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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

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  • Simone Cesaro
  • Francesca Nesi
  • Gloria Tridello
  • Massimo Abate
  • Irene Sara Panizzolo
  • Rita Balter
  • Elisabetta Calore

Abstract

Purpose: 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

Suggested Citation

  • Simone Cesaro & Francesca Nesi & Gloria Tridello & Massimo Abate & Irene Sara Panizzolo & Rita Balter & Elisabetta Calore, 2013. "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," PLOS ONE, Public Library of Science, vol. 8(1), pages 1-8, January.
    • Handle: RePEc:plo:pone00:0053252
    DOI: 10.1371/journal.pone.0053252
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    References listed on IDEAS

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    1. Catherine Sebban & Anne Lefranc & Lionel Perrier & Philippe Moreau & Daniel Espinouse & A. Schmidt & L. Kammoun & Hervé Ghesquières & C. Ferlay & Jacques-Olivier Bay & Séverine Lissandre & David Pérol, 2012. "A randomised phase II study of the efficacy, safety and cost-effectiveness of pegfilgrastim and filgrastim after autologous stem cell transplant for lymphoma and myeloma (PALM study)," Post-Print halshs-00680996, HAL.
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