Rapidly progressive autosomal dominant parkinsonism and dementia with pallido-ponto-nigral degeneration

Ann Neurol. 1992 Sep;32(3):312-20. doi: 10.1002/ana.410320303.

Abstract

We describe a family with nearly 300 members over 8 generations with 32 affected individuals who have an autosomal dominant neurodegenerative disease characterized by progressive parkinsonism with dystonia unrelated to medications, dementia, ocular motility abnormalities, pyramidal tract dysfunction, frontal lobe release signs, perseverative vocalizations, and urinary incontinence. The course is exceptionally aggressive; symptom onset and death consistently occur in the fifth decade. Positron emission tomographic studies with [18F]6-fluoro-L-dopa (6FD) were performed in 4 patients and 7 individuals at risk for development of the disease. All affected subjects had markedly reduced striatal uptake of 6FD (p less than 0.001). All individuals at risk had normal striatal uptake, but high 6FD uptake rate constants were noted in 3 of the 7 studied. Autopsy findings revealed severe neuronal loss with gliosis in substantia nigra, pontine tegmentum, and globus pallidus, with less involvement of the caudate and the putamen. There were no plaques, tangles, Lewy bodies, or amyloid bodies. This kindred appears to represent a neurodegenerative disease not heretofore described. We propose the following name for this new genetic disease: autosomal dominant parkinsonism and dementia with pallido-ponto-nigral degeneration.

Publication types

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

MeSH terms

  • Adult
  • Dementia / diagnostic imaging
  • Dementia / genetics*
  • Dementia / pathology
  • Female
  • Genes, Dominant
  • Gliosis / diagnostic imaging
  • Gliosis / genetics
  • Gliosis / pathology
  • Globus Pallidus / pathology*
  • Humans
  • Lewy Bodies / pathology
  • Male
  • Middle Aged
  • Nerve Degeneration*
  • Parkinson Disease / diagnostic imaging
  • Parkinson Disease / genetics*
  • Parkinson Disease / pathology
  • Pedigree
  • Pons / pathology*
  • Substantia Nigra / pathology*
  • Tomography, Emission-Computed