Severe neutropenia (absolute neutrophil count < 500 eells/mm(3)) is as
sociated with life-threatening infections. Therapeutic regimens used t
o ameliorate severe neutropenia have been of limited benefit. The newl
y discovered cytokines acting primarily on granulocytic differentiatio
n are an important new therapeutic modality in inherited and acquired
neutropenic disorders. Very encouraging results in early phase I-II tr
ials in neutropenic patients led to the implementation of a phase III
randomized multi-center trial to investigate the efficacy and safety o
f G-CSF in these disorders. Of 120 patients with severe neutropenia en
tered on the phase III study, 108 experienced a complete response, 4 a
partial response and 8 failed to respond. Response was associated wit
h improvement in quality of life with reductions in infections and ant
ibiotic usage. Reasons for non-response are currently be investigated.
The long-term effects of cytokine administration to neutropenic patie
nts is unknown. Osteopenia and secondary myelodysplasia or leukemia ar
e beginning to be reported. For this reason only patients with severe
neutropenia or those exhibiting serious infection should receive cytok
ine therapy at this time.