Real-world experience with odevixibat in children with progressive familial intrahepatic cholestasis
Published data on the use of odevixibat in a real-world scenario are lacking. We explored the effectiveness of odevixibat in a heterogenous cohort of children diagnosed with PFIC (including patients with classic as well as rarer types of PFIC, and with advanced liver disease and associated comorbidities). Our results demonstrate that odevixibat is effective for the treatment of cholestasis and pruritus in children with different PFIC subtypes in a real-life scenario. These results support the use of odevixibat in children with any type of PFICs, including those with different stages of liver disease and comorbidities.
Authors
- Giuseppe Indolfi (ORCID: https://orcid.org/0000-0003-3830-9823)
- Angelo Di Giorgio (ORCID: https://orcid.org/0000-0003-0363-5565)
- Claudia Mandato (ORCID: https://orcid.org/0000-0003-2425-5449)
- Greta Carioli (ORCID: https://orcid.org/0000-0002-7012-707X)
- Emanuele Nicastro (ORCID: https://orcid.org/0000-0002-4518-9375)
- Maurizio Fuoti (ORCID: https://orcid.org/0000-0002-9182-822X)
- Marco Sciveres (ORCID: https://orcid.org/0000-0002-4092-9071)
- Lorenzo D’Antiga (ORCID: https://orcid.org/0000-0001-7150-3148)
- Federica Nuti (ORCID: https://orcid.org/0000-0003-4240-4524)
- Ana Lleò (ORCID: https://orcid.org/0000-0002-0561-7902)
- Pier Luigi Calvo (ORCID: https://orcid.org/0000-0001-9264-3465)
- Annalisa Madeo (ORCID: https://orcid.org/0000-0002-2889-6959)
- Arianna Ghirardi (ORCID: https://orcid.org/0000-0001-9391-6963)
- Simona Gatti (ORCID: https://orcid.org/0000-0002-5923-5213)
- Mara Cananzi (ORCID: https://orcid.org/0000-0002-7177-2847)
- Chiara Zanchi (ORCID: https://orcid.org/0000-0001-8513-1963)
Institutions
- Marche Polytechnic University (IT)
- University of Salerno (IT)
- Humanitas University (IT)
- University of Padua (IT)
- IRCCS Materno Infantile Burlo Garofolo (IT)
- Istituto Giannina Gaslini (IT)
- Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico (IT)
- Azienda Ospedaliera Citta' della Salute e della Scienza di Torino (IT)
- Azienda Socio Sanitaria Territoriale degli Spedali Civili di Brescia (IT)
- Ospedale Papa Giovanni XXIII (IT)
- Meyer Children's Hospital (IT)
- Bambino Gesù Children's Hospital (IT)
- Istituti di Ricovero e Cura a Carattere Scientifico (IT)
- IRCCS Humanitas Research Hospital (IT)
- Istituto Mediterraneo per i Trapianti e Terapie ad Alta Specializzazione (IT)
- University of Milano-Bicocca (IT)
Publication Details
- Journal
- JHEP Reports
- Published
- 2024-12-20
- DOI
- https://doi.org/10.1016/j.jhepr.2024.101309
- Citations
- 6
- Primary Topic
- Pediatric Hepatobiliary Diseases and Treatments
- Type
- article
- Field-Weighted Citation Impact
- 3.44