Ileal Bile Acid Transporter Blockers for Cholestatic Liver Disease in Pediatric Patients with Alagille Syndrome: A Systematic Review and Meta-Analysis

Muntaha, Hafiza Sidra Tul; Munir, Mubashar; Sajid, Syeda Haleema; Sarfraz, Zouina; Sarfraz, Azza; Robles‐Velasco, Karla; Sarfraz, Muzna; Félix, Miguel; Chérrez-Ojeda, Iván

doi:10.3390/jcm11247526

Cited by 5 publications

(3 citation statements)

References 43 publications

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“…Recent discoveries of ileal bile acid transport (IBAT) inhibitors, like odevixibat and maralixibat, approved by the FDA for Alagille syndrome treatment, present additional options for BRIC patients [72,73].…”

Section: Treatment Options and Future Challenges In Bricmentioning

confidence: 99%

Benign Recurrent Intrahepatic Cholestasis: Where Are We Now?

Geladari,

Vallianou,

Margellou

et al. 2024

Gastroenterology Insights

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Benign recurrent intrahepatic cholestasis (BRIC) stands as a rare genetic contributor to cholestasis, aligning itself within the spectrum of inherited intrahepatic cholestasis syndromes, such as progressive familial intrahepatic cholestasis (PFIC) and intrahepatic cholestasis of pregnancy. Manifesting in infancy or early adulthood, BRIC is marked by recurrent episodes of jaundice accompanied by intense pruritus, enduring from weeks to years across the lifespan. Normal gamma-glutamyl transferase (GGT) levels are a characteristic laboratory finding. Initially considered unlikely to progress to chronic liver disease or cirrhosis, some reports suggest BRIC may evolve into a continuous and progressive form of cholestasis. Moreover, these recurrent cholestatic episodes significantly impact quality of life, and certain mutations elevate the risk of hepatobiliary malignancy. Between episodes, histological findings of centrilobular cholestasis and abnormal laboratory parameters revert to normal, potentially obviating the need for liver biopsy. This review focuses on the genetic aspects of BRIC, its pathophysiology, clinical presentation, and prognosis. Additionally, it outlines triggering factors and available treatment options.

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Section: Treatment Options and Future Challenges In Bricmentioning

confidence: 99%

Benign Recurrent Intrahepatic Cholestasis: Where Are We Now?

Geladari,

Vallianou,

Margellou

et al. 2024

Gastroenterology Insights

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“…Several researchers strongly indicate the beneficial effect of IBAT inhibition in on reduction in skin itching and improvement of quality of life of patients with AGS (EE). Recent studies suggest that IBAT inhibitor-maralixibat may also delay or eliminate the need for LTx in this group of patients [44][45][46].…”

Section: Agsmentioning

confidence: 99%

Modified by the Innovative Drugs and Strategies—Pattern of Selected Indications for Pediatric Liver Transplantation

Jankowska,

Socha,

Gliwicz

et al. 2024

Pediatric Transplantation

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BackgroundLiver transplantation (LTx) constitutes a major life‐saving routine treatment for children with end‐stage liver disease. However, the analysis of LTx registries in children provides much information about changes in the indication profiles in the recent years.MethodsThe article provides a comprehensive review about the successes, hopes, and challenges related to changing indications for LTx in children based on the literature review and our own experience. Retrospective review of the indications for LTx at a tertiary referral pediatric hospital was also presented.Results and ConclusionsIn the context of the new therapies that have emerged, the need for LTx has decreased in patients with chronic hepatitis B and C infection and tyrosinemia type 1. In primary hyperoxaluria type 1, new RNAi‐based therapy has eliminated the requirement for LTx (both isolated or combined). There is a hope that introduction of ileal bile acid transporter (IBAT) blockers reduces the need for LTx in patients with Alagille syndrome or progressive familial intrahepatic cholestasis. The number of children qualified for LTx with urea cycle disorders (UCDs) as a prophylaxis of neurodevelopmental impairment is increasing.

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Section: Treatment Options and Future Challenges In Bricmentioning

confidence: 99%

Benign Recurrent Intrahepatic Cholestasis: Where are we now?

Geladari,

Vallianou,

Margellou

et al. 2023

Preprint

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Benign recurrent intrahepatic cholestasis (BRIC) is a rare genetic cause of cholestasis. It is considered as part of inherited intrahepatic cholestasis syndromes, such as progressive familial intrahepatic cholestasis (PFIC), and intrahepatic cholestasis of pregnancy. BRIC is presented in infancy or in early adulthood. It is characterized by exacerbations and remissions of jaundice with accompanying intense itching, lasting from weeks to years throughout lifetime. Normal gamma-glutamyl transferase (GGT) is a characteristic laboratory finding. Contrary to PFIC, which may progress to cirrhosis, BRIC does not progress to chronic liver disease or cirrhosis. However, incessant episodes of cholestasis result in marked reduction in quality of life and distinct mutations increase the risk of hepatobiliary malignancy. In intervals between the exacerbations, the histological findings of centrilobular cholestasis together with the abnormal laboratory parameters return to normal. In this context, liver biopsy might be avoided. In this review, we will focus on the genetic aspects of BRIC, its pathophysiology, clinical presentation, and prognosis of this autosomal recessive genetically determined cholestatic disorder. Moreover, triggering factors as well as treatment options will be further elucidated.

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Ileal Bile Acid Transporter Blockers for Cholestatic Liver Disease in Pediatric Patients with Alagille Syndrome: A Systematic Review and Meta-Analysis

Cited by 5 publications

References 43 publications

Benign Recurrent Intrahepatic Cholestasis: Where Are We Now?

Benign Recurrent Intrahepatic Cholestasis: Where Are We Now?

Modified by the Innovative Drugs and Strategies—Pattern of Selected Indications for Pediatric Liver Transplantation

Benign Recurrent Intrahepatic Cholestasis: Where are we now?

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