title: Pegfilgrastim in children with severe congenital neutropenia.
abstract: Two pediatric patients affected by severe congenital neutropenia (SCN) were treated with 100 mcg/L/dose every 9-12 days within a pilot study (Observatory of the Italian Ministry of Health, Eudract Code 2005-003096-20) on the use of pegfilgrastim in patients with chronic neutropenia. Both children increased their absolute neutrophil count, reduced their infectious load, and improved their quality of life. Serum concentrations of G-CSF observed in pegfilgrastim mirrored those seen in filgrastim. These data suggest that pegfilgrastim may be beneficial in SCN patients with an exposure of hematopoietic cells to G-CSF similar to that on filgrastim.
authors.affiliation: Haematology Unit, Giannina Gaslini Childrens' Hospital, Genoa, Italy. francescafioredda@ospedale-gaslini.ge.it
authors.forename: Francesca
authors.initials: F
authors.lastname: Fioredda
authors.forename: Michaela
authors.initials: M
authors.lastname: Calvillo
authors.forename: Marina
authors.initials: M
authors.lastname: Lanciotti
authors.forename: Tiziana
authors.initials: T
authors.lastname: Lanza
authors.forename: Laura
authors.initials: L
authors.lastname: Giunti
authors.forename: Elio
authors.initials: E
authors.lastname: Castagnola
authors.forename: Ines
authors.initials: I
authors.lastname: Lorenzi
authors.forename: Rossella
authors.initials: R
authors.lastname: Tonelli
authors.forename: Piero
authors.initials: P
authors.lastname: Ghezzi
authors.forename: Carlo
authors.initials: C
authors.lastname: Dufour
mesh_terms: D002648:Child; D004334:Drug Administration Schedule; D005260:Female; D000069585:Filgrastim; D016179:Granulocyte Colony-Stimulating Factor / Q000097:blood / Q000627:therapeutic use*; D006801:Humans; D007958:Leukocyte Count; D008297:Male; D009503:Neutropenia / Q000097:blood / Q000151:congenital / Q000188:drug therapy*; D009504:Neutrophils / Q000187:drug effects / Q000473:pathology; D010865:Pilot Projects; D011092:Polyethylene Glycols; D011994:Recombinant Proteins; D016896:Treatment Outcome
chemical_list: D011994:Recombinant Proteins; D016179:Granulocyte Colony-Stimulating Factor; C455861:pegfilgrastim; D011092:Polyethylene Glycols; D000069585:Filgrastim