Infectious and digestive complications in glycogen storage disease type Ib: Study of a French cohort.

ANC, Absolute Neutrophil Count Anti-inflammatory solutions CD, Crohn's disease CRP, C-reactive protein EEN, Exclusive Enteral Nutrition EN, Enteral Nutrition ENT, Ear, Nose and Throat ESR, erythrocyte sedimentation rate G-CSF, Granulocyte colony-stimulating factor G6PT, glucose-6-phosphate translocase GSD1, Glycogen storage disease type I Glycogen storage disease type 1B Harvey Bradshaw score IBD, Inflammatory Bowel Disease Inflammatory bowel disease Neutropenia PEN, Partial Enteral Nutrition SD, Standard Deviation

Journal

Molecular genetics and metabolism reports
ISSN: 2214-4269
Titre abrégé: Mol Genet Metab Rep
Pays: United States
ID NLM: 101624422

Informations de publication

Date de publication:
Jun 2020
Historique:
received: 21 03 2020
accepted: 22 03 2020
entrez: 18 4 2020
pubmed: 18 4 2020
medline: 18 4 2020
Statut: epublish

Résumé

Glycogenosis type Ib (GSD1B) causes not only hypoglycemia but also infections and "Crohn's disease like" inflammatory bowel disease (IBD) that can significantly impair patient's quality of life. We retrospectively evaluated infectious and digestive complications in 9 French patients (3 girls, 6 boys) diagnosed at 0.8 years on average, with a mean follow-up of 19.1 years. Infections occurred earlier than IBD, at mean ages of 1.7 and 3.8 years, respectively. The number of acute hospitalizations was 0.7/year due to infectious (0.4/year) or digestive symptoms (0.4/year). Clinical presentations allowed separating patients into mild (

Identifiants

pubmed: 32300528
doi: 10.1016/j.ymgmr.2020.100581
pii: S2214-4269(20)30027-6
pii: 100581
pmc: PMC7152669
doi:

Types de publication

Journal Article

Langues

eng

Pagination

100581

Informations de copyright

© 2020 The Authors.

Déclaration de conflit d'intérêts

FR has received speaker fees from: Shering-Plough, Nestlé, MeadJohnson, Ferring, MSD, Johnson & Johnson, Centocor, AbbVie; serves as a board member for: SAC:DEVELOP (Johnson & Johnson), and has been invited to MSD France, Nestlé Nutrition Institute, Nestlé Health Science, Danone, MeadJohnson; TAKEDA, CELLGENE, BIOGENE, ARKOPHARMA. The other authors have no conflicts of interest.

Références

Eur J Pediatr. 2002 Oct;161 Suppl 1:S88-92
pubmed: 12373579
Ann Nutr Metab. 2016;68 Suppl 1:33-41
pubmed: 27355913
J Crohns Colitis. 2014 Oct;8(10):1179-207
pubmed: 24909831
World J Gastroenterol. 2016 Jan 21;22(3):1045-66
pubmed: 26811646
Lancet. 1980 Mar 8;1(8167):514
pubmed: 6102236
J Pediatric Infect Dis Soc. 2018 May 9;7(suppl_1):S18-S24
pubmed: 29746679
J Inherit Metab Dis. 2008 Dec;31 Suppl 3:505-9
pubmed: 18172743
Chest. 1992 Jun;101(6):1644-55
pubmed: 1303622
Curr Opin Hematol. 2019 Jan;26(1):16-21
pubmed: 30451720
Acta Paediatr. 2003 Dec;92(12):1415-21
pubmed: 14971792
Curr Opin Hematol. 2010 Jan;17(1):36-42
pubmed: 19741523
Eur J Pediatr. 2002 Oct;161 Suppl 1:S120-3
pubmed: 12373585
Dig Dis. 2013;31(3-4):321-7
pubmed: 24246982
Pediatr Blood Cancer. 2010 Dec 15;55(7):1410-3
pubmed: 20830779
Aliment Pharmacol Ther. 2000 Mar;14(3):281-9
pubmed: 10735920
Inflamm Bowel Dis. 2001 May;7(2):128-32
pubmed: 11383585
Blood. 2008 Jun 15;111(12):5704-11
pubmed: 18420828
J Pediatr. 2000 Aug;137(2):187-91
pubmed: 10931410
J Crohns Colitis. 2019 Jul 25;13(7):846-855
pubmed: 30541015
World J Gastroenterol. 2015 Jun 14;21(22):6809-16
pubmed: 26078556
Genet Med. 2014 Nov;16(11):e1
pubmed: 25356975
Blood. 2014 May 1;123(18):2843-53
pubmed: 24565827
Eur J Pediatr. 2002 Oct;161 Suppl 1:S83-7
pubmed: 12373578
Orphanet J Rare Dis. 2011 May 20;6:27
pubmed: 21599942
Eur J Pediatr. 2002 Oct;161 Suppl 1:S20-34
pubmed: 12373567
Nat Rev Endocrinol. 2010 Dec;6(12):676-88
pubmed: 20975743
Proc Natl Acad Sci U S A. 2019 Jan 22;116(4):1241-1250
pubmed: 30626647

Auteurs

Camille Wicker (C)

Reference Center for Inherited Metabolic Diseases, Necker Hospital, APHP, Filière G2 M, MetabERN, Paris, France.

Célina Roda (C)

Paris University, CRESS, HERA (Health Environmental Risk Assessment) team, INSERM, INRA, F-75004 Paris, France.

Ariane Perry (A)

Reference Center for Inherited Metabolic Diseases, Antoine Béclère Hospital, APHP, Filière G2M, MetabERN, Clamart, France.
Paris Sud University, Paris Saclay, and INSERM, U 1995, France.

Jean Baptiste Arnoux (JB)

Reference Center for Inherited Metabolic Diseases, Necker Hospital, APHP, Filière G2 M, MetabERN, Paris, France.

Anais Brassier (A)

Reference Center for Inherited Metabolic Diseases, Necker Hospital, APHP, Filière G2 M, MetabERN, Paris, France.

Martin Castelle (M)

Hematology, Necker Hospital, APHP, Paris Descartes University, Paris, France.

Aude Servais (A)

Reference Center for Inherited Metabolic Diseases, Necker Hospital, APHP, Filière G2 M, MetabERN, Paris, France.

Jean Donadieu (J)

Hematology Department, Trousseau Hospital, APHP, Paris, France.

Juliette Bouchereau (J)

Reference Center for Inherited Metabolic Diseases, Necker Hospital, APHP, Filière G2 M, MetabERN, Paris, France.
Paris Descartes University- Sorbonne Paris Cité, Paris Faculty of Medecine, Paris, France.

Bénédicte Pigneur (B)

Paediatric Gastroentérology Department, Necker Hospital, APHP, Paris, France.

Philippe Labrune (P)

Reference Center for Inherited Metabolic Diseases, Antoine Béclère Hospital, APHP, Filière G2M, MetabERN, Clamart, France.
Paris Sud University, Paris Saclay, and INSERM, U 1995, France.

Frank M Ruemmele (FM)

Paediatric Gastroentérology Department, Necker Hospital, APHP, Paris, France.
Paris Descartes University- Sorbonne Paris Cité, Paris Faculty of Medecine, Paris, France.
Institut Imagine, INSERM U 1163, Paris, France.

Pascale de Lonlay (P)

Reference Center for Inherited Metabolic Diseases, Necker Hospital, APHP, Filière G2 M, MetabERN, Paris, France.
Paris Descartes University- Sorbonne Paris Cité, Paris Faculty of Medecine, Paris, France.
Institut Imagine, INSERM U 1163, Paris, France.
Institut Necker Enfants Malades, INSERM, Unit 1151, Paris, France.

Classifications MeSH