T. Lehrnbecher, Hematopoietic growth factors in prophylaxis and therapy of infections complications in children with neutropenia, KLIN PADIAT, 213(4), 2001, pp. 212-238
Hematopoietic colony-stimulating factors have been introduced into clinical
practice as additional supportive measures to reduce infectious complicati
ons associated with congenital or acquired neutropenia. Over the past decad
e, we have begun to appreciate the subtler aspects of the proper use of G-C
SF and GM-CSF, identifying appropriate indications and contraindications. I
n the course of evaluating the corpus of studies, a set of formal recommend
ations have been propagated for the judicious use of these expensive growth
factors [3,4,152,174]. To prevent serious infection, the use of G-CSF or G
M-CSF is recommended in a subset of pediatric cancer patients shortly after
having received chemotherapy or a form of a marrow transplant. Children wi
th highly intensive chemotherapy (e.g., children with high risk ALL or NHL)
seem to benefit from hematopoietic growth factors whereas this is still un
clear for children undergoing therapy for solid tumors. An exciting develop
ment is the use of G-CSF and GM-CSF to mobilize peripheral-blood progenitor
cells for autologous or allogeneic transplantation. In pediatric patients
with hematological diseases, there are only few data on the use of hematopo
ietic growth factors in children with myelodysplastic syndrome. Experts rec
ommend the early administration of G-CSF in children with very severe aplas
tic anemia. The use of G-CSF is also recommended in children with severe ch
ronic neutropenia, but these patients have to be monitored regularly for cy
togenetic abnormalities. No larger study has shown a clinical benefit of he
matopoietic growth factor in preterm or term infants. Future studies in ped
iatric patients are clearly warranted to address several issues. Prospectiv
e clinical trials are still needed to define specific treatment groups who
can benefit from growth factor support. in this regard, efforts must be dir
ected at better defining the endpoints and in particular assigning value to
reduction in treatment of possible infectious complications, such as days
in hospital, antibiotic usage and costs. In addition, randomized studies ar
e required to evaluate the proper dosage and duration of therapy, which mos
t likely will vary between groups of patients. In addition, combinations of
different growth factors have to be tested, particularly if ex vivo expans
ion and the storage of hematopoietic stem cells are to be utilized in a wid
er spectrum of patients.