Real-world experience with odevixibat in children with progressive familial intrahepatic cholestasis.

ABSTRACT:

Background & aims

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 with selective eligibility criteria. We present our 'real-life experience' of the effectiveness and safety of odevixibat in patients with different PFIC subtypes.

Methods

We carried out a 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

SUBMITTER: Di Giorgio A 

PROVIDER: S-EPMC11937657 | biostudies-literature | 2025 Apr

REPOSITORIES: biostudies-literature