Isavuconazole: Real-World Data and Clinical Considerations for Pediatric Patients

Isavuconazole (ISAV) is a broad-spectrum triazole antifungal approved for the treatment of invasive aspergillosis and mucormycosis and has recently received approval for use in children ≥1 year of age. Pediatric approval was based on limited registration data, underscoring the importance of real-world evidence to better define efficacy and safety in children and adolescents. In this manuscript, we reviewed pediatric registration trials and real-world studies evaluating ISAV, with a focus on treatment and prophylaxis indications, pharmacokinetics, and safety. The literature review was conducted using PubMed, Embase, and Cochrane databases to identify relevant real-world and clinical studies published between January 1, 2005, and October 31, 2025. Studies reporting ISAV use in patients <18 years of age, including registration trials, observational cohorts, case series, and pharmacokinetic-pharmacodynamic studies, were included. Twenty-six real-world publications and two clinical registration trials met inclusion criteria. Among the real-world publications, ISAV was used for treatment in 23 studies and for prophylaxis in 5 studies, while 17 studies evaluated drug exposure or pharmacokinetics (several studies involved more than one mode of administration). Most patients had underlying hematologic or oncologic conditions. Across heterogeneous study populations and outcome definitions, complete or partial response was reported descriptively in approximately 72% of evaluable patients treated for invasive fungal disease, with breakthrough fungal infections reported in approximately 10% of patients receiving prophylaxis. Adverse events were generally mild and predominantly consisted of reversible hepatic laboratory abnormalities; serious drug-related toxicity, including one reported case of acute liver failure, was rare. Pharmacokinetic data demonstrated predictable exposure and achievement of target plasma concentrations in the majority of patients. Taken together, current evidence, although limited and derived largely from retrospective and salvage-treatment cohorts, suggests promising clinical activity and generally favorable tolerability of ISAV for invasive mold infections in pediatric populations. Real-world data support its use as a therapeutic option in selected patients, while underscoring the need for prospective studies to confirm efficacy, optimize dosing, and assess long-term safety.