The potential of dietary treatment in patients with glycogen storage disease type IV.
dietary intervention
glycogen storage disease
glycogen storage disease type IV
inherited metabolic disease
liver transplantation
Journal
Journal of inherited metabolic disease
ISSN: 1573-2665
Titre abrégé: J Inherit Metab Dis
Pays: United States
ID NLM: 7910918
Informations de publication
Date de publication:
05 2021
05 2021
Historique:
revised:
30
11
2020
received:
21
10
2020
accepted:
07
12
2020
pubmed:
18
12
2020
medline:
28
12
2021
entrez:
17
12
2020
Statut:
ppublish
Résumé
There is paucity of literature on dietary treatment in glycogen storage disease (GSD) type IV and formal guidelines are not available. Traditionally, liver transplantation was considered the only treatment option for GSD IV. In light of the success of dietary treatment for the other hepatic forms of GSD, we have initiated this observational study to assess the outcomes of medical diets, which limit the accumulation of glycogen. Clinical, dietary, laboratory, and imaging data for 15 GSD IV patients from three centres are presented. Medical diets may have the potential to delay or prevent liver transplantation, improve growth and normalize serum aminotransferases. Individual care plans aim to avoid both hyperglycaemia, hypoglycaemia and/or hyperketosis, to minimize glycogen accumulation and catabolism, respectively. Multidisciplinary monitoring includes balancing between traditional markers of metabolic control (ie, growth, liver size, serum aminotransferases, glucose homeostasis, lactate, and ketones), liver function (ie, synthesis, bile flow and detoxification of protein), and symptoms and signs of portal hypertension.
Identifiants
pubmed: 33332610
doi: 10.1002/jimd.12339
pmc: PMC8246821
doi:
Substances chimiques
Biomarkers
0
Glycogen
9005-79-2
Types de publication
Journal Article
Multicenter Study
Observational Study
Research Support, Non-U.S. Gov't
Langues
eng
Sous-ensembles de citation
IM
Pagination
693-704Informations de copyright
© 2020 The Authors. Journal of Inherited Metabolic Disease published by John Wiley & Sons Ltd on behalf of SSIEM.
Références
Mol Genet Metab Rep. 2020 May 18;24:100601
pubmed: 32455116
J Hepatol. 1995 Jun;22(6):696-9
pubmed: 7560864
J Inherit Metab Dis. 2021 May;44(3):693-704
pubmed: 33332610
J Inherit Metab Dis. 2020 Mar;43(2):279-289
pubmed: 31587328
J Pediatr Gastroenterol Nutr. 2019 Jun;68(6):763-767
pubmed: 30908382
Pediatr Transplant. 2008 Mar;12(2):137-45
pubmed: 18307661
Am J Med Genet A. 2005 Dec 1;139A(2):118-22
pubmed: 16278887
Mol Genet Metab. 2013 Jun;109(2):179-82
pubmed: 23578772
Mol Ther. 2018 Mar 7;26(3):814-821
pubmed: 29428299
Hepatology. 1988 Mar-Apr;8(2):302-6
pubmed: 3162725
Ann Saudi Med. 2014 Jul-Aug;34(4):354-7
pubmed: 25811211
Am J Transplant. 2012 Nov;12(11):2986-96
pubmed: 22882699
Genet Med. 2019 Dec;21(12):2686-2694
pubmed: 31263214
JIMD Rep. 2019;45:99-104
pubmed: 30569318
J Am Diet Assoc. 1993 Dec;93(12):1423-30
pubmed: 8245377
J Inherit Metab Dis. 2010 Dec;33 Suppl 3:S83-90
pubmed: 20058079
Am J Med Genet A. 2006 Apr 15;140(8):878-82
pubmed: 16528737
Pediatr Clin North Am. 2018 Apr;65(2):247-265
pubmed: 29502912
Hum Gene Ther. 2017 Mar;28(3):286-294
pubmed: 27832700
Pediatr Gastroenterol Hepatol Nutr. 2018 Oct;21(4):365-368
pubmed: 30345254
Mol Ther. 2018 Jul 5;26(7):1771-1782
pubmed: 29784585
J Inherit Metab Dis. 1996;19(1):51-8
pubmed: 8830177
Mol Genet Metab Rep. 2016 Oct 04;9:31-33
pubmed: 27747161
Neurology. 2004 Sep 28;63(6):1053-8
pubmed: 15452297
Mamm Genome. 2004 Jul;15(7):570-7
pubmed: 15366377
J Inherit Metab Dis. 2016 Sep;39(5):697-704
pubmed: 27106217
Hum Mol Genet. 2011 Nov 15;20(22):4430-9
pubmed: 21856731
Curr Mol Med. 2002 Mar;2(2):177-88
pubmed: 11949934
Eur J Pediatr. 1986 Aug;145(3):179-81
pubmed: 3464425
Eur J Pediatr. 1993;152 Suppl 1:S71-6
pubmed: 8319729
Nat Med. 2018 Oct;24(10):1519-1525
pubmed: 30297904
Arch Med Sci. 2018 Jan;14(1):237-247
pubmed: 29379554
Lab Invest. 1956 Jan-Feb;5(1):11-20
pubmed: 13279125
Adv Nutr. 2020 Mar 1;11(2):439-446
pubmed: 31665208
Pediatr Res. 1992 Dec;32(6):719-25
pubmed: 1337588