Clinical and pathological correlations in Charcot-Marie-Tooth neuropathy type 1A with the 17p11.2p12 duplication: a cross-sectional morphometric and immunohistochemical study in twenty cases

Muscle Nerve. 1998 Jul;21(7):869-77. doi: 10.1002/(sici)1097-4598(199807)21:7<869::aid-mus4>3.0.co;2-4.

Abstract

In a cross-sectional, clinical, and morphometric analysis we assessed the correlation between the clinical and pathological evolution of disease in 20 unrelated patients of various ages affected by Charcot-Marie-Tooth neuropathy type 1A (CMT1A) with the 17p11.2p12 (peripheral myelin protein 22, PMP22) duplication. The severity of neurologic deficits and slowing of motor conduction velocity at the median nerve did not vary significantly with the patients' age. The amount of demyelination was significantly higher below 15 years than in older age groups; in contrast, myelinated fiber and onion bulb densities were similar at all ages. The results indicate that in duplicated CMT1A, the pathological process develops early in life and progresses little during the course of the disease. Younger patients had lower g-ratio values, suggesting that the trigger of demyelination in early years could be a hypermyelination, resulting from PMP22 overexpression. Yet none of the 20 patients examined had immunohistochemical evidence of altered PMP22 expression. The early onset and development of the disorder make it difficult to detect PMP22 overdosage in nerve biopsies.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adolescent
  • Adult
  • Aged
  • Antibodies, Monoclonal
  • Biopsy
  • Blotting, Southern
  • Charcot-Marie-Tooth Disease / genetics*
  • Charcot-Marie-Tooth Disease / pathology*
  • Child
  • Chromosomes, Human, Pair 17*
  • Cross-Sectional Studies
  • Female
  • Fluorescent Antibody Technique
  • Humans
  • Immunoenzyme Techniques
  • Male
  • Middle Aged
  • Multigene Family*
  • Myelin P0 Protein / analysis
  • Myelin P0 Protein / genetics
  • Myelin P0 Protein / immunology
  • Myelin Proteins / analysis
  • Myelin Proteins / genetics
  • Myelin Proteins / immunology
  • Pedigree
  • Regression Analysis
  • Sural Nerve / chemistry
  • Sural Nerve / pathology

Substances

  • Antibodies, Monoclonal
  • Myelin P0 Protein
  • Myelin Proteins
  • PMP22 protein, human

Grants and funding