The Val30Met familial amyloid polyneuropathy specific Rasch-built overall disability scale (FAP-RODS(©))

Mariëlle H J Pruppers, Ingemar S J Merkies, Catharina G Faber, Ana M Da Silva, Vanessa Costa, Teresa Coelho

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Abstract

Familial amyloid polyneuropathy (FAP) is a chronic debilitating multi-organic disorder, mainly assessed using ordinal-based impairment measures. To date, no outcome measure at the activity and participation level has been constructed in FAP. The current study aimed to design an interval activity/participation scale for FAP through Rasch methodology. A preliminary FAP Rasch-built overall disability scale (pre-FAP-RODS) containing 146 activity/participation items was assessed twice (interval: 2-4 week; test-retest reliability) in 248 patients with Val30Met FAP examined in Porto, Portugal, of which 65.7% have received liver transplantation. An ordinal-based 24-item FAP-symptoms inventory questionnaire (FAP-SIQ) was also assessed (validity purposes). The pre-FAP-RODS and FAP-SIQ data were subjected to Rasch analyses. The pre-FAP-RODS did not meet model's expectations. On the basis of requirements such as misfit statistics, differential item functioning, and local dependency, items were systematically removed until a final 34-item FAP-RODS(©) was constructed fulfilling all Rasch requirements. Acceptable reliability/validity scores were demonstrated. In conclusion, the 34-item FAP-RODS(©) is a disease-specific interval measure suitable for detecting activity and participation restrictions in patients with FAP. The use of the FAP-RODS(©) is recommended for future international clinical trials in patients with Val30Met FAP determining its responsiveness and its cross-cultural validation. Its expansion to other forms of FAP should also be focus of future clinical studies.

Original languageEnglish
Pages (from-to)319-27
Number of pages9
JournalJournal of the Peripheral Nervous System
Volume20
Issue number3
DOIs
Publication statusPublished - Sep 2015

Keywords

  • Activities of Daily Living
  • Adult
  • Aged
  • Amyloid Neuropathies, Familial
  • Disability Evaluation
  • Female
  • Humans
  • Male
  • Methionine
  • Middle Aged
  • Mutation
  • Outcome Assessment (Health Care)
  • Portugal
  • Prealbumin
  • Reproducibility of Results
  • Retrospective Studies
  • Severity of Illness Index
  • Surveys and Questionnaires
  • Valine

Cite this

Pruppers, M. H. J., Merkies, I. S. J., Faber, C. G., Da Silva, A. M., Costa, V., & Coelho, T. (2015). The Val30Met familial amyloid polyneuropathy specific Rasch-built overall disability scale (FAP-RODS(©)). Journal of the Peripheral Nervous System, 20(3), 319-27. https://doi.org/10.1111/jns.12120