Hormone research in paediatrics · 2026
Glucagon-Like Peptide-1 Receptor Agonists in the Management of Mitochondrial Diabetes in an Adolescent: A Case Report.
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Hayes Eadaoin, Nillsen Ann, Rowe Peter W, Siafarikas Aris, Abraham Mary B
Аннотация
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.
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.