Adalimumab for the treatment of Crohn-like colitis and enteritis in glycogen storage disease type Ib.
J Inherit Metab Dis
; 31 Suppl 3: 505-9, 2008 Dec.
Article
in En
| MEDLINE
| ID: mdl-18172743
ABSTRACT
Glycogen storage disease (GSD) type Ib is a congenital disorder of glycogen metabolism that is associated with neutropenia, neutrophil dysfunction, and an inflammatory bowel disease that mimics a Crohn phenotype. Gastrointestinal inflammation in GSD Ib has been successfully treated with 5-aminosalicylic acid and granulocyte colony-stimulating factor (G-CSF). However, therapeutic options for patients not responding to traditional therapies have been limited owing to untoward effects of glucocorticoids and immunomodulators in this metabolic disorder. Adalimumab is a monoclonal antibody targeting tumour necrosis factor-α that has shown promise for the treatment of patients with Crohn disease. Due to the limited options for treating GSD-associated inflammatory bowel disease, use of adalimumab was attempted in a case unresponsive to aminosalicylate, G-CSF, and antibiotic therapy. Significant clinical and histological improvement was observed in our patient, and the medication was well tolerated.
Full text:
1
Collection:
01-internacional
Database:
MEDLINE
Language:
En
Journal:
J Inherit Metab Dis
Year:
2008
Document type:
Article
Affiliation country:
United States