Authors
Angelo Di Giorgio, Marco Sciveres, Maurizio Fuoti, PierLuigi Calvo, Mara Cananzi, Ana Lleo, Simona Gatti, Giuseppe Indolfi, Annalisa Madeo, Claudia Mandato, Federica Nuti, Chiara Zanchi, Greta Carioli, Arianna Ghirardi, Emanuele Nicastro, Lorenzo D'Antiga
Published in
Digestive and liver disease : official journal of the Italian Society of Gastroenterology and the Italian Association for the Study of the Liver. Sep 22, 2026. Epub Sep 22, 2026.
Abstract
In our previous study we reported the efficacy of odevixibat treatment (OT) in a real-world cohort of children with progressive familial intrahepatic cholestasis (PFIC). Here we aim to identify the best cut-off of serum bile acid (sBA) early reduction predicting the response of pruritus at 6 months of treatment.
We reviewed data on children with PFIC treated with odevixibat. ROC analysis was performed to calculate the best cut-off of sBA reduction at 1 and 3 months associated with reduction in pruritus scale ≥1 point at 6 months.
24 patients [median age 6.6 years (3.7-12.1), M/F = 11/13] were enrolled; 16 (67%) had classic PFIC types (PFIC-1 = 2; PFIC-2 = 11; PFIC-3 = 3), 8 (33%) had rarer forms (PFIC-4 = 5, PFIC-5 = 1; PFIC-6 = 1; PFIC-9 = 1). At 6 months, 73% were pruritus responders. Reductions of sBA levels ≥48% from baseline at 1 month and ≥52% at 3 months were associated with improvements of pruritus at 6 (1st month: ROC-AUC 0.771; SE=0.75, SP=0.83; 3rd month: ROC-AUC 0.885; SE=0.81, SP=1.00).
reduction of sBA levels of around 50% from baseline value at 1 and 3 months is associated with a pruritus response to OT at 6 months. This information could be useful to the management of children with PFIC.
PMID:
42773017
Bibliographic data and abstract were imported from PubMed on 23 Sep 2026.
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