Thrombocytopenia Clinical Trial
OBJECTIVES: I. Assess the efficacy of recombinant human granulocyte colony-stimulating
factor (G-CSF) in raising the absolute neutrophil count, platelet count, and hemoglobin
level in patients with inherited bone marrow failure syndromes.
II. Assess the efficacy of a reduced maintenance dose in patients who respond to daily
G-CSF.
III. Assess the toxic effects of G-CSF in these patients. IV. Measure bone marrow progenitor
colonies before and after G-CSF. V. Measure CD34-positive cells in marrow and blood before
and after G-CSF using flow cytometry and immunohistochemistry.
PROTOCOL OUTLINE: Patients receive granulocyte colony-stimulating factor (G-CSF)
subcutaneously every day for 8 weeks; nonresponders receive an increased dose for an
additional 8 weeks. Patients who respond at week 8 or 16 are then tapered to a lower
maintenance dose of G-CSF administered every other day through week 40. The dose is adjusted
to maintain an absolute neutrophil count above 1500.
Patients are removed from study for failure to achieve a complete response by week 16,
unacceptable nonhematologic toxicity, the identification of a clonal karyotype in marrow, or
the onset of leukemia.
;
Primary Purpose: Treatment
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