Pegfilgrastim in children with severe congenital neutropenia.
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.
Fioredda
Francesca
F
Haematology Unit, Giannina Gaslini Childrens' Hospital, Genoa, Italy. francescafioredda@ospedale-gaslini.ge.it
Calvillo
Michaela
M
Lanciotti
Marina
M
Lanza
Tiziana
T
Giunti
Laura
L
Castagnola
Elio
E
Lorenzi
Ines
I
Tonelli
Rossella
R
Ghezzi
Piero
P
Dufour
Carlo
C
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
D011994:Recombinant Proteins; D016179:Granulocyte Colony-Stimulating Factor; C455861:pegfilgrastim; D011092:Polyethylene Glycols; D000069585:Filgrastim