P.10.10 The Rasch-scaled motor function measure for patients with congenital disorders of muscle
| Autor: | C. Berard, M. Jain, Christine Payan, I. Poirot, M. Waite, Carole Vuillerot, V. Kinet, Carsten G. Bönnemann, Sungyoung Auh, Allan M. Glanzman, René Ecochard, P. Rippert, Dalil Hamroun |
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| Rok vydání: | 2013 |
| Předmět: |
medicine.medical_specialty
Measure (data warehouse) Rasch model business.industry Psychological intervention Polytomous Rasch model Patient specific behavioral disciplines and activities Motor function Clinical trial Physical medicine and rehabilitation Neurology Pediatrics Perinatology and Child Health medicine Physical therapy Neurology (clinical) business Genetics (clinical) Reliability (statistics) |
| Zdroj: | Neuromuscular Disorders. 23:791-792 |
| ISSN: | 0960-8966 |
| Popis: | Given recent progress toward therapeutic interventions in the congenital disorders of muscle (CDM), the validation of appropriate outcome measures for patients with congenital muscular dystrophies and myopathies becomes a high priority in order to precisely characterize natural history and the effects of treatments in future clinical trials. The aim of our study was to examine whether the Motor Function Measure (MFM), an ordinal functional scale designed for neuromuscular diseases, would fulfill all Rasch model expectations in a sample of 289 patients with CMD and to propose changes to improve its reliability and sensitivity to change. Three distinct analyses were performed considering the 3 domains (D1 Standing position and transfers, D2 Axial and proximal and D3 Distal motor function) of the MFM using RUMM 2030 software with a partial credit model. The original MFM failed to meet the model expectations in each domain. The reduction to a three-categorie response (0,1,2) scale in 20 items with disordered thresholds, produced ordered response thresholds for 18 items. Seven misfitting items were identified as not fitting and removed. Finally, the Rasch-scaled 10-item MFM D1, 9-item MFM D2 and 6-item MFM D3 conformed adequately to the Rasch measurement model. The Rasch-scaled-MFM D1 was well targeted to the patient population with quite similar mean location for items (0.000) and person (0.316) whereas the Rasch-scaled-MFM D2 and D3 were better targeted for more severely affected patients, as expected from the domains covered. Reliability coefficients of the Rasch scaled MFM suggest sufficient ability for the total score D1 (0.9) and D2 (0.8) and D3 (0.7) to distinguish between groups for research use. The results provide evidence for the measurement properties of the Rasch-scaled MFMCDM promoting its use as outcome measures in clinical trials for patients with congenital disorders of muscle. A raw score-to-Rasch person measure conversion is supplied for investigators. |
| Databáze: | OpenAIRE |
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