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

Humans Female Child Male Starch Adolescent Child, Preschool Adult Young Adult Middle Aged Double-Blind Method Glycogen Storage Disease / diet therapy Cross-Over Studies Delayed-Action Preparations

Journal

Orphanet journal of rare diseases
ISSN: 1750-1172
Titre abrégé: Orphanet J Rare Dis
Pays: England
ID NLM: 101266602

Informations de publication

Date de publication:
09 Jul 2024
Historique:
received: 18 12 2023
accepted: 01 07 2024
medline: 10 7 2024
pubmed: 10 7 2024
entrez: 9 7 2024
Statut: epublish

Résumé

Hypoglycaemia is the primary manifestation of all the hepatic types of glycogen storage disease (GSD). In 2008, Glycosade A 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. Sixty-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. The 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.

Sections du résumé

BACKGROUND BACKGROUND
Hypoglycaemia is the primary manifestation of all the hepatic types of glycogen storage disease (GSD). In 2008, Glycosade
METHODS METHODS
A 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.
RESULTS RESULTS
Sixty-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.
CONCLUSION CONCLUSIONS
The 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.

Identifiants

DOI: 10.1186/s13023-024-03274-y PMID: 38982397
pubmed: 38982397
doi: 10.1186/s13023-024-03274-y
pii: 10.1186/s13023-024-03274-y
doi:

Substances chimiques

Starch 9005-25-8
Delayed-Action Preparations 0

Types de publication

Journal Article Randomized Controlled Trial

Langues

eng

Sous-ensembles de citation

IM

Pagination

258

Subventions

Organisme : Vitaflo
ID : Vitaflo

Informations de copyright

© 2024. The Author(s).

Références

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Auteurs

Weinstein DA (W)

Glycogen Storage Disease Program, Connecticut Childrens Medical Center, Hartford, USA. weinsteingsd@gmail.com.
School of Medicine, Department of Pediatrics, University of Connecticut, Farmington, CT, USA. weinsteingsd@gmail.com.
ORCID: 0000-0002-1341-9367

Jackson Rj (J)

Liverpool Clinical Trials Centre, University of LiverpoolUK, Liverpool, UK.

Brennan Ea (B)

Vitaflo International Ltd, 182 Sefton Street, Liverpool, UK.

Williams M (W)

Glycogen Storage Disease Program, Connecticut Childrens Medical Center, Hartford, USA.

Davison Je (D)

Metabolic Medicine, Great Ormond Street Hospital, London, UK.

de Boer F (B)

Division of Metabolic Diseases, Beatrix Children's Hospital, University Medical Center Groningen, Groningen, The Netherlands.

Derks Tgj (D)

Division of Metabolic Diseases, Beatrix Children's Hospital, University Medical Center Groningen, Groningen, The Netherlands.

Ellerton C (E)

Charles Dent Metabolic Unit, National Hospital for Neurology and Neurosurgery, Queen Square, London, UK.

Faragher B (F)

Liverpool School of Tropical Medicine, Pembroke Place, Liverpool, UK.

Gribben J (G)

Evelina London Childrens Hospital, Westminster Bridge Road, London, UK.

Labrune P (L)

Centre de Référence des Maladies héréditaires du Métabolisme Hépatique, APHP, Hôpitaux Universitaires Paris-Saclay, Hôpital Antoine Béclère, Clamart, and Paris-Saclay University, Paris, France.

McKittrick Km (M)

Vitaflo International Ltd, 182 Sefton Street, Liverpool, UK.

Murphy E (M)

Charles Dent Metabolic Unit, National Hospital for Neurology and Neurosurgery, Queen Square, London, UK.

Ross Km (R)

Glycogen Storage Disease Program, Connecticut Childrens Medical Center, Hartford, USA.

Steuerwald U (S)

National Hospital of the Faroe Islands, Medical Center, Tórshavn, Faroe Islands.

Voillot C (V)

Centre de Référence des Maladies héréditaires du Métabolisme Hépatique, APHP, Hôpitaux Universitaires Paris-Saclay, Hôpital Antoine Béclère, Clamart, and Paris-Saclay University, Paris, France.

Woodward Ajm (W)

Evelina London Childrens Hospital, Westminster Bridge Road, London, UK.

Mundy Hr (M)

Evelina London Childrens Hospital, Westminster Bridge Road, London, UK.

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Classifications MeSH

questionsmedicales.fr › Eucaryotes › Animaux › Chordés › Vertébrés › Mammifères › Eutheria › Primates › Haplorhini › Catarrhini › Hominidae › Humains › Short and long-term acceptability and efficacy...
questionsmedicales.fr › Personnes › Tranches d'âge › Enfant › Short and long-term acceptability and efficacy...
questionsmedicales.fr › Glucides › Polyosides › Glucanes › Amidon › Short and long-term acceptability and efficacy...
questionsmedicales.fr › Personnes › Tranches d'âge › Adolescent › Short and long-term acceptability and efficacy...
questionsmedicales.fr › Personnes › Tranches d'âge › Enfant › Enfant d'âge préscolaire › Short and long-term acceptability and efficacy...
questionsmedicales.fr › Personnes › Tranches d'âge › Adulte › Short and long-term acceptability and efficacy...
questionsmedicales.fr › Personnes › Tranches d'âge › Adulte › Jeune adulte › Short and long-term acceptability and efficacy...
questionsmedicales.fr › Personnes › Tranches d'âge › Adulte › Adulte d'âge moyen › Short and long-term acceptability and efficacy...
questionsmedicales.fr › Environnement et santé publique › Santé publique › Méthodes épidémiologiques › Méthodologie en recherche épidémiologique › Méthode en double aveugle › Short and long-term acceptability and efficacy...
questionsmedicales.fr › Maladies métaboliques et nutritionnelles › Maladies métaboliques › Erreurs innées du métabolisme › Erreurs innées du métabolisme glucidique › Glycogénose › Short and long-term acceptability and efficacy...
questionsmedicales.fr › Environnement et santé publique › Santé publique › Méthodes épidémiologiques › Méthodologie en recherche épidémiologique › Études croisées › Short and long-term acceptability and efficacy...
questionsmedicales.fr › Thérapeutique › Traitement médicamenteux › Systèmes de délivrance de médicaments › Préparations à action retardée › Short and long-term acceptability and efficacy...