Phase II Pilot Study of Granulocyte Colony-Stimulating Factor for Inherited Bone Marrow Failure Syndromes
Status:
Completed
Trial end date:
1969-12-31
Target enrollment:
Participant gender:
Summary
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.