Evidence map›Paper›PMID 32663593›Full record

Observational studyThe Journal of pediatrics2020

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.

Jeffrey Teckman, Philip Rosenthal, Kieran Hawthorne, Cathie Spino, Lee M Bass, Karen F Murray, Nanda Kerkar, John C Magee, Saul Karpen, James E Heubi and 8 more

Open access · greenAbstract readMulticenter StudyObservational Study
PubMed Full text at PMC Publisher
In one paragraph

Observational study in The Journal of pediatrics, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 6 papers.

0numbers the graph read from it
0cells of the map it votes in
6citing papers in PubMed
2.2field-weighted citation impact, top 12% of its field
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

6 citing papers in PubMed, 17 citations in OpenAlex.

  1. 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 · 2024
    Article
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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

18 authors at 17 institutions in 2 countries.

Jeffrey TeckmanPediatrics and Biochemistry, Saint Louis University, Cardinal Glennon Children's Medical Center, Saint Louis, MO.
Philip RosenthalPediatrics and Surgery, University of California San Francisco, San Francisco, CA.
Kieran HawthorneArbor Research Collaborative for Health, Ann Arbor, MI.
Cathie SpinoBiostatistics, University of Michigan, Ann Arbor, MI.
Lee M BassPediatrics, Northwestern University Feinberg School of Medicine, Chicago, IL.
Karen F MurrayPediatric Gastroenterology and Hepatology, Seattle Children's Hospital, University of Washington School of Medicine, Seattle, WA.
Nanda KerkarPediatric Gastroenterology, Children's Hospital Los Angeles, Los Angeles, CA.
John C MageeSurgery, University of Michigan School of Medicine, Ann Arbor, MI.
Saul KarpenPediatrics, Emory University, Children's Healthcare Atlanta, Atlanta, GA.
James E HeubiPediatric Gastroenterology and Hepatology, Children's Hospital Medical Center, Cincinnati, OH.
Jean P MollestonPediatric Gastroenterology, Hepatology and Nutrition, James Whitcomb Riley Hospital for Children, Indianapolis, IN.
Robert H SquiresPediatrics, University of Pittsburgh, Pittsburgh, PA.
Binita M KamathPediatric Gastroenterology, Hepatology and Nutrition, The Hospital for Sick Children, Toronto, Ontario, Canada.
Stephen L GutheryDepartment of Pediatrics, Division of Gastroenterology, Hepatology and Nutrition, University of Utah, and Intermountain Primary Children's Hospital, Salt Lake City, UT.
Kathleen M LoomesPediatric Gastroenterology, Children's Hospital of Philadelphia, Philadelphia, PA.
Averell H SherkerNational Institute of Diabetes, Digestive and Kidney Disease, National Institutes of Health, Baltimore, MD.
Ronald J SokolPediatric Gastroenterology, Hepatology and Nutrition, University of Colorado School of Medicine, Children's Hospital Colorado, Aurora, CO.
ChiLDReN (Childhood Liver Disease Research Network)
University of Michigan–Ann Arbor · USUniversity of Pittsburgh · USCardinal Glennon Children’s Medical Center · USChildren's Healthcare of Atlanta · USChildren's Hospital Colorado · USChildren's Hospital of Philadelphia · USCincinnati Children's Hospital Medical Center · USHospital for Sick Children · CANational Institute of Diabetes and Digestive and Kidney Diseases · USNorthwestern University · USRiley Hospital for Children · USUniversity of California, San Francisco · USUniversity of Washington · USArbor Research Collaborative for Health · USChildren's Hospital of Los Angeles · USPrimary Children's Hospital · USUniversity of Utah · US

Funding

WU INSTITUTE OF CLINICAL AND TRANSLATIONAL SCIENCESUL1TR002345 · NCATS · WASHINGTON UNIVERSITY · PI William G. Powderly · 2017 to 2026
$97.8M
Southern California Clinical and Translational Science InstituteUL1TR001855 · NCATS · UNIVERSITY OF SOUTHERN CALIFORNIA · PI Thomas A Buchanan, Michele D. Kipke · 2016 to 2026
$81.3M
Engaging University of California Stakeholders for Biorespository ResearchUL1TR000004 · NCATS · UNIVERSITY OF CALIFORNIA, SAN FRANCISCO · PI GRANDIS, JENNIFER RUBIN · 2012 to 2015
$78.0M
QAQC Johns Hopkins Institute for Clinical and Translational ResearchUL1TR003098 · NCATS · JOHNS HOPKINS UNIVERSITY · PI FORD, DANIEL ERNEST · 2019 to 2023
$57.7M
Northwestern University Clinical and Translational Science Institute (NUCATS)UL1TR001422 · NCATS · NORTHWESTERN UNIVERSITY AT CHICAGO · PI D'AQUILA, RICHARD · 2015 to 2023
$56.8M
NIH Prior Approval Process ProfessionalUL1TR002535 · NCATS · UNIVERSITY OF COLORADO DENVER · PI SOKOL, RONALD J. · 2018 to 2022
$51.1M
University of Pittsburgh Clinical and Translational Science InstituteUL1TR000005 · NCATS · UNIVERSITY OF PITTSBURGH AT PITTSBURGH · PI REIS, STEVEN E · 2012 to 2015
$50.8M
Institute of Translational Health SciencesUL1TR000423 · NCATS · UNIVERSITY OF WASHINGTON · PI DISIS, MARY L. · 2012 to 2016
$49.4M
Washington University Institute of Clinical and Translational SciencesUL1TR000448 · NCATS · WASHINGTON UNIVERSITY · PI EVANOFF, BRADLEY A · 2012 to 2016
$41.4M
Institutional Clinical and Translational Science AwardUL1TR000003 · NCATS · UNIVERSITY OF PENNSYLVANIA · PI FITZGERALD, GARRET A · 2012 to 2015
$38.9M
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
Southern California Clinical and Translational Science InstituteUL1TR000130 · NCATS · UNIVERSITY OF SOUTHERN CALIFORNIA · PI BUCHANAN, THOMAS A · 2012 to 2015
$36.0M
AHRQ HHS K08 HS026510NCATS NIH HHS UL1 TR000003NCATS NIH HHS UL1 TR000004NCATS NIH HHS UL1 TR000005NCATS NIH HHS UL1 TR000006NCATS NIH HHS UL1 TR000077NCATS NIH HHS UL1 TR000130NCATS NIH HHS UL1 TR000150NCATS NIH HHS UL1 TR000423NCATS NIH HHS UL1 TR000424NCATS NIH HHS UL1 TR000448NCATS NIH HHS UL1 TR001422NCATS NIH HHS UL1 TR001855NCATS NIH HHS UL1 TR002345NCATS NIH HHS UL1 TR002535NCATS NIH HHS UL1 TR003098NIDDK NIH HHS P30 DK078392NIDDK NIH HHS U01 DK062436NIDDK NIH HHS U01 DK062445NIDDK NIH HHS U01 DK062452NIDDK NIH HHS U01 DK062453NIDDK NIH HHS U01 DK062456NIDDK NIH HHS U01 DK062466NIDDK NIH HHS U01 DK062470NIDDK NIH HHS U01 DK062481NIDDK NIH HHS U01 DK062497NIDDK NIH HHS U01 DK062500NIDDK NIH HHS U01 DK062503NIDDK NIH HHS U01 DK084536NIDDK NIH HHS U01 DK084538NIDDK NIH HHS U01 DK084575NIDDK NIH HHS U01 DK103135NIDDK NIH HHS U01 DK103140NIDDK NIH HHS U01 DK103149NIDDK NIH HHS U24 DK062456
6 · The paper itself

Abstract

objectivesTo identify predictors of portal hypertension, liver transplantation, and death in North American youth with alpha-1-antitrypsin (AAT) deficiency, and compare with patients with AAT deficiency elsewhere. STUDY

designThe Childhood Liver Disease Research Network Longitudinal Observational Study of Genetic Causes of Intrahepatic Cholestasis is a prospective, cohort study of pediatric cholestatic liver diseases, including AAT deficiency, enrolling PIZZ and PISZ subjects 0-25 years of age seen since November 2007 at 17 tertiary care centers in the US and Canada. Data from standard-of-care baseline and annual follow-up visits were recorded from medical records, history, physical examination, and laboratory studies. Participants with portal hypertension were identified based on data collected.

resultsWe enrolled 350 participants (60% male) with a native liver; 278 (79%) entered the cohort without portal hypertension and 18 developed portal hypertension during follow-up. Thirty participants required liver transplantation; 2 patients died during 1077 person-years of follow-up. There was no difference in participants with or without preceding neonatal cholestasis progressing to transplantation or death during the study (12% vs 7%; P = .09), or in experiencing portal hypertension (28% vs 21%; P = .16); the hazard ratio for neonatal cholestasis leading to portal hypertension was P = .04. Development of portal hypertension was associated with a reduced height Z-score.

conclusionsPortal hypertension in youth with AAT deficiency impacts growth measures. Progression to liver transplantation is slow and death is rare, but the risk of complications and severe liver disease progression persists throughout childhood. A history of neonatal cholestasis is a weak predictor of severe disease.

Indexed as

AdolescentAdultalpha 1-Antitrypsin DeficiencyChildChild, PreschoolCholestasis, IntrahepaticDisease ProgressionFemaleHumansHypertension, PortalInfantInfant, NewbornLiver TransplantationLongitudinal StudiesMaleYoung Adultcirrhosisjaundiceliver enzymesliver transplantmetabolic liver disease

Identifiers

PMID32663593
PMCPMC7686087
OpenAlexW3041936813

What OpenQuestion holds

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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.