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

PurposeTo 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).MethodsThe 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.ResultsSixty-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.ConclusionsWe conclude that pegfilgrastim was not inferior to daily filgrastim in pediatric patients who underwent PBSCT.EU Clinical Trial Register Number 2007-001430-14

Recommendations for the Use of WBC Growth Factors: American Society of Clinical Oncology Clinical Practice Guideline Update

“…9 A number of small RCTs comparing pegfilgrastim and filgrastim have also been conducted in other patient populations, including pediatric patients 63,64 and adults who have undergone autologous SCT. 9 A comparison of pegfilgrastim and filgrastim was based on five clinical trials and suggested that pegfilgrastim was more effective than filgrastim at reducing the risk of febrile neutropenia (RR, 0.66; 95% CI, 0.44 to 0.98).…”

Nursing Management of Sustained Rigors and Recurrent Fever as Symptoms of Filgrastim Hypersensitivity: A Case Report

“…Filgrastim-associated sustained episodes of rigors with or without fever have not been reported in the literature. However, fever of unknown origin without rigors has been reported in a randomized clinical trial comparing safety and efficacy of single dose of pegfilgrastim versus daily filgrastim in pediatric patients undergoing autologus stem cell transplantation (Cesaro et al, 2013). Other studies of adult patients undergoing stem cell transplantation also report fever as one of the reversible adverse events associated with filgrastim administration (Orciuolo et al, 2011).…”

High-dose chemotherapy combined with autologous peripheral blood stem cell transplantation in children with advanced malignant solid tumors: A retrospective analysis of 38 cases

“…These factors all increase the risk of the recruitment, collection and reinfusion process of APBSC in children with solid tumors (14,15). The present study reports the outcomes of 38 children with advanced malignant solid tumors who received high-dose chemotherapy (HDCT) and APBSCT treatment between September 2005 and November 2011 in Beijing Tongren Hospital (Capital Medical University, Beijing, China).…”