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Maintenance treatment of patients with myelodysplastic syndromes using
recombinant human granulocyte colony-stimulating factor
RS Negrin, DH Haeuber, A Nagler, Y Kobayashi, J Sklar, T Donlon, M Vincent and PL Greenberg
Department of Medicine, Stanford University Medical Center, CA 94305- 5112.
Myelodysplastic syndromes (MDS) are characterized by chronic refractory
cytopenias resulting in increased risk of infection, bleeding, and
conversion to acute leukemia. In an effort to improve these cytopenias we
have treated 18 patients over a 6- to 8-week period with increasing daily
subcutaneous doses of recombinant human granulocyte colony- stimulating
factor (G-CSF). Sixteen patients responded with improvement in neutrophil
counts. On cessation of treatment these counts returned to baseline values
over a 2- to 4-week period. To maintain these improved blood counts 11
patients were treated with G-CSF for more prolonged periods. Ten patients
again responded with an increase in total leukocyte counts (1.6- to
6.4-fold) and absolute neutrophil counts (ANC) (3.6- to 16.3-fold), with
responses persisting for 3 to 16 months. A significantly decreased risk of
developing bacterial infections was noted during periods with ANC greater
than 1,500/mm3 as compared with periods of time with ANC less than
1,500/mm3. Two anemic patients had a greater than 20% rise in hematocrit
over the study period, and 2 additional patients had a decrease in red
blood cell transfusion requirements during G-CSF treatment. Bone marrow
myeloid maturation improved in 7 of 9 maintenance phase patients. Three
patients progressed to acute myeloid leukemia during treatment. The drug
was generally well-tolerated and no severe toxicities were noted. These
data demonstrated that G-CSF administered to MDS patients by daily
subcutaneous administration was well-tolerated and effective in causing
persistent improvement of the neutrophil levels and marrow myeloid
maturation. These effects were associated with a decreased risk of
infection and, in some patients, with decreased red blood cell transfusion
requirements.
Volume 76,
Issue 1,
pp. 36-43,
07/01/1990
Copyright © 1990 by The American Society of Hematology
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