A novel TUFM homozygous variant in a child with mitochondrial cardiomyopathy expands the phenotype of combined oxidative phosphorylation deficiency 4

J Hum Genet. 2019 Jun;64(6):589-595. doi: 10.1038/s10038-019-0592-6. Epub 2019 Mar 22.

Abstract

Translation of mitochondrial-specific DNA is required for proper mitochondrial function and energy production. For this purpose, an elaborate network of dedicated molecular machinery including initiation, elongation and termination factors exists. We describe a patient with an unusual phenotype and a novel homozygous missense variant in TUFM (c.344A>C; p.His115Pro), encoding mtDNA translation elongating factor Tu (EFTu). To date, only four patients have been reported with bi-allelic mutations in TUFM, leading to combined oxidative phosphorylation deficiency 4 (COXPD4) characterized by severe early-onset lactic acidosis and progressive fatal infantile encephalopathy. The patient presented here expands the phenotypic features of TUFM-related disease, exhibiting lactic acidosis and dilated cardiomyopathy without progressive encephalopathy. This warrants the inclusion of TUFM in differential diagnosis of metabolic cardiomyopathy. Cases that further refine genotype-phenotype associations and characterize the molecular basis of mitochondrial disorders allow clinicians to predict disease prognosis, greatly impacting patient care, as well as provide families with reproductive planning options.

Publication types

  • Case Reports

MeSH terms

  • Acidosis, Lactic / genetics*
  • Acidosis, Lactic / physiopathology
  • Amino Acid Sequence / genetics
  • Cardiomyopathies / genetics*
  • Cardiomyopathies / physiopathology
  • Consanguinity
  • DNA, Mitochondrial / genetics
  • Exome Sequencing
  • Female
  • Homozygote
  • Humans
  • Infant
  • Male
  • Metabolism, Inborn Errors / genetics*
  • Metabolism, Inborn Errors / physiopathology
  • Mitochondria / genetics*
  • Mitochondrial Diseases / genetics*
  • Mitochondrial Diseases / physiopathology
  • Mitochondrial Proteins / genetics*
  • Mutation
  • Oxidative Phosphorylation
  • Peptide Elongation Factor Tu / genetics*

Substances

  • DNA, Mitochondrial
  • Mitochondrial Proteins
  • TUFM protein, human
  • Peptide Elongation Factor Tu

Supplementary concepts

  • Combined Oxidative Phosphorylation Deficiency 4