Evidence map›Paper›PMID 34258144›Full record

ArticleJIMD reports2021

Liver transplantation in children with inborn errors of metabolism: 30 years experience in NSW, Australia.

Noha Elserafy, Sue Thompson, Troy Dalkeith, Michael Stormon, Gordon Thomas, Albert Shun, Janine Sawyer, Shanti Balasubramanian, Kaustuv Bhattacharya, Nadia Badawi and 1 more

Open access · goldAbstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in JIMD reports, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 7 papers.

0numbers the graph read from it
0cells of the map it votes in
7citing papers in PubMed
1.4field-weighted citation impact, top 21% 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

7 citing papers in PubMed, 13 citations in OpenAlex.

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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

11 authors at 2 institutions in 1 country.

Noha ElserafyGenetic Metabolic Disorders Service, The Children's Hospital at Westmead Sydney Children's Hospital Network Sydney New South Wales Australia.ORCID https://orcid.org/0000-0002-5598-6677
Sue ThompsonGenetic Metabolic Disorders Service, The Children's Hospital at Westmead Sydney Children's Hospital Network Sydney New South Wales Australia.
Troy DalkeithGenetic Metabolic Disorders Service, The Children's Hospital at Westmead Sydney Children's Hospital Network Sydney New South Wales Australia.
Michael StormonPaediatric divison The University of Sydney Sydney New South Wales Australia.
Gordon ThomasPaediatric divison The University of Sydney Sydney New South Wales Australia.
Albert ShunPaediatric divison The University of Sydney Sydney New South Wales Australia.
Janine SawyerDepartment of Gastroenterology, The Children's Hospital at Westmead Sydney Children's Hospital Network Sydney New South Wales Australia.
Shanti BalasubramanianGenetic Metabolic Disorders Service, The Children's Hospital at Westmead Sydney Children's Hospital Network Sydney New South Wales Australia.
Kaustuv BhattacharyaGenetic Metabolic Disorders Service, The Children's Hospital at Westmead Sydney Children's Hospital Network Sydney New South Wales Australia.
Nadia BadawiPaediatric divison The University of Sydney Sydney New South Wales Australia.
Carolyn EllawayGenetic Metabolic Disorders Service, The Children's Hospital at Westmead Sydney Children's Hospital Network Sydney New South Wales Australia.
The University of Sydney · AUChildren's Hospital at Westmead · AU

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

backgroundInborn errors of metabolism (IEM) are a diverse group of genetic disorders that can result in significant morbidity and sometimes death. Metabolic management can be challenging and burdensome for families. Liver transplantation (LT) is increasingly being considered a treatment option for some IEMs. IEMs are now considered the second most common reason for pediatric LT.

aimTo review the data of all children with an IEM who had LT at The Children's Hospital at Westmead (CHW), NSW, Australia between January 1986 and January 2019.

methodsRetrospective data collected from the medical records and genetic files included patient demographics, family history, parental consanguinity, method of diagnosis of IEM, hospital and intensive care unit admissions, age at LT, graft type, clinical outcomes and metabolic management pre and post-LT.

resultsTwenty-four LT were performed for 21 patients. IEM diagnoses were MSUD (n = 4), UCD (n = 8), OA (n = 6), TYR type I (n = 2) and GSD Ia (n = 1). Three patients had repeat transplants due to complications. Median age at transplant was 6.21 years (MSUD), 0.87 years (UCD), 1.64 years (OA) and 2.2 years (TYR I). Two patients died peri-operatively early in the series, one died 3 months after successful LT due to septicemia. Eighteen LTs have been performed since 2008 in comparison to six LT prior to 2008. Dietary management was liberalized post LT for all patients.

conclusionsReferral for LT for IEMs has increased over the last 33 years, with the most referrals in the last 10 years. Early LT has resulted in improved clinical outcomes and patient survival.

Indexed as

clinical outcomeinborn errors of metabolismliver transplantationmetabolic management pre‐ and post‐liver transplantationorganic acidemiasurvivalurea cycle disorder

Identifiers

PMID34258144
PMCPMC8260479
OpenAlexW3157570865

What OpenQuestion holds

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LicenceCC BY
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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.