ArticleJHEP reports : innovation in hepatology2025
Real-world experience with odevixibat in children with progressive familial intrahepatic cholestasis.
Article in JHEP reports : innovation in hepatology, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 13 papers.
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Who cites it
13 citing papers in PubMed.
- Novel genetic variants and atypical phenotypes in pediatric progressive familial intrahepatic cholestasis.Pediatric research · 2026Article
- Clinical Relevance of Bile Acid and Bile Acid-Related Therapy in Liver Cirrhosis: A Review.Advances in therapy · 2026Review
- Odevixibat treatment for recurrent cholestasis in a patient with biallelic UNC45A variants causing osteo-oto-hepato-enteric syndrome.JHEP reports : innovation in hepatology · 2026Article
- Pediatric Cholestatic Diseases in the Era of Ileal Bile Acid Transporter (IBAT) Inhibitors.Pediatric reports · 2026Review
- [Research progress of inherited liver disease in 2025].Zhonghua gan zang bing za zhi = Zhonghua ganzangbing zazhi = Chinese journal of hepatology · 2026Review
- Microbial metabolites at the nexus of gut-brain communication and neurodevelopmental disorders.Frontiers in nutrition · 2026Review
- Case Report: Recurrent intrahepatic cholestasis: two rare cases with their novel variants of ATB8B1 and atypical clinical findings.Frontiers in medicine · 2026Article
- Functional inactivation of MDR3 caused by a homozygousFrontiers in genetics · 2026Article
- Reply to: "Managing PFIC 7 with odevixibat: Alleviation of pruritus and biochemical response".JHEP reports : innovation in hepatology · 2026Article
- Managing PFIC 7 with odevixibat: Alleviation of pruritus and biochemical response.JHEP reports : innovation in hepatology · 2026Article
- Odevixibat treatment reverses severe phenotype of PFIC in a young adult: A real-life experience beyond the genetic diagnosis.JHEP reports : innovation in hepatology · 2025Article
- New hope in treating progressive familial intrahepatic cholestasis in children.World journal of hepatology · 2025Review
- Case Report: Sustained biochemical remission following early initiation of odevixibat in an infant with monoallelic ABCB11 mutation and histologically confirmed PFIC2.Frontiers in pediatrics · 2025Article
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Authors and funding
16 authors.
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No grant is acknowledged in the PubMed record.
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 baseline (or a value <70 μmol/L) after 6 months; pruritus responders were patients who reported improvement in their pruritus score. Results: In total, 24 patients (median age 6.6 years [3.7-12.1], male:female = 11/13) were enrolled; 16 (67%) had classic PFIC types (PFIC-1, 2; PFIC-2, 11; and PFIC-3, 3), whereas eight (33%) had rarer forms (PFIC-4, 5, PFIC-5, 1; PFIC-6, 1; and PFIC-9, 1). All had high sBA levels and 22/24 (92%) had pruritus. Four (17%) had associated comorbidities.After 6 months of treatment, sBA decreased from a median of 317.1 μmol/L (range 82.3-369.0 μmol/L) to 45.6 μmol/L (range 7.2-120 μmol/L; Conclusions: Odevixibat is effective and safe in reducing sBA levels and improving pruritus in a real-life scenario in both patients with classic PFIC types and in those with other rarer subtypes. Dose escalation is required in some patients to improve the response to treatment. Impact and implications: 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.
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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the Socratic graph.