Dihydrolipoamide dehydrogenase (DLD) deficiency in a Spanish patient with myopathic presentation due to a new mutation in the interface domain

J Inherit Metab Dis. 2010 Dec:33 Suppl 3:S315-9. doi: 10.1007/s10545-010-9169-4. Epub 2010 Jul 21.

Abstract

We present a 32-year-old patient who, from age 7 months, developed photophobia, left-eye ptosis and progressive muscular weakness. At age 7 years, she showed normal psychomotor development, bilateral ptosis and exercise-induced weakness with severe acidosis. Basal blood and urine lactate were normal, increasing dramatically after effort. PDHc deficiency was demonstrated in muscle and fibroblasts without detectable PDHA1 mutations. Ketogenic diet was ineffective, however thiamine gave good response although bilateral ptosis and weakness with acidosis on exercise persisted. Recently, DLD gene analysis revealed a homozygous missense mutation, c.1440 A>G (p.I480M), in the interface domain. Both parents are heterozygous and DLD activity in the patient's fibroblasts is undetectable. The five patients that have been reported with DLD-interface mutations suffered fatal deteriorations. Our patient's disease is milder, only myopathic, more similar to that due to mutation p.G229C in the NAD(+)-binding domain. Two of the five patients presented mutations (p.D479V and p.R482G) very close to the present case (p.I480M). Despite differing degrees of clinical severity, all three had minimal clues to DLD deficiency, with occasional minor increases in α-ketoglutarate and branched-chain amino acids. In the two other patients, hypertrophic cardiomyopathy was a significant feature that has been attributed to moonlighting proteolytic activity of monomeric DLD, which can degrade other mitochondrial proteins, such as frataxin. Our patient does not have cardiomyopathy, suggesting that p.I480M may not affect the DLD ability to dimerize to the same extent as p.D479V and p.R482G. Our patient, with a novel mutation in the DLD interface and mild clinical symptoms, further broadens the spectrum of this enzyme defect.

Publication types

  • Case Reports
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Acidosis, Lactic / diagnosis
  • Acidosis, Lactic / drug therapy
  • Acidosis, Lactic / enzymology
  • Acidosis, Lactic / genetics*
  • Acidosis, Lactic / physiopathology
  • Adult
  • Amino Acid Sequence
  • Base Sequence
  • Biomarkers / blood
  • Biomarkers / urine
  • Blepharoptosis / diagnosis
  • Blepharoptosis / enzymology
  • Blepharoptosis / genetics
  • Cells, Cultured
  • DNA Mutational Analysis
  • Dietary Supplements
  • Female
  • Genetic Predisposition to Disease
  • Heredity
  • Heterozygote
  • Homozygote
  • Humans
  • Lactic Acid / blood
  • Lactic Acid / urine
  • Maple Syrup Urine Disease / diagnosis
  • Maple Syrup Urine Disease / drug therapy
  • Maple Syrup Urine Disease / enzymology
  • Maple Syrup Urine Disease / genetics*
  • Maple Syrup Urine Disease / physiopathology
  • Molecular Sequence Data
  • Muscle Strength / genetics
  • Muscle Weakness / diagnosis
  • Muscle Weakness / drug therapy
  • Muscle Weakness / enzymology
  • Muscle Weakness / genetics*
  • Muscle Weakness / physiopathology
  • Mutation, Missense*
  • Pedigree
  • Phenotype
  • Photophobia / diagnosis
  • Photophobia / enzymology
  • Photophobia / genetics
  • Protein Structure, Tertiary
  • Pyruvate Dehydrogenase Complex Deficiency Disease / diagnosis
  • Pyruvate Dehydrogenase Complex Deficiency Disease / enzymology
  • Pyruvate Dehydrogenase Complex Deficiency Disease / genetics
  • Spain
  • Thiamine / therapeutic use
  • Thioctic Acid / analogs & derivatives*
  • Thioctic Acid / chemistry
  • Thioctic Acid / deficiency
  • Thioctic Acid / genetics
  • Treatment Outcome

Substances

  • Biomarkers
  • Lactic Acid
  • dihydrolipoamide
  • Thioctic Acid
  • Thiamine

Supplementary concepts

  • Lactic Acidosis, Congenital Infantile, Due To LAD Deficiency

Associated data

  • OMIM/OMIM238331