Evidence map›Paper›PMID 26053138›Full record

ArticlePediatric research2015

Effects of temporary low-dose galactose supplements in children aged 5-12 y with classical galactosemia: a pilot study.

Ina Knerr, Karen Patricia Coss, Jürgen Kratzsch, Ellen Crushell, Anne Clark, Peter Doran, Yoon Shin, Henning Stöckmann, Pauline Mary Rudd, Eileen Treacy

Open access · bronzeAbstract read
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In one paragraph

Article in Pediatric research, 2015. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 19 papers, 2 of them syntheses that pooled it.

0numbers the graph read from it
0cells of the map it votes in
19citing papers in PubMed, 2 pooled it
3.0field-weighted citation impact, top 10% 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

19 citing papers in PubMed, 2 syntheses or guidelines pooled it, 31 citations in OpenAlex.

  1. Newborn screening for galactosaemia.The Cochrane database of systematic reviews · 2020
    Pooled it
  2. Pooled it
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  9. AbnormalJIMD reports · 2021
    Article
  10. Immunoglobulin G Glycosylation in Diseases.Experientia supplementum (2012) · 2021
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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

10 authors at 5 institutions in 3 countries.

Ina KnerrNational Centre for Inherited Metabolic Disorders, Temple Street Children's University Hospital, Dublin, Ireland.
Karen Patricia CossUniversity College Dublin, Clinical Research Centre, Mater Misericordiae University Hospital, Dublin, Ireland.
Jürgen KratzschInstitute of Laboratory Medicine, Clinical Chemistry and Molecular Diagnostics, University of Leipzig, Leipzig, Germany.
Ellen CrushellNational Centre for Inherited Metabolic Disorders, Temple Street Children's University Hospital, Dublin, Ireland.
Anne ClarkNational Centre for Inherited Metabolic Disorders, Temple Street Children's University Hospital, Dublin, Ireland.
Peter DoranUniversity College Dublin, Clinical Research Centre, Mater Misericordiae University Hospital, Dublin, Ireland.
Yoon ShinMolecular Genetics and Metabolism Laboratory, Munich, Germany.
Henning StöckmannThe National Institute for Bioprocessing Research and Training, Blackrock, Co., Dublin, Ireland.
Pauline Mary RuddThe National Institute for Bioprocessing Research and Training, Blackrock, Co., Dublin, Ireland.
Eileen TreacyNational Centre for Inherited Metabolic Disorders, Temple Street Children's University Hospital, Dublin, Ireland.
Temple Street Children's University Hospital · IENational Institute for Bioprocessing Research and Training · IEUniversity College Dublin · IELeipzig University · DETemple University · US

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

backgroundClassical galactosemia is caused by severe galactose-1-phosphate uridyltransferase deficiency. Despite life-long galactose-restriction, many patients experience long-term complications. Intoxication by galactose and its metabolites as well as over-restriction of galactose may contribute to the pathophysiology. We provided temporary low-dose galactose supplements to patients. We assessed tolerance and potential beneficial effects with clinical monitoring and measurement of biochemical, endocrine, and IgG N-glycosylation profiles.

methodsWe enrolled 26 patients (8.6 ± 1.9 y). Thirteen were provided with 300 mg of galactose/day followed by 500 mg for 2 wk each (13 patient controls).

resultsWe observed no clinical changes with the intervention. Temporary mild increase in galactose-1-phosphate occurred, but renal, liver, and bone biochemistry remained normal. Patients in the supplementation group had slightly higher leptin levels at the end of the study than controls. We identified six individuals as "responders" with an improved glycosylation pattern (decreased G0/G2 ratio, P < 0.05). There was a negative relationship between G0/G2 ratio and leptin receptor sOb-R in the supplementation group (P < 0.05).

conclusionTemporary low-dose galactose supplementation in children over 5 y is well tolerated in the clinical setting. It leads to changes in glycosylation in "responders". We consider IgG N-glycan monitoring to be useful for determining individual optimum galactose intake.

Indexed as

Dietary SupplementsBone and BonesChildChild, PreschoolCohort StudiesEndocrine SystemFemaleGalactoseGalactosemiasGlycosylationHEK293 CellsHomozygoteHumansImmunoglobulin GKidneyLactoseGalactoseImmunoglobulin GLactoseLeptinReceptors, Leptin

Identifiers

PMID26053138
OpenAlexW618343152

What OpenQuestion holds

Textmetadata
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.