Sodium-Dependent Glucose Transporter Type 2 Inhibitors as a Breakthrough in Neutropenia and Neutrophil Dysfunction Management in Patients with Glycogen Storage Disease Type Ib
Andrey N. Surkov,
Leyla S. Namazova-Baranova,
Anna L. Arakelyan,
Evgeny E. Bessonov,
Natalia V. Zhurkova https://doi.org/10.15690/vsp.v23i3.2761
Abstract
Glycogen storage disease type Ib (GSD Ib) is a rare and extremely severe disease included in the group of hereditary carbohydrate metabolism disorders. The condition is caused by pathogenic variants in the SLC37A4 gene leading to glucose metabolic disorder in the liver and kidneys, and as a result to severe organomegaly, hypoglycemia, and metabolic decompensation. Moreover, neutropenia and neutrophil dysfunction are noted in patients with GSD Ib. The use of granulocyte colony stimulating factor only increases the number of dysfunctional neutrophils without affecting their functional activity, what determines the inefficacy of such treatment. In recent years, the mechanism of neutropenia in GSD Ib has been clarified, so new therapeutic agents for its relief have been created. This article presents the overview of data on the successful results of renal sodium-glucose cotransporter type 2 inhibitors (gliflozins) usage in patients with GSD Ib.
Keywords
glycogen storage disease type Ib,
neutropenia,
granulocyte colony-stimulating factor,
sodium-glucose cotransporter type 2 inhibitors,
gliflozins
Supporting agencies: Not declared
About the Authors
Andrey N. Surkov
Research Institute of Pediatrics and Children’s Health in Petrovsky National Research Centre of Surgery; Pirogov Russian National Research Medical University
Russian Federation
Disclosure of interest:
Russian Federation
Moscow
Disclosure of interest:
lecturing for pharmaceutical companies PTC Therapeutics, Swixx BioPharma, Sanofi Aventis Group, AstraZeneca, OTCPharm, Opella Healthcare, Leovit
Leyla S. Namazova-Baranova
Research Institute of Pediatrics and Children’s Health in Petrovsky National Research Centre of Surgery; Pirogov Russian National Research Medical University
Russian Federation
Disclosure of interest:
Russian Federation
Moscow
Disclosure of interest:
receiving research grants and fees for scientific counseling and lecturing from pharmaceutical companies MSD Pharmaceuticals, FORT, Shire Biothech Rus LLC, Pfizer Innovations, Sanofi Aventis Group, AbbVie, Pierre Fabre
Anna L. Arakelyan
Research Institute of Pediatrics and Children’s Health in Petrovsky National Research Centre of Surgery
Russian Federation
Disclosure of interest:
Russian Federation
Moscow
Disclosure of interest:
Other authors confirmed the absence of a reportable conflict of interests
Evgeny E. Bessonov
Research Institute of Pediatrics and Children’s Health in Petrovsky National Research Centre of Surgery
Russian Federation
Disclosure of interest:
Russian Federation
Moscow
Disclosure of interest:
Other authors confirmed the absence of a reportable conflict of interests
Natalia V. Zhurkova
Research Institute of Pediatrics and Children’s Health in Petrovsky National Research Centre of Surgery
Russian Federation
Disclosure of interest:
Russian Federation
Moscow
Disclosure of interest:
lecturing for pharmaceutical companies Takeda, Sanofi Aventis Group, AstraZeneca, Chiesi pharmaceuticals, Nutricia
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For citations:
Surkov A.N., Namazova-Baranova L.S., Arakelyan A.L., Bessonov E.E., Zhurkova N.V. Sodium-Dependent Glucose Transporter Type 2 Inhibitors as a Breakthrough in Neutropenia and Neutrophil Dysfunction Management in Patients with Glycogen Storage Disease Type Ib. Current Pediatrics. 2024;23(3):162-167. (In Russ.) https://doi.org/10.15690/vsp.v23i3.2761
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