Glucagon-Like Peptide-1 Receptor Agonists 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.

CASE PRESENTATION: A 16-year-old adolescent girl with a genetically confirmed diagnosis of MELAS due to an m.3243A>G variant presented with clinical and biochemical features of diabetes. She was successfully transitioned from subcutaneous insulin therapy to weekly GLP-1RA monotherapy shortly after diagnosis of diabetes. Treatment with 0.25 mg semaglutide SC weekly led to a reduction in HbA1c from 13.7% (126 mmol/mol) to 5.6% (38 mmol/mol) over a 7-month period. Insulin was completely ceased within 5 days of starting semaglutide. CGM data from a 11 months follow-up period are presented, which demonstrate CGM data meeting glycaemic targets.

CONCLUSION

Low-dose semaglutide 0.25 mg sc weekly was efficacious and well tolerated without lactic acidosis. Use of GLP-1RA could be considered a novel and effective treatment in the pharmacological management of paediatric mitochondrial diabetes.