Randomized double-blind controlled trial (RCT) of pegfilgrastim as prophylactic therapy in pediatric patients with solid tumors during myelosuppressive chemotherapy.

9565 Background: The objective of this study was to evaluate the effectiveness and safety of pegfilgrastim in pediatric patients with solid tumor as prophylactic therapy during dose-intensive combination chemotherapy, in comparison to filgrastim. Methods: Thirty-two cancer patients older than 2 years-old previously untreated with chemotherapy, and with Lansky scale > 50 were randomly assigned to receive a single pegfilgrastim dose of 100 mcgr/kg (n=16) or daily (during five days) filgrastim doses of 10 mcgr/kg (n=16) after chemotherapy. Outcome measures included: duration and incidence of neutropenia (<1,000 cel./mm3) and febrile neutropenia, time to neutrophil recovery, and adverse events (AE). Statistical analysis: Chi-square and Mann-Whitney U test were used. Results: One-hundred thirty-two cycles were evaluated; 66 from pegfilgrastim group (PG) and 66 from filgrastim group (FG). Their age varied from 2- to 16 years-old. Between groups, there were no differences in age, sex and type of neoplasm. Central nervous system tumors and osteosarcoma were the most frequent tumors (n=6 and n=5 in each group, respectively). Ifosfamide-carboplatin-etoposide (ICE) and cisplatin-epirubicin (CDDP/EPI) were the most common chemotherapy used (n=7 and n=4 vs. n=6 and n=5, respectively). There were 10 episodes (15.1%) of neutropenia in FG vs. 9 (13.6%) in PG; prolonged neutropenia was seen in 2 (3%) and 1 (1.5%), respectively (p>0.05). Incidence of febrile neutropenia was more frequent in FG (6 cycles, 9% vs. 1, 1.5% p=0.057). One chemotherapy cycle in each group was deferred. Regarding of AE, pain at the application site was more frequent in FG (20 cycles, 30.3% vs. 1, 1.5%; p<0.001), but bone pain was only observed in PG (10 cycles, 15.1%, p<0.01). Frequency of hyperleukocytosis (>11,000 cel./mm3) was lower in FG (5 cycles, 7.5% vs. 20 cycles, 30.3%, p=0.001). Conclusions: In children with solid tumors the effectiveness and safety of pegfilgrastim as prophylactic therapy during chemotherapy is similar to filgrastim. These results warrant a pharmacoeconomic evaluation.