Severe chronic neutropenia (SCN) is a rare but important cause of recurrent
fevers, oropharyngeal ulcerations and severe
infections. In three forms of SCN, i.e.,
congenital neutropenia (
Kostmann's syndrome and related syndromes), idiopathic
neutropenia (both childhood and adult), and
cyclic neutropenia, it is now established that long-term treatment with the hematopoietic
growth factor, recombinant human
granulocyte colony stimulating factor (rHuG-CSF or
Filgrastim), can elevate blood neutrophil counts to the normal range in most patients, with a concomitant reduction in
infection-related events including
fever, oral ulcerations,
antibiotic use and symptoms of
inflammation. Treatment with this
growth factor causes an increase in the number and maturity of marrow cells of the neutrophilic series; other cell lines are largely unaffected. Marrow stimulation and expansion are reflected by the occurrence of bone
pain early in
therapy, as well as some increase in spleen size in most cases. Adverse effects of
therapy are infrequent in both children and adults, and long-term treatment with daily or every-other-day s.c.
injections of rHuG-CSF are well accepted. Because of the risk that some patients with chronic
neutropenia may have or develop myelodysplasia and/or
leukemia, careful pretreatment evaluations (blood, bone marrow and cytogenetics) and long-term observations are extremely important. An international registry for patients with SCN has been established to maintain records and further investigate these conditions.