L-AmB as the Real First-Line Therapy for Paediatric Visceral Leishmaniasis: A Multicentre Retrospective Study from an Endemic Area of Southern Italy

Abstract

Background/Objectives: visceral leishmaniasis (VL) remains an important zoonotic disease in endemic Mediterranean regions, where children represent one of the populations at highest risk of developing symptomatic infection. Although pentavalent antimonials have historically been considered first-line therapy for VL, their use in Italy has become increasingly limited because of restricted availability, safety concerns, and the widespread adoption of liposomal amphotericin B (L-AmB). Methods: this multicentre retrospective observational study evaluated the contemporary management of paediatric VL in three tertiary referral centres in Catania, Southern Italy, between January 2016 and December 2025. Results: 19 children were included, with a mean age of 4.13 years. The most common clinical manifestations were prolonged fever, pancytopenia, and hepatosplenomegaly. Three different L-AmB regimens were identified, with the 10 mg/kg/day for two consecutive days schedule representing the most frequently adopted protocol. L-AmB was well tolerated; three patients (15.8%) experienced disease relapse during follow-up, all of whom responded successfully to additional L-AmB treatment, resulting in an overall clinical cure rate of 100%. Notably, no patient received pentavalent antimonials, reflecting their complete replacement in routine clinical practice. Conclusions: these findings provide real-world evidence that L-AmB has effectively become the contemporary first-line treatment and standard of care for paediatric VL in an endemic Mediterranean setting, highlighting the discrepancy between historical therapeutic paradigms and current clinical practice.