Evidence map›Paper›PMID 41046337›Full record

ArticleJournal of pediatric gastroenterology and nutrition2025

Odevixibat after liver transplant in patients with progressive familial intrahepatic cholestasis type 1: A case series.

Georg-Friedrich Vogel, Simone Kathemann, Andrea Pietrobattista, Giuseppe Maggiore, Denise Aldrian, Marco Sciveres, Henkjan J Verkade, Etienne Sokal, Giulia Jannone, Magdalena Salcedo and 4 more

Abstract readMulticenter Study
In one paragraph

Article in Journal of pediatric gastroenterology and nutrition, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 3 papers.

0numbers the graph read from it
0cells of the map it votes in
3citing papers in PubMed
–field-weighted citation impact
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

3 citing papers in PubMed.

  1. Article
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4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

14 authors.

Georg-Friedrich VogelDepartment of Paediatrics I, Medical University of Innsbruck, Innsbruck, Austria.
Simone KathemannDepartment of Pediatric Gastroenterology, Transplant Medicine, University Children's Hospital, Essen, Germany.
Andrea PietrobattistaHepatology and Liver Transplantation Unit, Bambino Gesù Children's Hospital, IRCCS, Rome, Italy.
Giuseppe MaggioreHepatology and Liver Transplantation Unit, Bambino Gesù Children's Hospital, IRCCS, Rome, Italy.
Denise AldrianDepartment of Paediatrics I, Medical University of Innsbruck, Innsbruck, Austria.
Marco SciveresHepatology and Liver Transplantation Unit, Bambino Gesù Children's Hospital, IRCCS, Rome, Italy.
Henkjan J VerkadePediatric Gastroenterology/Hepatology, Department of Pediatrics, University of Groningen, Beatrix Children's Hospital/University Medical Center Groningen, Groningen, the Netherlands.
Etienne SokalUniversité Catholique de Louvain, Cliniques St Luc, Brussels, Belgium.
Giulia JannoneUniversité Catholique de Louvain, Cliniques St Luc, Brussels, Belgium.
Magdalena SalcedoHepatology and Liver Transplantation Unit, CIBER-Ehd Hospital General Universitario Gregorio Marañón, Universidad Complutense, Madrid, Spain.
Peter RauschkolbIpsen Pharma GmBH, Munich, Germany.
Christof MauckschIpsen Pharma GmBH, Munich, Germany.
Velichka ValchevaIpsen Ireland, Dublin, Ireland.
Elke LainkaDepartment of Pediatric Gastroenterology, Transplant Medicine, University Children's Hospital, Essen, Germany.ORCID https://orcid.org/0000-0002-7624-2968

Funding

Ipsen
6 · The paper itself

Abstract

objectivesPatients with progressive familial intrahepatic cholestasis type 1 (PFIC1) who have undergone liver transplantation (LT) may have unmet needs and impacts on daily life due to post-LT complications, including diarrhea and hepatic steatosis. Here, we describe the effects of the ileal bile acid transporter inhibitor odevixibat on diarrhea and hepatic steatosis in a cohort of patients with PFIC1 post-LT.

methodsTreating physicians from six centers retrospectively collected data through July 2023 on patients with PFIC1 who received odevixibat post-LT. Data collected included demographics, medical history, and symptom presentation, characteristics of diarrhea, and liver imaging and/or histopathology.

resultsOverall, nine male patients with PFIC1 (seven aged <18 years at initial completion of the case report form) were included. In most patients, the primary indication for odevixibat treatment was diarrhea and/or steatosis post-LT. Odevixibat was initiated at a daily dose of 30-120 µg/kg (median exposure: 13 months). All patients had post-LT diarrhea, which was generally associated with negative impacts on daily life (e.g., ability to attend school, needing to wear diapers due to fecal urgency). After odevixibat initiation, most patients had improved diarrhea and positive impacts on daily life. Among five patients with post-LT steatosis and data available before and after odevixibat initiation, steatosis appeared to improve in three and did not change in two.

conclusionsOverall, the majority of patients with PFIC1 post-LT complications in this case series experienced improvements in diarrhea and daily activities with odevixibat. Treatment with odevixibat following LT also appeared to reduce steatosis in some patients. Further studies, particularly those with a prospective design, are needed to confirm these findings.

Indexed as

Cholestasis, IntrahepaticDiarrheaFatty LiverLiver TransplantationPostoperative ComplicationsAdolescentChildChild, PreschoolHumansInfantMaleRetrospective StudiesTreatment Outcomebile acids and saltsdiarrheaenterohepatic circulationliver diseasessteatosis

Identifiers

PMID41046337
PMCPMC12666498

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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 OpenQuestion graph.