319, No. 25. pp. 1628-1634.
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AU - LeMaistre, A.

AU - Lepe-Zuniga, J.

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