Clinical Remission of Severe Crohn’s Disease with Empagliflozin Monotherapy in a Pediatric Patient with Glycogen Storage Disease Type 1b

Author:

Collen Lauren V.1,Newburger Peter E.23,Snapper Scott B.1

Affiliation:

1. Division of Gastroenterology, Hepatology, and Nutrition, Boston Children’s Hospital, Harvard Medical School, Boston, MA

2. Departments of Pediatrics and Molecular, Cell, and Cancer Biology, UMass Chan Medical School, Worcester, MA

3. Dana-Farber/Boston Children’s Hospital Cancer and Blood Disorders Center, Harvard Medical School, Boston, MA.

Abstract

Glycogen storage disease type 1b (GSD1b) is associated with inflammatory bowel disease and congenital neutropenia. Neutropenia in GSD1b is caused by the accumulation of 1,5-anhydroglucitol-6-phosphate. Empagliflozin is an antidiabetic drug that promotes renal excretion of this metabolite. We report on a patient with refractory GSD1b-associated inflammatory bowel disease who is in clinical remission on empagliflozin monotherapy.

Publisher

Wiley

Subject

General Earth and Planetary Sciences,General Environmental Science

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