The facioscapulohumeral muscular dystrophy Rasch-built overall disability scale (FSHD-RODS)

K. Mul*, T. Hamadeh, C.G.C. Horlings, R. Tawil, J.M. Statland, S. Sacconi, A.J. Corbett, N.C. Voermans, C.G. Faber, B.G.M. van Engelen, I.S.J. Merkies

*Corresponding author for this work

Research output: Contribution to journalArticleAcademicpeer-review

2 Citations (Web of Science)

Abstract

Background and objectives Facioscapulohumeral muscular dystrophy (FHSD) is a debilitating inherited muscle disease for which various therapeutic strategies are being investigated. Thus far, little attention has been given in FSHD to the development of scientifically sound outcome measures fulfilling regulatory authority requirements. The aim of this study was to design a patient-reported Rasch-built interval scale on activity and participation for FSHD.Methods A pre-phase FSHD-Rasch-built overall disability scale (pre-FSHD-RODS; consisting of 159 activity/participation items), based on the World Health Organization international classification of disease-related functional consequences was completed by 762 FSHD patients (Netherlands: n = 171; UK: n = 287; United States: n = 221; France: n = 52; Australia: n = 32). A proportion of the patient cohort completed it twice (n = 230; interval 2-4 weeks; reliability studies). The pre-FSHD-RODS was subjected to Rasch analyses to create a model fulfilling its requirements. Validity studies were performed through correlation with the motor function measure.Results The pre-FSHD-RODS did not meet the Rasch model expectations. Based on determinants such as misfit statistics and misfit residuals, differential item functioning, and local dependency, we systematically removed items until a final 38-inquiry (originating from 32 items; six items split) FSHD-RODS was constructed achieving Rasch model expectations. Adequate test-retest reliability and (cross-cultural and external) validity scores were obtained.Conclusions The FSHD-RODS is a disease-specific interval measure suitable for detecting activity and participation restrictions in patients with FSHD with good item/person reliability and validity scores. The use of this scale is recommended in the near future, to determine the functional deterioration slope in FSHD per year as a preparation for the upcoming clinical intervention trials in FSHD.
Original languageEnglish
Pages (from-to)2339-2348
Number of pages10
JournalEuropean Journal of Neurology
Volume28
Issue number7
DOIs
Publication statusPublished - 1 Jul 2021

Keywords

  • activity and participation
  • facioscapulohumeral dystrophy
  • FSHD
  • outcome research
  • Rasch&#8208
  • built disability scale
  • reliability
  • validity
  • CLINICAL-TRIAL PREPAREDNESS
  • ENMC INTERNATIONAL WORKSHOP
  • QUALITY-OF-LIFE
  • OUTCOME MEASURES
  • NATIONAL REGISTRY
  • MEASUREMENT MODEL
  • DRUG DEVELOPMENT
  • ORDINAL SCALES
  • DISEASE
  • EXAMPLE

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