CAG-repeat length and the age of onset in Huntington disease (HD): a review and validation study of statistical approaches

Am J Med Genet B Neuropsychiatr Genet. 2010 Mar 5;153B(2):397-408. doi: 10.1002/ajmg.b.30992.

Abstract

CAG-repeat length in the gene for HD is inversely correlated with age of onset (AOO). A number of statistical models elucidating the relationship between CAG length and AOO have recently been published. In the present article, we review the published formulae, summarize essential differences in participant sources, statistical methodologies, and predictive results. We argue that unrepresentative sampling and failure to use appropriate survival analysis methodology may have substantially biased much of the literature. We also explain why the survival analysis perspective is necessary if any such model is to undergo prospective validation. We use prospective diagnostic data from the PREDICT-HD longitudinal study of CAG-expanded participants to test conditional predictions derived from two survival models of AOO of HD. A prior model of the relationship of CAG and AOO originally published by Langbehn et al. yields reasonably accurate predictions, while a similar model by Gutierrez and MacDonald substantially overestimates diagnosis risk for all but the highest risk participants in this sample. The Langbehn et al. model appears accurate enough to have substantial utility in various research contexts. We also emphasize remaining caveats, many of which are relevant for any direct application to genetic counseling.

Publication types

  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't
  • Review

MeSH terms

  • Adult
  • Age of Onset
  • Aged
  • Aged, 80 and over
  • Female
  • Humans
  • Huntington Disease / diagnosis*
  • Huntington Disease / genetics*
  • Longitudinal Studies
  • Male
  • Middle Aged
  • Models, Statistical
  • Models, Theoretical*
  • Peptides / genetics*
  • Peptides / metabolism*
  • Prognosis
  • Prospective Studies
  • Trinucleotide Repeats

Substances

  • Peptides
  • polyglutamine