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Liposomal amphotericin B prophylaxis in paediatrics: a systematic review

Summary

This study reviews how a fungal medication called liposomal amphotericin B is used to prevent serious fungal infections in children with weakened immune systems, particularly those with blood cancers or who have had bone marrow transplants. The researchers found that this medicine prevents infections in about 93% of children, though it does cause side effects like low potassium levels in about 23% of patients. However, the study shows that current use of this medicine is quite varied and more high-quality research is needed to determine the best dosing approach.

Background

Liposomal amphotericin B (LAmB) is widely used for prophylaxis in paediatric patients at high risk of invasive fungal diseases (IFD), despite being off-label with significant variability in dosage and frequency. Invasive fungal diseases are a significant concern in the immunocompromised paediatric population, particularly in haemato-oncology and post-hematopoietic cell transplantation patients.

Objective

To evaluate published data on prophylactic LAmB use in the paediatric population and present reported proportions of breakthrough IFD and associated toxicity profile. The review aimed to synthesize evidence on dosing regimens, efficacy, and safety outcomes.

Results

Overall breakthrough IFD rate was 7.2% (49/676 patients), with 75.5% proven, 18.4% probable, and 6.1% possible cases. Most common adverse effects were hypokalaemia (23.2%), liver function test derangement (15.0%), and renal impairment (10.2%). Drug discontinuation due to toxicity occurred in 6.0% of patients, with infusion-related reactions being the most common reason.

Conclusion

Despite widespread prophylactic use of LAmB in paediatrics, this systematic review highlights paucity of paediatric data supporting its use. Significant heterogeneity in populations, dosing regimens, and study design prevents conclusions on efficacy or superiority of one regimen. Clear need exists for further high-quality clinical data and targeted pharmacokinetic studies.
  • Published in:Journal of Antimicrobial Chemotherapy,
  • Study Type:Systematic Review,
  • Source: PMID: 40493030, DOI: 10.1093/jac/dkaf171
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