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Improving the measurement properties of the Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R): deriving a valid measurement total for the calculation of change.

Carolyn Anne YoungAmina ChaouchChristopher J McDermottAmmar Al-ChalabiSuresh K ChhetriKevin TalbotAndrea MalaspinaRoger MillsAlan Tennant
Published in: Amyotrophic lateral sclerosis & frontotemporal degeneration (2024)
A total ALSFRS-R ordinal raw score can lead to inferential bias in clinical trial results due to its non-linear nature. On the interval level transformation, more than 5 points difference is required before a statistically significant detectable difference can be observed. Transformation to interval level data should be mandatory in clinical trials.
Keyphrases
  • clinical trial
  • amyotrophic lateral sclerosis
  • phase ii
  • double blind
  • open label
  • study protocol
  • electronic health record
  • phase iii
  • big data
  • machine learning
  • data analysis
  • artificial intelligence
  • monte carlo