SynthesisJournal of pediatric gastroenterology and nutrition2018
Systematic Review: The Epidemiology, Natural History, and Burden of Alagille Syndrome.
Synthesis in Journal of pediatric gastroenterology and nutrition, 2018. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 62 papers, 2 of them syntheses that pooled it.
What it found
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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.
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.
Who cites it
62 citing papers in PubMed, 2 syntheses or guidelines pooled it.
- Efficacy of ileal bile acid transport inhibitors in children with Alagille syndrome: a meta-analysis.European journal of pediatrics · 2025Pooled it
- The burden of Alagille syndrome: uncovering the potential of emerging therapeutics - a comprehensive systematic literature review.Journal of comparative effectiveness research · 2025Pooled it
- Indirect treatment comparison of odevixibat and maralixibat for the treatment of cholestatic pruritus in patients with Alagille syndrome.Journal of comparative effectiveness research · 2026Article
- Clinical and genetic characteristics of a large cohort of children with Alagille syndrome: identification of 57 new variants in the JAG1 gene.Journal of human genetics · 2026Article
- The disease burden and health-related quality of life in Chinese children with genetic cholestatic liver diseases: a cross-sectional study of progressive familial intrahepatic cholestasis and Alagille syndrome.Orphanet journal of rare diseases · 2026Article
- [Clinical characteristics and follow-up in children with Alagille syndrome].Zhonghua gan zang bing za zhi = Zhonghua ganzangbing zazhi = Chinese journal of hepatology · 2026Article
- Maralixibat for the treatment of severe xanthomas in two children with Alagille syndrome: Case reports.JPGN reports · 2026Article
- Treatment of intractable pruritus with maralixibat in patients with Alagille syndrome before and after reversal of biliary diversion.JPGN reports · 2025Article
- Odevixibat improves pruritus and bile acid level in Alagille syndrome: A case report.World journal of gastrointestinal pharmacology and therapeutics · 2025Article
- A Retrospective Study of Clinical and Genetic Features in a Long-Term Cohort of Mexican Children with Alagille Syndrome.International journal of molecular sciences · 2025Article
- Genetic disorders leading to severe hyperlipidemia in children: A case report.World journal of clinical cases · 2025Article
- Maralixibat Reduces Serum Bile Acids and Improves Cholestatic Pruritus in Adolescents With Alagille Syndrome.Liver international : official journal of the International Association for the Study of the Liver · 2025Article
- Pediatric Hepatocellular Carcinoma: A Review of Predisposing Conditions, Molecular Mechanisms, and Clinical Considerations.International journal of molecular sciences · 2025Review
- A rare cause of neonatal cholestasis: congenital portosystemic shunt.Proceedings (Baylor University. Medical Center) · 2025Article
- Beyond pruritus in Alagille syndrome: potential effects of maralixibat on fibrosis and portal hypertension-insights from two case studies.Frontiers in medicine · 2025Article
- Clinical and genetic characteristics of patients with Alagille syndrome in China: identification of six novelTranslational pediatrics · 2024Article
- Heritable Chronic Cholestatic Liver Diseases: A Review.Journal of clinical and translational hepatology · 2024Review
- Diseases of bile duct in children.World journal of gastroenterology · 2024Review
- An Approach to Investigations of Chronic Liver Disease.Indian journal of pediatrics · 2024Review
- Hepatoblastoma in a cirrhotic child with Alagille syndrome.BMJ case reports · 2024Article
2 more citing papers are in PubMed but not listed here.
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
5 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
BACKGROUND AND
aimAlagille syndrome (ALGS) is an inherited multisystem disorder typically manifesting as cholestasis, and potentially leading to end-stage liver disease and death. The aim of the study was to perform the first systematic review of the epidemiology, natural history, and burden of ALGS with a focus on the liver component.
methodsElectronic databases and proceedings from key congresses were searched in accordance with Preferred Reporting Items for Systematic Reviews and Meta-Analyses (PRISMA) 2009 guidelines. This analysis included publications reporting epidemiology, natural history, economic burden or health-related quality of life (HRQoL) outcomes in patients with ALGS.
resultsOf 525 screened publications, 20 met the inclusion criteria. Liver-related features included cholestasis (87%-100% of patients), jaundice (66%-85%), and cirrhosis (44%-95%). Between 15% and 47% of patients underwent liver transplantation and 4% to 14% received partial biliary diversion. Pruritus affected the majority of patients (59%-88%, of whom up to 45% had severe pruritus) and manifested during the first 10 years of life. Children with ALGS had significantly impaired HRQoL compared with healthy controls and those with other diseases. Itching was the symptom that most affected children with ALGS. No study assessed the economic burden of ALGS.
conclusionsOur findings consolidate information on the clinical course of ALGS, and highlight gaps in knowledge, most notably the absence of any research on the economic consequences of the disease. Further research is needed to establish the incidence of genetically confirmed ALGS. Disease-specific tools are also needed to improve the measurement of symptoms, such as itching, and better understand the impact of ALGS on HRQoL.
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