The overall benefits of empagliflozin treatment in adult siblings with glycogen storage disease type Ib: one year experience

Abstract

Introduction: Recently published case reports suggest the benefit of empagliflozin use in subjects with glycogen storage disease Ib (GSD Ib). Methods: We present the clinical and laboratory data of 2 adult brothers with GSD Ib treated with empagliflozin for 12 months. Results: There was no severe infection during administration of empagliflozin. The improvement of clinical symptoms of inflammatory bowel disease and arthritis along with reduction in serum CRP levels and urinary albumin excretion was noted. Neutrophil count increased, allowing for reduction or temporary withdrawal of G-CSF treatment. Conclusions: Empagliflozin may be a new safe treatment in GSD Ib patients with an advanced stage of the disease.