Case Report: Sustained biochemical remission following early initiation of odevixibat in an infant with monoallelic ABCB11 mutation and histologically confirmed PFIC2
This case study highlights the effectiveness of odevixibat in managing PFIC2, demonstrating sustained disease suppression and symptomatic relief. It also emphasizes the importance of comprehensive clinical evaluation and accurate disease characterization as well as the potential of targeted therapies in improving outcomes for patients with PFIC2.
Authors
- Jan Thomas Schaefer
- Michael Melter (ORCID: https://orcid.org/0000-0002-4194-0758)
- Maren Zapke
- André Hoerning (ORCID: https://orcid.org/0000-0001-6241-7168)
- Joachim Woelfle (ORCID: https://orcid.org/0000-0002-9132-6174)
- Katja Evert (ORCID: https://orcid.org/0000-0003-0984-5563)
- Thomas Kehler (ORCID: https://orcid.org/0000-0003-1973-201X)
Institutions
- Friedrich-Alexander-Universität Erlangen-Nürnberg (DE)
- University Hospital Regensburg (DE)
- University of Regensburg (DE)
Publication Details
- Journal
- Frontiers in Pediatrics
- Published
- 2025-11-27
- DOI
- https://doi.org/10.3389/fped.2025.1648663
- Primary Topic
- Immunodeficiency and Autoimmune Disorders
- Type
- article
- Field-Weighted Citation Impact
- 0.00
Funders
- Ipsen