Glucagon-like peptide-1 receptor agonists (GLP1RA) in the management of mitochondrial diabetes in an adolescent: A Case Report.

INTRODUCTION: Mitochondrial diabetes is rare and typically treated with dietary intervention and oral hypoglycaemic agents. Most require insulin. Novel treatments are available but are not well-characterised. PRESENTATION: A 16-year-old adolescent girl with a genetically confirmed diagnosis of MELAS due to a m.3243A>G presented with clinical and biochemical features of diabetes. She was successfully transitioned from subcutaneous insulin therapy to weekly GLP1RA monotherapy shortly after diagnosis of diabetes. Treatment with 0.25mg semaglutide SC weekly led to a reduction in HbA1c from 13.7% (126mmol/mol) to 5.6% (38mmol/mol) over a 7-month period. Insulin was completely ceased within 5 days of starting semaglutide. CGM data from a 5 month follow up period are presented, which demonstrate CGM data meeting glycaemic targets. CONCLUSION: Low-dose semaglutide 0.25mg sc weekly was efficacious and well tolerated without lactic acidosis. Use of GLP1RA could be considered a novel and effective treatment in the pharmacological management of paediatric mitochondrial diabetes.