Efficacy and Safety of Pegylated Filgrastim as Primary Prophylaxis Following Pediatric Dose-intensive Chemotherapy: A Randomized Controlled Trial.

Abstract

Objectives: Large trials and meta-analyses in adults suggest a similar efficacy and safety of a single dose of peg-filgrastim compared with daily filgrastim in the prevention of chemotherapy-induced neutropenia. However, there is no large prospective data in the pediatric population. This trial was designed to demonstrate the efficacy and safety of peg-filgrastim in children.

Methods: This was an open-labelled randomized trial. Children with solid tumors receiving chemotherapy in which growth factor support was indicated were screened before chemotherapy and randomized to either the filgrastim arm (5 mcg/kg daily) or peg-filgrastim arm (100 mcg/kg single dose 24 h postchemotherapy). Patients were followed up till absolute neutrophil count (ANC) recovery clinically as well as by thrice weekly complete blood counts. The primary outcome was the incidence of FN, and secondary outcomes included the reduction in the depth of ANC nadir, shortening of the duration of grade-4 neutropenia, reduction in ANC recovery time, and adverse events.

Results: A total of 344 chemotherapy cycles were screened. After the exclusion of 29 cycles, 315 chemotherapy cycles in 61 patients were randomized to peg-filgrastim (n=160) and filgrastim arm (n=155). The median age of included patients was 3 years (2; 6.5), and males were predominant. The incidence of FN in peg-filgrastim and filgrastim arms was 8% and 18% (P=0.01) (RR: 0.45; 95% CI: 0.24-0.84). Duration of ANC recovery, depth of ANC nadir, and adverse events reported were not significantly different in both arms.

Conclusions: The incidence of FN in the peg-filgrastim arm was significantly lesser. No significant adverse events attributable to the study drug were reported.