Transition from Leigh syndrome to MELAS syndrome in a patient with heteroplasmic MT-ND3 m.10158T>C

Brain Dev. 2019 Oct;41(9):803-807. doi: 10.1016/j.braindev.2019.05.006. Epub 2019 Jun 6.

Abstract

An m.10158T>C mutation in MT-ND3, encoding a subunit of respiratory complex I, causes early-onset Leigh syndrome (LS), mitochondrial encephalomyopathy with lactic acid and stroke-like episodes (MELAS) syndrome, and LS and MELAS overlapping syndrome, presumably dependent on the ratio of heteroplasmy. Herein, we report a 4-year-old girl with heteroplasmic m.10158T>C mutation, showing an evolving age-dependent phenotype from LS to MELAS syndromes. She showed mild developmental delay during infancy, which was associated with magnetic resonance imaging lesions in the brain stem and basal ganglia. At the age of 4 years, she developed rapid neurological deterioration and intractable seizures, which was associated with recurrent multiple cerebral lesions as well as basal ganglia lesions. Her cerebral lesions were located predominantly in white matter and appeared at multiple areas simultaneously, unique characteristics that are distinct from typical MELAS. Two patients with LS-MELAS overlapping syndrome with m.10158T>C have been previously reported, however, this is the first patient with m.10158T>C showing significant age-dependent changes in clinical features and neuro-images, implying an age-dependent role of complex I in the developing brain.

Keywords: Mitochondrial disease; Neuroimaging; Respiratory complex I.

Publication types

  • Case Reports

MeSH terms

  • Brain / diagnostic imaging
  • Brain / growth & development
  • Child, Preschool
  • Disease Progression
  • Electron Transport Complex I / genetics*
  • Female
  • Humans
  • Leigh Disease / diagnostic imaging
  • Leigh Disease / genetics*
  • Leigh Disease / physiopathology
  • MELAS Syndrome / diagnostic imaging
  • MELAS Syndrome / genetics*
  • MELAS Syndrome / physiopathology
  • Mutation*
  • Phenotype

Substances

  • Electron Transport Complex I
  • MT-ND3 protein, human