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Efficacy and safety of empagliflozin in glycogen storage disease type Ib: Data from an international questionnaire
- Source :
- Grünert, Sarah C; Derks, Terry G J; Adrian, Katarina; et al; Häberle, Johannes; Horka, Laura Maria; Huemer, Martina (2022). Efficacy and safety of empagliflozin in glycogen storage disease type Ib: Data from an international questionnaire. Genetics in Medicine, 24(8):1781-1788.
- Publication Year :
- 2022
-
Abstract
- Purpose: This paper aims to report collective information on safety and efficacy of empagliflozin drug repurposing in individuals with glycogen storage disease type Ib (GSD Ib). Methods: This is an international retrospective questionnaire study on the safety and efficacy of empagliflozin use for management of neutropenia/neutrophil dysfunction in patients with GSD Ib, conducted among the respective health care providers from 24 countries across the globe. Results: Clinical data from 112 individuals with GSD Ib were evaluated, representing a total of 94 treatment years. The median age at start of empagliflozin treatment was 10.5 years (range = 0-38 years). Empagliflozin showed positive effects on all neutrophil dysfunction-related symptoms, including oral and urogenital mucosal lesions, recurrent infections, skin abscesses, inflammatory bowel disease, and anemia. Before initiating empagliflozin, most patients with GSD Ib were on G-CSF (94/112; 84%). At the time of the survey, 49 of 89 (55%) patients previously treated with G-CSF had completely stopped G-CSF, and another 15 (17%) were able to reduce the dose. The most common adverse event during empagliflozin treatment was hypoglycemia, occurring in 18% of individuals. Conclusion: Empagliflozin has a favorable effect on neutropenia/neutrophil dysfunction-related symptoms and safety profile in individuals with GSD Ib. Keywords: GSD Ib; Glycogen storage disease type Ib; Neutropenia; SGLT2 inhibitors; SLC37A4
Details
- Database :
- OAIster
- Journal :
- Grünert, Sarah C; Derks, Terry G J; Adrian, Katarina; et al; Häberle, Johannes; Horka, Laura Maria; Huemer, Martina (2022). Efficacy and safety of empagliflozin in glycogen storage disease type Ib: Data from an international questionnaire. Genetics in Medicine, 24(8):1781-1788.
- Notes :
- application/pdf, info:doi/10.5167/uzh-226420, English, English
- Publication Type :
- Electronic Resource
- Accession number :
- edsoai.on1443049746
- Document Type :
- Electronic Resource