title: A randomized, non-inferiority study comparing efficacy and safety of a single dose of pegfilgrastim versus daily filgrastim in pediatric patients after autologous peripheral blood stem cell transplant.
abstract: OBJECTIVE
  To assess the non-inferiority of pegfilgrastim versus filgrastim in speeding the recovery of polymorphonuclear cells (PMN) in pediatric patients who underwent autologous peripheral blood stem cell transplant (PBSCT).
  
  
  METHODS
  The sample size of this randomized, multicenter, phase III study, was calculated assuming that a single dose of pegfilgrastim of 100 ug/kg was not inferior to 9 doses of filgrastim of 5 ug/kg/day. Randomization was performed by a computer-generated list and stored by sequentially numbered sealed envelopes.
  
  
  RESULTS
  Sixty-one patients, with a median age of 11.5 years, were recruited: 29 in the filgrastim arm and 32 in the pegfilgrastim arm. Twenty percent were affected by lymphoma/leukaemia and eighty percent by solid tumors. The mean time to PMN engraftment was 10.48 days (standard deviation [SD] 1.57) and 10.44 days (SD 2.44) in the filgrastim and pegfilgrastim arms, respectively. Having fixed a non-inferiority margin Delta of 3, the primary endpoint of non-inferiority was reached. No differences were observed for other secondary endpoints: platelet engraftment, mean time to platelet recovery (28 days vs. 33 days), fever of unknown origin (79% vs. 78%), proven infection (34% vs. 28%), mucositis (76% vs. 59%). After a median follow-up of 2.3 years (95% C.I.: 1.5, 3.3), 20 deaths were observed due to disease progression.
  
  
  CONCLUSIONS
  We conclude that pegfilgrastim was not inferior to daily filgrastim in pediatric patients who underwent PBSCT.  EU CLINICAL TRIAL REGISTER NUMBER: 2007-001430-14.
authors: Pediatric Hematology Oncology, Azienda Ospedaliera Universitaria Integrata, Verona, Italy. simone.cesaro@ospedaleuniverona.it
authors: Simone
authors: S
authors: Cesaro
authors: Francesca
authors: F
authors: Nesi
authors: Gloria
authors: G
authors: Tridello
authors: Massimo
authors: M
authors: Abate
authors: Irene Sara
authors: IS
authors: Panizzolo
authors: Rita
authors: R
authors: Balter
authors: Elisabetta
authors: E
authors: Calore
mesh_terms: D000293:Adolescent; D002648:Child; D002675:Child, Preschool; D000069585:Filgrastim; D016179:Granulocyte Colony-Stimulating Factor / Q000008:administration & dosage / Q000627:therapeutic use*; D006801:Humans; D007223:Infant; D008297:Male; D009369:Neoplasms / Q000601:surgery; D036102:Peripheral Blood Stem Cell Transplantation / Q000379:methods; D011092:Polyethylene Glycols; D011994:Recombinant Proteins / Q000008:administration & dosage / Q000627:therapeutic use; D016019:Survival Analysis; D019172:Transplantation Conditioning / Q000379:methods; D016896:Treatment Outcome
chemical_list: D011994:Recombinant Proteins; D016179:Granulocyte Colony-Stimulating Factor; C455861:pegfilgrastim; D011092:Polyethylene Glycols; D000069585:Filgrastim