Recombinant human granulocyte colony-stimulating factor therapy for patients with neutropenia and/or neutrophil dysfunction secondary to glycogen to disease type 1b

The purpose of this study was to evaluate the efficacy and toxicity of reco mbinant human granulocyte colony-stimulating factor (rhG-CSF) therapy in pa tients with neutropenia and/or neutrophil dysfunction secondary to glycogen storage disease (GSD) type 1b, Thirteen patients with neutropenia and/or n eutrophil dysfunction secondary to GSD type 1b were treated with rhG-CSF, T he effects of therapy on neutrophil numbers and in vitro neutrophil functio n 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 s ignificant increase in circulating neutrophil numbers (P < .01) and an impr ovement in neutrophil function as assessed in vitro. In addition, rhG-CSF t herapy produced a significant increase in marrow cellularity and an increas e in myeloid:erythroid (M:E) ratio, indicating stimulation of granulopoeisi s, No adverse effects on marrow function were noted; in particular, no myel odysplasia or marrow exhaustion was seen. Use of rhG-CSF therapy was associ ated with objective and subjective improvements in infection-related morbid ity, The therapy was well tolerated, although ail patients developed spleno megaly, and 5 patients developed mild hypersplenism that did not require an y specific treatment. rhG-CSF therapy is efficacious in the management of n eutropenia and neutrophil dysfunction associated with GSD type 1b, Patients on this therapy need to be monitored for hypersplenism. Continued follow-u p will be necessary to confirm long-term safety; however, no significant sh ort-term toxicity was noted. (C) 2001 by The American Society of Hematology .