Evidence map›Paper›PMID 39280919›Full record

ArticleGastro hep advances2024

Biomarkers Associated With Future Severe Liver Disease in Children With Alpha-1-Antitrypsin Deficiency.

Jeffrey H Teckman, Paula Buchanan, Keith Steven Blomenkamp, Nina Heyer-Chauhan, Keith Burling, David A Lomas

Abstract read
In one paragraph

Article in Gastro hep advances, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 4 papers.

0numbers the graph read from it
0cells of the map it votes in
4citing 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

4 citing papers in PubMed.

  1. Review
  2. Review
  3. Review
  4. Article
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

6 authors.

Jeffrey H TeckmanDepartment of Pediatrics and Biochemistry and Molecular Biology, St. Louis University School of Medicine, Cardinal Glennon Children's Hospital, St. Louis, Missouri.
Paula BuchananDepartment of Health and Clinical Outcomes Research, St. Louis University School of Medicine, St. Louis, Missouri.
Keith Steven BlomenkampDepartment of Pediatrics and Biochemistry and Molecular Biology, St. Louis University School of Medicine, Cardinal Glennon Children's Hospital, St. Louis, Missouri.
Nina Heyer-ChauhanDivision of Medicine, UCL Respiratory, University College London, London, UK.
Keith BurlingCore Biochemical Assay Laboratory, Cambridge University Hospitals NHS Foundation Trust, Cambridge, UK.
David A LomasDivision of Medicine, UCL Respiratory, University College London, London, UK.

Funding

NIH Prior Approval Process ProfessionalUL1TR002535 · NCATS · UNIVERSITY OF COLORADO DENVER · PI SOKOL, RONALD J. · 2018 to 2022
$51.1M
Childhood Liver Disease Research Network Data Coordinating CenterU01DK062456 · NIDDK · UNIVERSITY OF MICHIGAN AT ANN ARBOR · PI MAGEE, JOHN C, MERION, ROBERT M · 2002 to 2019
$36.8M
Childhood Liver Disease Research Network Data Coordinating CenterU24DK062456 · NIDDK · UNIVERSITY OF MICHIGAN AT ANN ARBOR · PI JOHN C MAGEE · 2019 to 2026
$18.9M
Continuation of the Childhood Liver Disease Research Network Seattle Clinical CenterU01DK084575 · NIDDK · SEATTLE CHILDREN'S HOSPITAL · PI Pamela Valentino · 2009 to 2026
$5.2M
NCATS NIH HHS UL1 TR002535NIDDK NIH HHS U01 DK062456NIDDK NIH HHS U01 DK084575NIDDK NIH HHS U24 DK062456
6 · The paper itself

Abstract

Background and Aims: Children with alpha-1-antitrypsin deficiency (AATD) exhibit a wide range of liver disease outcomes from portal hypertension and transplant to asymptomatic without fibrosis. Individual outcomes cannot be predicted. Liver injury in AATD is caused by the accumulation in hepatocytes of the mutant Z alpha-1-antitrypsin (AAT) protein, especially the toxic, intracellular polymerized conformation. AATD patients have trace Z polymer detectable in serum with unknown significance. Methods: The Childhood Liver Disease Research Network is an NIH consortium for the study of pediatric liver diseases, including AATD. We obtained data and samples with the aim of identifying biomarkers predictive of severe AATD liver disease. Results: We analyzed prospective AATD Childhood Liver Disease Research Network data and serum samples in 251 subjects from 2007 to 2015 for outcomes and Z polymer levels. Fifty-eight of 251 had clinically evident portal hypertension (CEPH) at enrollment, and 10 developed CEPH during follow-up. Higher Z AAT polymer levels were associated with existing CEPH ( Conclusion: High circulating Z polymer levels and high GGT early in life are associated with future CEPH in AATD, and the use of predictive cutoffs may assist in future clinical trial design.

Indexed as

Alpha-1-AntitrypsinCholestasisGamma Glutamyl TransferasePolymerPortal Hypertension

Identifiers

PMID39280919
PMCPMC11401556

What OpenQuestion holds

Textmetadata
LicenceCC BY-NC-ND
Read underepoch 390

Registered trials

None linked

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.