What is the outlook for children with congenital neutropenia?
While bone marrow transplant is the only cure for congenital neutropenia, giving G-CSFs can raise your child’s neutrophil count making him or her less prone to infections and therefore feeling in better health generally. Unfortunately there appears to be a risk that children with severe congenital neutropenia develop leukaemia or myelodysplasia (effectively a preleukaemia). It seems that the more severely affected children are at a greater risk. This was recognised before the use of G-CSF therapy, but is being seen more often now. The most likely explanation for this is that before G-CSF therapy, many children died of infections before living long enough to develop leukaemia, rather than the G-CSF itself being to blame. In other conditions with neutropenia where G-CSF has been used (that is, cyclical, autoimmune and idiopathic), this increase in leukaemia has not been described. It seems that this problem only affects children with congenital neutropenia. Children therefore have annual
