Efficacy and safety of posaconazole and liposomal amphotericin B use for prophylaxis of invasive fungal infections in a paediatric and young adult haemato-oncological population

Background: The prevention of invasive fungal infections (IFIs) is crucial for paediatric haemato-oncological patients. This study evaluates the clinical efficacy and side-effects of posaconazole and liposomal amphotericin B (L-AmB) as primary prophylaxis. Materials and methods: This cohort study included patients aged 3 months to 21 years who received posaconazole or L-AmB (5 mg/kg twice weekly) as prophylaxis from January 2017 to March 2022 at the Hemato-oncological Pediatric Unit, University Hospital of Padua, Italy. Outcomes included adverse events and IFI diagnoses after the start of prophylaxis. Separate analyses were performed for patients with ALL and non-ALL diagnoses, and high-risk and low-risk groups. Cumulative incidence was calculated using the Kaplan-Meier estimator, with significant differences assessed using the log-rank test. Hazard ratios (HR) were estimated using Cox regression. Results: Fifty-one patients received posaconazole, and 37 received L-AmB. Adverse events occurred in 26% of L-AmB patients and 5.6% of posaconazole patients. IFI breakthrough events were similar in both groups (four events each). In ALL patients, 41% experienced adverse events with L-AmB, compared to 5% with posaconazole. After 1 year, the probability of adverse events was lower in the posaconazole group (54% versus 65%, P < 0.001). Overall, posaconazole was associated with a 91% lower risk of adverse events (HR: 0.07, P < 0.001). Among high-risk patients, IFI breakthrough rates were similar between groups (P = 0.964). Conclusions: Posaconazole was associated with fewer adverse events than L-AmB, and both drugs showed similar efficacy in preventing IFI breakthroughs, making posaconazole a viable alternative for primary prophylaxis.