Real-world experience with Odevixibat in children with Progressive Familial Intrahepatic Cholestasis

A previously published trial demonstrated that odevixibat is effective in the treatment of cholestatic pruritus of children with progressive familial intrahepatic cholestasis (PFIC). Real-world experience is necessary to confirm the results of registration trials having selective eligibility criteri...

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Bibliographic Details
Published in:JHEP reports 2024-12, p.101309, Article 101309
Main Authors: Di Giorgio, Angelo, Sciveres, Marco, Fuoti, Maurizio, Calvo, PierLuigi, Cananzi, Mara, Lleo, Ana, Gatti, Simona, Indolfi, Giuseppe, Madeo, Annalisa, Mandato, Claudia, Nuti, Federica, Zanchi, Chiara, Carioli, Greta, Ghirardi, Arianna, Nicastro, Emanuele, D’Antiga, Lorenzo
Format: Article
Language:English
Subjects:
bile acids
children
cholestasis
jaundice
odevixibat
PFIC
pruritus
real-life scenario
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Summary:A previously published trial demonstrated that odevixibat is effective in the treatment of cholestatic pruritus of children with progressive familial intrahepatic cholestasis (PFIC). Real-world experience is necessary to confirm the results of registration trials having selective eligibility criteria. We present our “real life experience” on odevixibat effectiveness and safety in patients with different PFIC subtypes. Multicenter prospective study of patients with PFIC treated with odevixibat (40 or escalated to 120 μg/kg/day). Pruritus was assessed by “Physician Global Impression of Symptom” at baseline and monthly up to 6 months. Serum bile acids (sBA) responders were patients who achieved a reduction in sBA levels ≥70% from baseline (or a value
ISSN:2589-5559
2589-5559
DOI:10.1016/j.jhepr.2024.101309