Article
G-CSF treatment of severe congenital neutropenia reverses neutropenia but does not correct the underlying functional deficiency of the neutrophil in defending against microorganisms.
Blood - 1 Jun 2007
Donini Marta, Fontana Stefania, Savoldi Gianfranco, Vermi William, Tassone Laura, Gentili Francesca, Zenaro Elena, Ferrari Daniela, Notarangelo Lucia D, Porta Fulvio, Facchetti Fabio, Notarangelo Luigi D, Dusi Stefano, Badolato Raffaele
Abstract excerpt
The treatment of children affected by severe congenital neutropenia (SCN) with G-CSF strongly reduces the risk of sepsis by reversing neutropenia. However, SCN patients who respond to the treatment with the growth factor still have an elevated risk of succumbing to sepsis. Because the disease is usually caused by heterozygous mutations of ELA2, a gene encoding for neutrophil elastase (NE), we have investigated in...
Read the complete abstract on PubMedTopics
Share this publication in a Topic to start or enrich a Post.