Localized cerebral energy failure in DNA polymerase gamma-associated encephalopathy syndromes

Brain. 2010 May;133(Pt 5):1428-37. doi: 10.1093/brain/awq067. Epub 2010 Apr 16.

Abstract

Mutations in the catalytic subunit of the mitochondrial DNA-polymerase gamma cause a wide spectrum of clinical disease ranging from infantile hepato-encephalopathy to juvenile/adult-onset spinocerebellar ataxia and late onset progressive external ophthalmoplegia. Several of these syndromes are associated with an encephalopathy that characteristically shows episodes of rapid neurological deterioration and the development of acute cerebral lesions. The purpose of this study was to investigate the nature, distribution and natural evolution of central nervous system lesions in polymerase gamma associated encephalopathy focusing particularly on lesions identified by magnetic resonance imaging. We compared radiological, electrophysiological and pathological findings where available to study potential mechanisms underlying the episodes of exacerbation and acute cerebral lesions. We studied a total of 112 magnetic resonance tomographies and 11 computed tomographies in 32 patients with polymerase gamma-encephalopathy, including multiple serial examinations performed during both the chronic and acute phases of the disease and, in several cases, magnetic resonance spectroscopy and serial diffusion weighted studies. Data from imaging, electroencephalography and post-mortem examination were compared in order to study the underlying disease process. Our findings show that magnetic resonance imaging in polymerase gamma-related encephalopathies has high sensitivity and can identify patterns that are specific for individual syndromes. One form of chronic polymerase gamma-encephalopathy, that is associated with the c.1399G > A and c.2243G > C mutations, is characterized by progressive cerebral and cerebellar atrophy and focal lesions of the thalamus, deep cerebellar structures and medulla oblongata. Acute encephalopathies, both infantile and later onset, show similar pictures with cortical stroke-like lesions occurring during episodes of exacerbation. These lesions can occur both with and without electroencephalographic evidence of concurrent epileptic activity, and have diffusion, spectroscopic and histological profiles strongly suggestive of neuronal energy failure. We suggest therefore that both infantile and later onset polymerase gamma related encephalopathies are part of a continuum.

Publication types

  • Comparative Study
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Arginine
  • Brain / metabolism*
  • Brain / pathology
  • Brain Diseases / complications
  • Brain Diseases / diagnosis
  • Brain Diseases / genetics*
  • Brain Diseases / metabolism*
  • Cerebellum / pathology
  • Cysteine
  • DNA Polymerase gamma
  • DNA, Mitochondrial / genetics*
  • DNA-Directed DNA Polymerase / genetics*
  • Diffuse Cerebral Sclerosis of Schilder / genetics
  • Diffuse Cerebral Sclerosis of Schilder / metabolism
  • Diffusion Magnetic Resonance Imaging
  • Disease Progression
  • Electroencephalography
  • Energy Metabolism*
  • Epilepsy / etiology
  • Epilepsy / genetics
  • Epilepsy / metabolism
  • Glycine
  • Humans
  • Magnetic Resonance Angiography
  • Magnetic Resonance Imaging
  • Magnetic Resonance Spectroscopy
  • Mitochondrial Diseases / genetics
  • Mitochondrial Diseases / metabolism
  • Mutation*
  • Neocortex / pathology
  • Sensitivity and Specificity
  • Spinocerebellar Ataxias / genetics
  • Spinocerebellar Ataxias / metabolism
  • Stroke / etiology
  • Syndrome
  • Thalamus / pathology
  • Tomography, X-Ray Computed

Substances

  • DNA, Mitochondrial
  • Arginine
  • DNA Polymerase gamma
  • DNA-Directed DNA Polymerase
  • Cysteine
  • Glycine