Twice-Weekly Micafungin as Antifungal Prophylaxis in Children Undergoing Hematopoietic Stem Cell Transplantation.
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BACKGROUND: Children undergoing hematopoietic stem cell transplantation are at increased risk of developing invasive fungal infection. Micafungin is an echinocandin with broad-spectrum antifungal properties. Recent trials have reported on the efficacy of intermittent daily dosing of micafungin for fungal prophylaxis in pediatric patients undergoing transplantation, with success rates of 76%-99%, success defined as absence of proven, probable, or possible invasive fungal infection. We implemented a twice-weekly micafungin regimen as antifungal prophylaxis in our unit. PATIENTS AND METHODS: One hundred consecutive transplants performed between May 2018 and February 2024 were analyzed. Micafungin prophylaxis was dosed at 3-5 mg/kg daily given twice weekly from Day 0 till stable neutrophil engraftment, after which oral fluconazole was prescribed till Day +100. Fungal infection status was evaluated by radiological procedures and cultures from blood or sterile body sites as clinically indicated. The primary endpoint of the study was the evaluation of efficacy; secondary endpoints were safety and tolerability. RESULTS: Ninety three patients who underwent 95 transplants fulfilled the inclusion criteria. Underlying diagnoses were: leukemia (47), inborn errors of immunity (11), other malignancies (11), hemoglobinopathy (8), and others (16). There were no proven or probable fungal infections. Seventeen patients (18%) required change from prophylaxis to empirical antifungal treatment for persistent fever (n = 5) and possible fungal infections, mainly oesophagitis (n = 8). All patients who failed micafungin prophylaxis received allogeneic transplants, and HLA-haploidentical was the majority (14/17). None of the patients had to discontinue micafungin due to safety or tolerability concerns. CONCLUSIONS: Twice-weekly IV micafungin is a safe and effective option as antifungal prophylaxis for children undergoing HSCT.