Early onset cerebellar ataxia and preservation of tendon reflexes: clinical phenotypes associated with GAA trinucleotide repeat expanded and non-expanded genotypes

J Peripher Nerv Syst. 1999;4(1):58-62.

Abstract

The aim of this study was to determine phenotypie characteristics of patients with early onset cerebellar ataxia (EOCA) with preserved tendon reflexes. The series comprises 25 patients, representing 10% of all ataxic patients who have been genetically studied in our laboratory since 1990. There were 11 males and 14 females. Fourteen patients were homozygous for the GAA expansion on chromosome 9q13 (group 1) and therefore a diagnosis of Friedreich's ataxia with retained reflexes (FARR) was given. The remaining 11 patients had two normal non-expanded alleles (group 2) and a working diagnosis of EOCA with retained reflexes (EOCARR) was established. Mean ages of onset were 13.7 +/- 5.9 years (3-25) for group 1 and 10.3 +/- 7.3 for group 2; the difference was not significant. Frequencies of symptoms and signs were also comparable for both groups the only significant differences being the higher frequency of nystagmus, cardiomyopathy and sensory neuropathy in group 1 patients. There was a tendency for FARR patients to have higher frequencies of hypopallesthesia in the lower limbs and skeletal deformities. In none of the cases diabetes mellitus was observed. We conclude that differentiation of FARR and EOCARR may be suspected by classical clinical and electrophysiological data and confirmed by analysis of the GAA repeat.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adolescent
  • Adult
  • Age Factors
  • Age of Onset
  • Child
  • Child, Preschool
  • Female
  • Frataxin
  • Friedreich Ataxia / genetics*
  • Friedreich Ataxia / physiopathology*
  • Genotype
  • Humans
  • Iron-Binding Proteins*
  • Male
  • Middle Aged
  • Phenotype
  • Phosphotransferases (Alcohol Group Acceptor) / genetics*
  • Reflex, Stretch / physiology*
  • Sequence Analysis, DNA
  • Trinucleotide Repeats*

Substances

  • Iron-Binding Proteins
  • Phosphotransferases (Alcohol Group Acceptor)