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PERSPECTIVE article

Front. Pediatr.

Sec. Children and Health

Volume 13 - 2025 | doi: 10.3389/fped.2025.1635252

Optimization and prioritization of paediatric drugs for visceral leishmaniasis

Provisionally accepted
Tiziana  MasiniTiziana Masini1Ana Nilce  Silveira Maia-ElkhouryAna Nilce Silveira Maia-Elkhoury2Dinesh  MondalDinesh Mondal3Piero  L OlliaroPiero L Olliaro4Khechar  PaudelKhechar Paudel5Martina  PenazzatoMartina Penazzato6Samantha Yuri  Oshiro Branco ValadasSamantha Yuri Oshiro Branco Valadas2Aya  YajimaAya Yajima7Abate  BeshahAbate Beshah8Supriya  WarusavithanaSupriya Warusavithana9SAURABH  JAINSAURABH JAIN6*
  • 1World Health Organization (Switzerland), Geneva, Switzerland
  • 2Pan American Health Organization - World Health Organization, Regional Office of the Americas, Brazil, Sao Paulo, Brazil
  • 3icddr,b, 68Shaheed Tajuddin Ahmed Saranai, Mokakhali, Dhaka, Bangladesh
  • 4International Severe Acute Respiratory and emerging Infection Consortium, Pandemic Sciences Institute, University of Oxford, Oxford, United Kingdom
  • 5Province Hospital, Surkhet, Nepal
  • 6World Health Organization, Geneva, Switzerland
  • 7World Health Organization, Regional Office for South-East Asia, New Delhi, India
  • 8World Health Organization, Regional Office for Africa, Brazzaville, Republic of Congo
  • 9World Health Organization, Regional Office of the Eastern Mediterranean, Cairo, Egypt

The final, formatted version of the article will be published soon.

Visceral leishmaniasis (VL) is a fatal disease if left untreated. Globally, at least 50% of VL cases are reported to be children younger than 15 years, with a higher incidence among males. VL is intrinsically associated with poverty and poor social determinants of health. Malnutrition and immune suppression are risk factors for severe VL, making children particularly vulnerable to this disease. Available treatment options vary depending on the eco-epidemiological context, but are in general suboptimal, especially for children. In 2023, the World Health Organization convened a paediatric drug optimization exercise (PADO) for VL, bringing together more than 60 experts globally in the field of VL to identify formulations of VL medicines to prioritize for development to address the specific needs of children. The group prioritized a 20 mg scored, dispersible tablet formulation of miltefosine and an oral solid dosage form of amphotericin B, acknowledging recent developments in allometric dosing for miltefosine and ongoing research for the development of oral amphotericin B. For miltefosine, this prompted an ongoing update of the WHO Prequalification Expression of interest to promote generic manufacturing. A compound with a new mechanism of action, LXE408, which is currently being investigated in Phase II, was included in the PADO watch list, signalling that paediatric investigations should start as soon as enough data are available from adult studies, not to delay access to latest available innovations for children with VL.

Keywords: Visceral leishmaniasis, kala-azar, Paediatrics, miltefosine, Amphotericin B

Received: 13 Jun 2025; Accepted: 31 Jul 2025.

Copyright: © 2025 Masini, Maia-Elkhoury, Mondal, Olliaro, Paudel, Penazzato, Branco Valadas, Yajima, Beshah, Warusavithana and JAIN. This is an open-access article distributed under the terms of the Creative Commons Attribution License (CC BY). The use, distribution or reproduction in other forums is permitted, provided the original author(s) or licensor are credited and that the original publication in this journal is cited, in accordance with accepted academic practice. No use, distribution or reproduction is permitted which does not comply with these terms.

* Correspondence: SAURABH JAIN, World Health Organization, Geneva, Switzerland

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