Cost-Effectiveness of Early Empirical Antibiotic Cessation in Pediatric High-Risk Febrile Neutropenia

Early discontinuation of empirical antibiotic therapy (EAT) is non-inferior to continuation of EAT until absolute neutrophil count (ANC) ≥500 cells/mm³ for children with high-risk febrile neutropenia (FN). In this prospective trial-based economic evaluation, we aimed to estimate the healthcare resource utilization and cost-effectiveness of early discontinuation of EAT versus continuation of EAT in children with high-risk FN in India.

Clinically stable children aged 2-18 years with acute leukemia, high-risk FN, and afebrile status for ≥72 h were randomized to stop EAT or continue EAT until ANC recovery. Healthcare utilization data, including antibiotic exposure, antifungal use, hospitalization, laboratory investigations, and organ support, were prospectively collected. Individual patient data were used to estimate the direct medical costs, quality-adjusted life years (QALYs), and the incremental monetary benefit (iNMB).

A total of 76 patients were included in the stop arm and 73 in the continuation arm. Early discontinuation reduced total antibiotic duration and antifungal use. The mean total cost per patient was ₹67,894 in the stop arm versus ₹77,291 in the continuation arm (difference: ₹9397). Antibiotic and antifungal expenditures were lower with early discontinuation of EAT. Mean QALYs were 0.0255 in the stop arm and 0.0236 in the continuation arm (difference: 0.0018). The iNMB favored early discontinuation (₹9785.6; 95% credible interval ₹6949.1-₹12,534.2). In probabilistic sensitivity analysis, early discontinuation was cost-effective in 100% of simulations.

Early discontinuation of EAT in children with high-risk FN is cost-saving and the preferred strategy for children with acute leukemia and high-risk FN in India.