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J Inherit Metab Dis. 2008 Dec;31 Suppl 3:505-9. doi: 10.1007/s10545-007-0774-9. Epub 2008 Jan 5.

Adalimumab for the treatment of Crohn-like colitis and enteritis in glycogen storage disease type Ib.

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1
Division of Pediatric Gastroenterology, Department of Pediatrics, University of Florida College of Medicine, Gainesville, Florida, USA.

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.

PMID:
18172743
DOI:
10.1007/s10545-007-0774-9
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