Model-informed repurposing of eliglustat for treatment and prophylaxis of Shiga toxin-producing Escherichia coli hemolytic-uremic syndrome (STEC-HUS) in children
Based on pharmacokinetic modeling, we developed oral and intravenous eliglustat dosing regimens that are likely safe and effective for treatment of STEC-HUS and prophylaxis in case of outbreaks of STEC infections. Clinical evaluation of these dosing regimens in children suspected of or diagnosed with STEC-HUS is required and should include assessment of pharmacokinetics, efficacy, and safety (e.g., ECG monitoring).
Authors
- Reena V. Kartha (ORCID: https://orcid.org/0000-0002-1692-1539)
- Rob ter Heine (ORCID: https://orcid.org/0000-0003-2185-8201)
- Jolien J. M. Freriksen (ORCID: https://orcid.org/0000-0002-4114-5412)
- Nicole C. A. J. van de Kar (ORCID: https://orcid.org/0000-0002-1990-1189)
- Mendy ter Avest (ORCID: https://orcid.org/0000-0001-5757-4919)
- Saskia N. de Wildt (ORCID: https://orcid.org/0000-0002-0502-0647)
- Kioa L. Wijnsma (ORCID: https://orcid.org/0000-0003-0019-109X)
- David F.G.J. Wolthuis (ORCID: https://orcid.org/0000-0002-2152-4490)
Institutions
- University of Minnesota (US)
- Radboud University Nijmegen (NL)
- Radboud University Medical Center (NL)
- Radboud Institute for Molecular Life Sciences (NL)
- Amalia Kinderziekenhuis (NL)
- Erasmus University Rotterdam (NL)
Publication Details
- Journal
- Pediatric Nephrology
- Published
- 2025-02-03
- DOI
- https://doi.org/10.1007/s00467-025-06688-3
- Citations
- 1
- Primary Topic
- Complement system in diseases
- Type
- article
- Field-Weighted Citation Impact
- 0.80