Treatment of Refractory Aplastic Anemia With Recombinant Human Granulocyte-Macrophage-Colony-Stimulating Factor

Treatment of Refractory Aplastic Anemia With Recombinant Human Granulocyte-Macrophage-Colony-Stimulating Factor

Fifteen patients with refractory aplastic anemia or agranulocytosis received treatment with recombinant human granulocyte-macrophage-colony-stimulating factor (rhGM-CSF) in doses from 4 to 64 μg/kg/d by continuous intravenous (IV) infusion. Ten of 11 evaluable patients with aplastic anemia had subst...

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Bibliographic Details
Published in:Blood Vol. 73; no. 3; pp. 694 - 699
Main Authors: Champlin, R.E., Nimer, S.D., Ireland, P., Oette, D.H., Golde, D.W.
Format: Journal Article
Language:English
Published: Washington, DC Elsevier Inc 15-02-1989
The Americain Society of Hematology
Subjects:
Anemia, Refractory - drug therapy
Anemia, Refractory - pathology
Biological and medical sciences
Blood. Blood coagulation. Reticuloendothelial system
Bone Marrow - pathology
Colony-Stimulating Factors - therapeutic use
Dose-Response Relationship, Drug
Granulocyte-Macrophage Colony-Stimulating Factor
Growth Substances - therapeutic use
Hematopoiesis
Humans
Leukocyte Count
Medical sciences
Pharmacology. Drug treatments
Recombinant Proteins
Time Factors
Human
Chemotherapy
Treatment
Intravenous administration
Agranulocytosis
Aplastic anemia
Granulocyte
Hemopathy
Colony stimulating factor
Macrophage
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Summary:Fifteen patients with refractory aplastic anemia or agranulocytosis received treatment with recombinant human granulocyte-macrophage-colony-stimulating factor (rhGM-CSF) in doses from 4 to 64 μg/kg/d by continuous intravenous (IV) infusion. Ten of 11 evaluable patients with aplastic anemia had substantial increments in granulocytes, monocytes, and eosinophils associated with myeloid and eosinophilic hyperplasia in the bone marrow. Patients with pretreatment granulocytes >0.3 × 109/L had greater increments in circulating myeloid cells than patients with more severe granulocytopenia. Only one patient had improvement in erythrocytes and platelets. Blood counts fell to baseline after rhGM-CSF treatment was discontinued. Doses up to 16 μg/kg/d were relatively well tolerated in the absence of extreme leukocytosis. Fatigue and myalgia were common. Three patients developed pulmonary infiltrates that resolved with discontinuation of treatment. Patients tended to have recurrent inflammation in previously diseased tissues. These data indicate that rhGM-CSF will increase circulating granulocytes, monocytes, and eosinophils in patients with refractory aplastic anemia. Further studies are necessary to determine if rhGM-CSF treatment will reduce morbidity or improve survival.©1989 by Grune & Stratton, Inc.
ISSN:0006-4971
1528-0020
DOI:10.1182/blood.V73.3.694.694