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2. Efficacy and safety of empagliflozin in glycogen storage disease type Ib: Data from an international questionnaire

Author

Grünert, Sarah C., primary, Derks, Terry G.J., additional, Adrian, Katarina, additional, Al-Thihli, Khalid, additional, Ballhausen, Diana, additional, Bidiuk, Joanna, additional, Bordugo, Andrea, additional, Boyer, Monica, additional, Bratkovic, Drago, additional, Brunner-Krainz, Michaela, additional, Burlina, Alberto, additional, Chakrapani, Anupam, additional, Corpeleijn, Willemijn, additional, Cozens, Alison, additional, Dawson, Charlotte, additional, Dhamko, Helena, additional, Milosevic, Maja Djordjevic, additional, Eiroa, Hernan, additional, Finezilber, Yael, additional, Moura de Souza, Carolina Fischinger, additional, Garcia-Jiménez, Maria Concepción, additional, Gasperini, Serena, additional, Haas, Dorothea, additional, Häberle, Johannes, additional, Halligan, Rebecca, additional, Fung, Law Hiu, additional, Hörbe-Blindt, Alexandra, additional, Horka, Laura Maria, additional, Huemer, Martina, additional, Uçar, Sema Kalkan, additional, Kecman, Bozica, additional, Kilavuz, Sebile, additional, Kriván, Gergely, additional, Lindner, Martin, additional, Lüsebrink, Natalia, additional, Makrilakis, Konstantinos, additional, Mei-Kwun Kwok, Anne, additional, Maier, Esther M., additional, Maiorana, Arianna, additional, McCandless, Shawn E., additional, Mitchell, John James, additional, Mizumoto, Hiroshi, additional, Mundy, Helen, additional, Ochoa, Carlos, additional, Pierce, Kathryn, additional, Fraile, Pilar Quijada, additional, Regier, Debra, additional, Rossi, Alessandro, additional, Santer, René, additional, Schuman, Hester C., additional, Sobieraj, Piotr, additional, Spenger, Johannes, additional, Spiegel, Ronen, additional, Stepien, Karolina M., additional, Tal, Galit, additional, Tanšek, Mojca Zerjav, additional, Torkar, Ana Drole, additional, Tchan, Michel, additional, Thyagu, Santhosh, additional, Schrier Vergano, Samantha A., additional, Vucko, Erika, additional, Weinhold, Natalie, additional, Zsidegh, Petra, additional, and Wortmann, Saskia B., additional

Published
2022
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3. Efficacy and safety of empagliflozin in glycogen storage disease type Ib: Data from an international questionnaire

Author

Grünert, Sarah C; https://orcid.org/0000-0001-5986-0468, Derks, Terry G J, Adrian, Katarina, et al, Häberle, Johannes; https://orcid.org/0000-0003-0635-091X, Horka, Laura Maria, Huemer, Martina; https://orcid.org/0000-0002-0590-678X, Grünert, Sarah C; https://orcid.org/0000-0001-5986-0468, Derks, Terry G J, Adrian, Katarina, et al, Häberle, Johannes; https://orcid.org/0000-0003-0635-091X, Horka, Laura Maria, and Huemer, Martina; https://orcid.org/0000-0002-0590-678X

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

Published
2022

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