title: Is pegfilgrastim safe and effective in congenital neutropenia? An analysis of the French Severe Chronic Neutropenia registry.
abstract: OBJECTIVE
  To examine the efficacy and safety of pegfilgrastim in patients with congenital neutropenia (CN).
  
  
  METHODS
  Seventeen patients enrolled in the French Severe CN Register received pegfilgrastim.
  
  
  RESULTS
  Median age at pegfilgrastim introduction was 19.1 years (range 3.9-52.3 years). In 14 cases pegfilgrastim replaced GCSF (filgrastim or lenograstim), after a median of 6.9 years of GCSF therapy. The dose of pegfilgrastim was usually one full vial per injection (except in five children, who received 1/6 to 1/2 a vial), resulting in a dose of between 50 and 286 microg/kg. The pegfilgrastim schedule ranged from two injections every 7 days to one injection every 30 days, with treatment-free periods. The median interval between the first and last dose of pegfilgrastim was 0.8 years (0.01-4.1 years). The absolute neutrophil count tended to increase more strongly on pegfilgrastim than on GCSF, but the difference was not statistically significant. During pegfilgrastim therapy, a severe infection occurred in two patients and recurrent ENT infections in two other patients. Bone pain was reported by nine patients, anemia and thrombocytopenia occurred in one patient (WHO grade III), chronic urticaria occurred in one patient (WHO grade III), and a single pegfilgrastim injection was followed by respiratory distress and death 15 days later in a patient with GDSIb. At the last update, 10 patients had stopped receiving pegfilgrastim and seven patients were still receiving pegfilgrastim.
  
  
  CONCLUSIONS
  Compared to conventional GCSF, pegfilgrastim is more difficult to use in congenital neutropenia, with more frequent adverse events and sometimes poor efficacy.
authors.affiliation: Service d'Hémato-Oncologie Pédiatrique, Registre Français des Neutropénies Chroniques Sévères, Centre de Référence des Déficits Immunitaires Héréditaires, Hôpital Trousseau, Paris, France.
authors.forename: Blandine
authors.initials: B
authors.lastname: Beaupain
authors.forename: Thierry
authors.initials: T
authors.lastname: Leblanc
authors.forename: Oumédaly
authors.initials: O
authors.lastname: Reman
authors.forename: Olivier
authors.initials: O
authors.lastname: Hermine
authors.forename: Jean-Pierre
authors.initials: JP
authors.lastname: Vannier
authors.forename: Felipe
authors.initials: F
authors.lastname: Suarez
authors.forename: Patrick
authors.initials: P
authors.lastname: Lutz
authors.forename: Pierre
authors.initials: P
authors.lastname: Bordigoni
authors.forename: Anne
authors.initials: A
authors.lastname: Jourdain
authors.forename: Michele
authors.initials: M
authors.lastname: Schoenvald
authors.forename: Marie
authors.initials: M
authors.lastname: Ouachee
authors.forename: Sylvie
authors.initials: S
authors.lastname: François
authors.forename: Fréderic
authors.initials: F
authors.lastname: Kohser
authors.forename: Fabrice
authors.initials: F
authors.lastname: Jardin
authors.forename: Gilles
authors.initials: G
authors.lastname: Devouassoux
authors.forename: Yves
authors.initials: Y
authors.lastname: Bertrand
authors.forename: Raphaele
authors.initials: R
authors.lastname: Nove-Josserand
authors.forename: Jean
authors.initials: J
authors.lastname: Donadieu
mesh_terms: D000293:Adolescent; D000328:Adult; D002452:Cell Count; D002648:Child; D002675:Child, Preschool; D004341:Drug Evaluation; D064420:Drug-Related Side Effects and Adverse Reactions; D000069585:Filgrastim; D016179:Granulocyte Colony-Stimulating Factor / Q000009:adverse effects* / Q000627:therapeutic use; D006801:Humans; D008875:Middle Aged; D009503:Neutropenia / Q000150:complications / Q000151:congenital / Q000188:drug therapy* / Q000453:epidemiology; D009504:Neutrophils; D010146:Pain; D011092:Polyethylene Glycols; D011994:Recombinant Proteins; D012042:Registries; D016896:Treatment Outcome; D055815:Young Adult
chemical_list: D011994:Recombinant Proteins; D016179:Granulocyte Colony-Stimulating Factor; C455861:pegfilgrastim; D011092:Polyethylene Glycols; D000069585:Filgrastim