Observational studyJournal of pediatric gastroenterology and nutrition2015
Baseline Analysis of a Young α-1-Antitrypsin Deficiency Liver Disease Cohort Reveals Frequent Portal Hypertension.
Observational study in Journal of pediatric gastroenterology and nutrition, 2015. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT02922751 (Childhood Liver Disease Research Network), which is not on this map. Cited by 14 papers.
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.
Childhood Liver Disease Research Network (ChiLDReN): FibroScan™ in Pediatric Cholestatic Liver Disease (FORCE) Study Protocol
Who cites it
14 citing papers in PubMed, 49 citations in OpenAlex.
- Fibrosis, biomarkers and liver biopsy in AAT deficiency and relation to liver Z protein polymer accumulation.Liver international : official journal of the International Association for the Study of the Liver · 2024Article
- Alpha-1-Antitrypsin Deficiency in Children-Unmet Needs Concerning the Liver Manifestation.Children (Basel, Switzerland) · 2024Article
- Biomarkers Associated With Future Severe Liver Disease in Children With Alpha-1-Antitrypsin Deficiency.Gastro hep advances · 2024Article
- Neonatal cholestasis in children with Alpha-1-AT deficiency is a risk for earlier severe liver disease with male predominance.Hepatology communications · 2023Article
- Risk of variceal hemorrhage and pretransplant mortality in children with biliary atresia.Hepatology (Baltimore, Md.) · 2022Article
- Alpha-1-Antitrypsin Deficiency.Clinical liver disease · 2022Review
- Liver disease with unknown etiology - have you ruled out alpha-1 antitrypsin deficiency?Therapeutic advances in chronic disease · 2021Review
- Alpha-1 antitrypsin deficiency liver disease.Translational gastroenterology and hepatology · 2021Review
- Longitudinal Outcomes in Young Patients with Alpha-1-Antitrypsin Deficiency with Native Liver Reveal that Neonatal Cholestasis is a Poor Predictor of Future Portal Hypertension.The Journal of pediatrics · 2020Observational
- Alpha-1 Antitrypsin Deficiency-Mediated Liver Toxicity: Why Do Some Patients Do Poorly? What Do We Know So Far?Chronic obstructive pulmonary diseases (Miami, Fla.) · 2020Review
- Article
- Liver Involvement in Children with Alpha-1 Antitrypsin Deficiency: A Multicenter Study.Pediatric gastroenterology, hepatology & nutrition · 2020Article
- Clinically Evident Portal Hypertension: An Operational Research Definition for Future Investigations in the Pediatric Population.Journal of pediatric gastroenterology and nutrition · 2019Article
- Heterozygosity for the alpha-1-antitrypsin Z allele in cirrhosis is associated with more advanced disease.Liver transplantation : official publication of the American Association for the Study of Liver Diseases and the International Liver Transplantation Society · 2018Article
Corrections and comments
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Authors and funding
22 authors at 17 institutions in 2 countries.
Funding
Abstract
objectivesα-1-Antitrypsin (A1AT) deficiency is a common genetic disease with an unpredictable and highly variable course. The Childhood Liver Disease Research and Education Network is a National Institutes of Health, multicenter, longitudinal consortium studying pediatric liver diseases, with the objective of prospectively defining natural history and identifying disease modifiers.
methodsLongitudinal, cohort study of A1AT patients' birth through 25 years diagnosed as having liver disease, type PIZZ or PISZ. Medical history, physical examination, laboratory, imaging, and standardized survey tool data were collected during the provision of standard of care.
resultsIn the present report of the cohort at baseline, 269 subjects were enrolled between November 2008 and October 2012 (208 with their native livers and 61 postliver transplant). Subjects with mild disease (native livers and no portal hypertension [PHT]) compared to severe disease (with PHT or postliver transplant) were not different in age at presentation. A total of 57% of subjects with mild disease and 76% with severe disease were jaundiced at presentation (P = 0.0024). A total of 29% of subjects with native livers had PHT, but age at diagnosis and growth were not different between the no-PHT and PHT groups (P > 0.05). Subjects with native livers and PHT were more likely to have elevated bilirubin, ALT, AST, INR, and GGTP than the no-PHT group (P << 0.001), but overlap was large. Chemistries alone could not identify PHT.
conclusionsMany subjects with A1AT presenting with elevated liver tests and jaundice improve spontaneously. Subjects with PHT have few symptoms and normal growth. Longitudinal cohort follow-up will identify genetic and environmental disease modifiers.
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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 OpenQuestion graph.