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Updated: May 7, 2026

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Liposomal amphotericin B prophylaxis in paediatrics: a systematic review

Emma V Thorley1,2, Jennifer Hatch3, Monica Li3

  • 1Centre for Neonatal and Paediatric Infection, City St. George's, University of London, London, UK.

The Journal of Antimicrobial Chemotherapy
|June 10, 2025
PubMed

Insights

This review found limited pediatric data for liposomal amphotericin B (LAmB) prophylaxis, with breakthrough invasive fungal diseases (IFD) in 7.2% of patients. Further research is needed to establish optimal dosing and efficacy.

Area of Science:

  • Pediatric pharmacology
  • Infectious diseases
  • Systematic reviews

Background:

  • Liposomal amphotericin B (LAmB) is frequently used off-label for invasive fungal disease (IFD) prophylaxis in high-risk pediatric patients.
  • Significant variability exists in LAmB dosing and frequency, necessitating a review of current evidence.

Purpose of the Study:

  • To systematically evaluate published data on prophylactic LAmB use in children.
  • To report breakthrough IFD rates and associated toxicity profiles in pediatric patients receiving LAmB prophylaxis.

Main Methods:

  • Systematic search of EMBASE, Medline, Web of Science, and Cochrane Database up to December 7, 2023.
  • Inclusion of 20 studies (3 trials, 12 cohort, 2 surveys, 3 PK studies) involving 2015 patients, adhering to PRISMA guidelines.

Main Results:

  • Breakthrough IFD occurred in 7.2% of patients (49/676).
  • Common toxicities included hypokalemia (23.2%) and abnormal liver function tests (15.0%).
  • Discontinuation due to toxicity was observed in 6.0% of patients.

Conclusions:

  • A significant lack of robust pediatric data supports the widespread use of prophylactic LAmB.
  • Heterogeneity in study designs and dosing regimens precludes definitive conclusions on efficacy or optimal dosing strategies.
  • High-quality clinical trials and targeted pharmacokinetic studies are essential for evidence-based LAmB prophylaxis in children.
Abstract

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