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Liposomal amphotericin B prophylaxis in paediatrics: a systematic review

Summary

This review examined how well liposomal amphotericin B (a fungal medication) works to prevent serious fungal infections in children. Researchers looked at 20 studies involving over 2000 children and found that about 7% still developed fungal infections despite the medication, with common side effects being low potassium levels and liver problems. The main finding is that doctors use this medication in very different ways, and we need better research to figure out the best dosing strategy for children.

Background

Liposomal amphotericin B (LAmB) is widely used for prophylaxis in paediatric patients at high risk of invasive fungal diseases (IFD), but its use is off-label with significant variability in dosage and frequency. Despite widespread clinical use, there is limited evidence supporting optimal prophylactic regimens in the paediatric population.

Objective

This systematic review evaluated published data on prophylactic LAmB use in paediatric patients to determine efficacy in preventing breakthrough invasive fungal disease and to characterize the associated toxicity profile across different dosing regimens.

Results

Twenty studies with 2015 patients were included; 717 had individual patient data. Breakthrough IFD occurred in 7.2% (49/676 patients), with 75.5% proven cases predominantly caused by Candida species (56%) and Aspergillus (32%). Most common adverse effects were hypokalaemia (23.2%) and liver function abnormalities (15.0%); 6.0% discontinued due to toxicity. Four pharmacokinetic studies showed variable serum and CSF amphotericin B concentrations depending on dosing regimens.

Conclusion

Despite widespread prophylactic use of LAmB in paediatrics, significant heterogeneity in dosing regimens, study designs, and patient populations prevents firm conclusions on optimal efficacy. The review highlights the critical need for high-quality clinical trials and targeted pharmacokinetic/pharmacodynamic studies to establish evidence-based prophylactic protocols in children.
  • Published in:Journal of Antimicrobial Chemotherapy,
  • Study Type:Systematic Review,
  • Source: PMID: 40493030, DOI: 10.1093/jac/dkaf171
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