1. The facioscapulohumeral muscular dystrophy Rasch‐built overall disability scale (FSHD‐RODS)
- Author
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Nicol C. Voermans, Sabrina Sacconi, Corinne G.C. Horlings, Jeffrey Statland, Karlien Mul, Rabi Tawil, Ingemar S. J. Merkies, Alastair Corbett, Catharina G. Faber, Tatiana Hamadeh, Baziel G.M. van Engelen, RS: MHeNs - R1 - Cognitive Neuropsychiatry and Clinical Neuroscience, Klinische Neurowetenschappen, and MUMC+: MA Med Staf Spec Neurologie (9)
- Subjects
validity ,DISEASE ,Muscle and MNJ Disorders ,Disability Evaluation ,0302 clinical medicine ,Quality of life ,QUALITY-OF-LIFE ,Surveys and Questionnaires ,Facioscapulohumeral muscular dystrophy ,030212 general & internal medicine ,Reliability (statistics) ,EXAMPLE ,Disorders of movement Donders Center for Medical Neuroscience [Radboudumc 3] ,CLINICAL-TRIAL PREPAREDNESS ,Muscular Dystrophy, Facioscapulohumeral ,Neurology ,NATIONAL REGISTRY ,Scale (social sciences) ,Cohort ,Original Article ,musculoskeletal diseases ,congenital, hereditary, and neonatal diseases and abnormalities ,medicine.medical_specialty ,ENMC INTERNATIONAL WORKSHOP ,03 medical and health sciences ,ORDINAL SCALES ,medicine ,Humans ,Disabled Persons ,DRUG DEVELOPMENT ,FSHD ,reliability ,Rasch model ,business.industry ,Reproducibility of Results ,facioscapulohumeral dystrophy ,Rasch‐built disability scale ,OUTCOME MEASURES ,Interval Scale ,medicine.disease ,built disability scale ,Differential item functioning ,MEASUREMENT MODEL ,Physical therapy ,outcome research ,Rasch‐ ,Neurology (clinical) ,activity and participation ,business ,030217 neurology & neurosurgery - 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., This paper presents the development and validation of a patient‐reported Rasch‐built interval scale on activity and participation for facioscapulohumeral muscular dystrophy (FSHD‐RODS). The final 38‐inquiry scale met Rasch model expectations and showed adequate discriminative power, test‐retest reliability and validity. The use of this scale is recommended in the near future to determine the functional deterioration slope in facioscapulohumeral muscular dystrophy (FSHD) per year as a preparation for the upcoming clinical intervention trials in FSHD.
- Published
- 2021
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