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Prolonged administration of granulocyte colony-stimulating factor
(filgrastim) to patients with Fanconi anemia: a pilot study
WR Rackoff, A Orazi, CA Robinson, RJ Cooper, BP Alter, MH Freedman, RE Harris and DA Williams
James Whitcomb Riley Hospital for Children, Department of Pathology, Herman
B Wells Center for Pediatric Research, Indiana University School of
Medicine, Indianapolis.
This report examines the effect of filgrastim (granulocyte colony-
stimulating factor, [G-CSF] in 12 patients with neutropenia [absolute
neutrophil count [ANC] < 1,000/mm3]) caused by Fanconi anemia (FA). Two
of 14 patients who were evaluated for study entry were ineligible because
of unsuspected cytogenetic abnormalities in their bone marrow (BM). G-CSF
was started at 5 micrograms/kg/d. All patients had an increase in their ANC
at week 8 (mean increase = 15,664/mm3). The median ANC during therapy was
5,030/mm3. Eight of 10 patients who completed 40 weeks on study maintained
an ANC > 1,500/mm3 on G-CSF given every-otherday. Four patients had an
increase in their platelet count by week 8 without transfusion (maximum
increase = 23,000 to 45,000/mm3); however, platelet counts fell toward
baseline levels as the G-CSF dose was reduced. BM CFU-MK were increased at
week 8 in three of four evaluable patients. Four patients who did not
receive red blood cell transfusions had an increase in their hemoglobin
level of at least 2.0 g/dL. A fifth patient had a red blood cell
transfusion in week 2 and then had a similar increase in hemoglobin level
without subsequent transfusion. Eight of 10 patients who completed 40 weeks
of treatment showed increases in the percentage of BM CD34+ cells measured
by flow cytometry. The same proportion showed increases in peripheral blood
CD34+ cells. Increased BM cellularity and myeloid hyperplasia were constant
findings and were associated with increased expression of the proliferating
cell nuclear antigen. Adverse experiences were mild fever (1 patient) and a
new BM cytogenetic abnormality at week 40 (1 patient). This study shows
that prolonged administration of G-CSF exerts a stimulatory effect on the
BM of FA patients and may be used to maintain a clinically adequate ANC in
these patients. G-CSF has beneficial effects on multiple hematopoietic
lineages in some patients and may be a good candidate for use in
combination cytokine protocols for FA patients with progressive aplastic
anemia. G-CSF use results in an increase in circulating CD34+ cells, a
finding with important implications for future gene transfer protocols.
Volume 88,
Issue 5,
pp. 1588-1593,
09/01/1996
Copyright © 1996 by The American Society of Hematology
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