Evidence map›Paper›PMID 42666930›Full record

ArticleFrontiers in pediatrics2026

Real-world use of maralixibat in biliary atresia: a case series.

Natasha Dilwali, Douglas B Mogul, Shannon M Vandriel, Tony Tokman, Catherine A Chapin, Bertrand Roquelaure, Rossitsa Rousseva, Johanna Ferreira, Mercedes Martinez, Benno Kohlmaier

Abstract readCase Reports
In one paragraph

Article in Frontiers in pediatrics, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.

0numbers the graph read from it
0cells of the map it votes in
0citing 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

0 citing papers in PubMed.

No citing paper in PubMed yet.

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

10 authors.

Natasha DilwaliDivision of Pediatric Gastroenterology, Hepatology, and Nutrition, Johns Hopkins University, Baltimore, MD, United States.
Douglas B MogulMirum Pharmaceuticals, Inc., Foster City, CA, United States.
Shannon M VandrielMirum Pharmaceuticals, Inc., Foster City, CA, United States.
Tony TokmanMirum Pharmaceuticals, Inc., Foster City, CA, United States.
Catherine A ChapinDepartment of Pediatrics, Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago, IL, United States.
Bertrand RoquelaureAPHM, Multidisciplinary Pediatrics Department, Timone Children's Hospital, Marseille, France.
Rossitsa RoussevaKing's College Hospital, London, United Kingdom.
Johanna FerreiraDivision of Pediatric Gastroenterology, Liver Disease and Nutrition, Cohen Children's Medical Center, New Hyde Park, NY, United States.
Mercedes MartinezDepartment of Pediatrics, Columbia University Irving Medical Center, New York, NY, United States.
Benno KohlmaierDepartment of Pediatrics, Medizinische Universität Graz, Graz, Austria.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Biliary atresia (BA) is the most common cause of cholestasis in infants, and pruritus may be a prominent and debilitating complication. Maralixibat is the first US Food and Drug Administration-approved drug for the treatment of cholestatic pruritus in children with Alagille syndrome and has been subsequently approved for treatment of progressive familial intrahepatic cholestasis as well. Several patients with BA have received maralixibat as part of a compassionate use program. We report the use of maralixibat for the treatment of cholestatic pruritus in BA for six patients. Demographics, past medical history, laboratory markers, and medications were collected. Pruritus was assessed using the Clinician Scratch Scale (CSS) at baseline and last clinical follow-up. All patients reported improved CSS with no increased usage of other antipruritic medications, and the medication was well tolerated.

Indexed as

case reportcholestasisIBAT inhibitorliver transplantpruritus

Identifiers

PMID42666930
PMCPMC13523770

What OpenQuestion holds

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Registered trials

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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.