Norfolk QOL-DN: validation of a patient reported outcome measure in transthyretin familial amyloid polyneuropathy

Etta J. Vinik, Aaron I. Vinik*, James F. Paulson, Ingemar S. J. Merkies, Jeff Packman, Donna R. Grogan, Teresa Coelho

*Corresponding author for this work

Research output: Contribution to journalArticleAcademicpeer-review

51 Citations (Web of Science)

Abstract

The Norfolk Quality of Life-Diabetic Neuropathy (QOL-DN) questionnaire is an instrument to assess QOL in diabetic polyneuropathy. The objective of this observational, cross-sectional study in 61 patients with V30M transthyretin familial amyloid polyneuropathy (TTR-FAP) and 16 healthy volunteers was to validate the Norfolk QOL-DN for assessment of QOL in TTR-FAP. Comparisons were conducted to identify the best items to discriminate disease stages and assess which individual Norfolk domains (symptoms, large fiber, small fiber, autonomic, and activities of daily living) would be most affected by disease stage. Analysis of individual items revealed a significant pattern of discrimination among disease stages (p
Original languageEnglish
Pages (from-to)104-114
JournalJournal of the Peripheral Nervous System
Volume19
Issue number2
DOIs
Publication statusPublished - Jun 2014

Keywords

  • nerve-fiber function
  • neurological function
  • Norfolk quality of life
  • outcome measure
  • transthyretin amyloid polyneuropathy

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