Evidence map›Paper›PMID 38982397›Full record

Trial reportOrphanet journal of rare diseases2024

Short and long-term acceptability and efficacy of extended-release cornstarch in the hepatic glycogen storage diseases: results from the Glyde study.

D A Weinstein, R J Jackson, E A Brennan, M Williams, J E Davison, F de Boer, Tgj Derks, C Ellerton, B Faragher, J Gribben and 8 more

Erratum issuedAbstract readRandomized Controlled Trial
PubMed Full text at PMC Publisher
In one paragraph

Trial report in Orphanet journal of rare diseases, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. An erratum has been issued. Cited by 11 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
11citing papers in PubMed, 1 pooled it
–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

11 citing papers in PubMed, 1 synthesis or guideline pooled it.

  1. Pooled it
  2. Article
  3. Observational
  4. Review
  5. Article
  6. Observational
  7. Review
  8. Review
  9. Article
  10. Review
  11. Article
4 · The record

Corrections and comments

5 · Who and what money

Authors and funding

18 authors.

D A WeinsteinGlycogen Storage Disease Program, Connecticut Childrens Medical Center, Hartford, USA. weinsteingsd@gmail.com.ORCID 0000-0002-1341-9367
R J JacksonLiverpool Clinical Trials Centre, University of LiverpoolUK, Liverpool, UK.
E A BrennanVitaflo International Ltd, 182 Sefton Street, Liverpool, UK.
M WilliamsGlycogen Storage Disease Program, Connecticut Childrens Medical Center, Hartford, USA.
J E DavisonMetabolic Medicine, Great Ormond Street Hospital, London, UK.
F de BoerDivision of Metabolic Diseases, Beatrix Children's Hospital, University Medical Center Groningen, Groningen, The Netherlands.
Tgj DerksDivision of Metabolic Diseases, Beatrix Children's Hospital, University Medical Center Groningen, Groningen, The Netherlands.
C EllertonCharles Dent Metabolic Unit, National Hospital for Neurology and Neurosurgery, Queen Square, London, UK.
B FaragherLiverpool School of Tropical Medicine, Pembroke Place, Liverpool, UK.
J GribbenEvelina London Childrens Hospital, Westminster Bridge Road, London, UK.
P LabruneCentre de Référence des Maladies héréditaires du Métabolisme Hépatique, APHP, Hôpitaux Universitaires Paris-Saclay, Hôpital Antoine Béclère, Clamart, and Paris-Saclay University, Paris, France.
K M McKittrickVitaflo International Ltd, 182 Sefton Street, Liverpool, UK.
E MurphyCharles Dent Metabolic Unit, National Hospital for Neurology and Neurosurgery, Queen Square, London, UK.
K M RossGlycogen Storage Disease Program, Connecticut Childrens Medical Center, Hartford, USA.
U SteuerwaldNational Hospital of the Faroe Islands, Medical Center, Tórshavn, Faroe Islands.
C VoillotCentre de Référence des Maladies héréditaires du Métabolisme Hépatique, APHP, Hôpitaux Universitaires Paris-Saclay, Hôpital Antoine Béclère, Clamart, and Paris-Saclay University, Paris, France.
Ajm WoodwardEvelina London Childrens Hospital, Westminster Bridge Road, London, UK.
H R MundyEvelina London Childrens Hospital, Westminster Bridge Road, London, UK.

Funding

Vitaflo Vitaflo
6 · The paper itself

Abstract

backgroundHypoglycaemia is the primary manifestation of all the hepatic types of glycogen storage disease (GSD). In 2008, Glycosade

methodsA randomised double-blind cross-over fasting study assessing the tolerance and efficacy of Glycosade compared with cornstarch was performed across disease types and ages. Participants and clinicians chose the product deemed superior, whilst still blinded. Participants were followed for 2 years to assess long-term metabolic control, growth, and quality of life.

resultsSixty-one participants (age 2-62 years; 59% female) were enrolled, and 58 participants completed the fasting studies (28 GSD I; 30 GSD III, VI, IX). Glycosade improved duration of fasting in GSD I and duration of fasting without ketosis in the ketotic forms. Chronic Glycosade use was chosen by 69% of participants. Those treated with Glycosade for the 2-year chronic phase used fewer doses of therapy while markers of metabolic control remained stable.

conclusionThe Glyde study is the first multi-centre international trial demonstrating the efficacy and tolerance of Glycosade in a large cohort of hepatic GSD patients across a diverse international population. The ability to use fewer doses of therapy per day and avoidance of overnight therapy may improve compliance, safety, and quality of life without sacrificing metabolic control.

Indexed as

StarchAdolescentAdultChildChild, PreschoolCross-Over StudiesDelayed-Action PreparationsDouble-Blind MethodFemaleGlycogen Storage DiseaseHumansMaleMiddle AgedYoung AdultDelayed-Action PreparationsStarch

Identifiers

PMID38982397
PMCPMC11232220

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Registered trials

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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.