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Blood, 15 January 2001, Vol. 97, No. 2, pp. 376-382
CLINICAL OBSERVATIONS, INTERVENTIONS, AND THERAPEUTIC TRIALS
Recombinant human granulocyte colony-stimulating factor therapy
for patients with neutropenia and/or neutrophil dysfunction secondary
to glycogen storage disease type 1b
Stanley Calderwood,
Laurie Kilpatrick,
Steven D. Douglas,
Melvin Freedman,
Kim Smith-Whitley,
Martha Rolland, and
Joanne Kurtzberg
From the City of Hope/Samaritan Bone Marrow Transplant
Program, Good Samaritan Regional Medical Center, Phoenix, AZ; the
Division of Immunologic and Infectious Diseases and the Division of
Hematology, The Children's Hospital of Philadelphia, Philadelphia, PA;
the Division of Hematology, The Hospital for Sick Children, Toronto,
Ontario, Canada; and the Division of Pediatrics, Duke University
Medical Center, Durham, NC.
The purpose of this study was to evaluate the efficacy and
toxicity of recombinant human granulocyte colony-stimulating factor (rhG-CSF) therapy in patients with neutropenia and/or neutrophil dysfunction secondary to glycogen storage disease (GSD) type 1b. Thirteen patients with neutropenia and/or neutrophil dysfunction secondary to GSD type 1b were treated with rhG-CSF. The effects of
therapy on neutrophil numbers and in vitro neutrophil function and on
bone marrow cellularity and morphology were studied. The clinical
status of the patients and the occurrence of adverse events associated
with rhG-CSF use were monitored. Use of rhG-CSF therapy was associated
with a significant increase in circulating neutrophil numbers
(P < .01) and an improvement in neutrophil function as
assessed in vitro. In addition, rhG-CSF therapy produced a significant
increase in marrow cellularity and an increase in myeloid:erythroid
(M:E) ratio, indicating stimulation of granulopoeisis. No adverse
effects on marrow function were noted; in particular, no myelodysplasia
or marrow exhaustion was seen. Use of rhG-CSF therapy was
associated with objective and subjective improvements in
infection-related morbidity. The therapy was well tolerated, although
all patients developed splenomegaly, and 5 patients developed mild hypersplenism that did not require any specific treatment. rhG-CSF
therapy is efficacious in the management of neutropenia and neutrophil
dysfunction associated with GSD type 1b. Patients on this therapy need
to be monitored for hypersplenism. Continued follow-up will be
necessary to confirm long-term safety; however, no significant
short-term toxicity was noted.
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