The quick motor function test: a new tool to rate clinical severity and motor function in Pompe patients
| Autor: | Hugo J. Duivenvoorden, Pieter A. van Doorn, M.L.C. Hagemans, Juna M. de Vries, Nadine A. M. E. van der Beek, Ans T. van der Ploeg, Carine I. van Capelle, Robert T. Leshner |
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| Přispěvatelé: | Pediatrics, Neurology, Psychiatry |
| Rok vydání: | 2012 |
| Předmět: |
Adult
Male medicine.medical_specialty Psychometrics Adolescent Intraclass correlation Manual Muscle Testing Cohort Studies 03 medical and health sciences Disability Evaluation Young Adult 0302 clinical medicine Physical medicine and rehabilitation Glycogen storage disease type II Outcome Assessment Health Care Genetics medicine Humans Genetics(clinical) 030212 general & internal medicine Muscle Strength Child Survival rate Genetics (clinical) Aged business.industry Glycogen Storage Disease Type II Reproducibility of Results Enzyme replacement therapy Middle Aged medicine.disease 3. Good health Survival Rate Child Preschool Cohort Physical therapy Original Article Female business 030217 neurology & neurosurgery Cohort study |
| Zdroj: | Journal of Inherited Metabolic Disease, 35, 317-323. Springer Netherlands Journal of Inherited Metabolic Disease Journal of Inherited Metabolic Disease; Vol 35 |
| ISSN: | 1573-2665 0141-8955 |
| DOI: | 10.1007/s10545-011-9388-3 |
| Popis: | Pompe disease is a lysosomal storage disorder characterized by progressive muscle weakness. With the emergence of new treatment options, psychometrically robust outcome measures are needed to monitor patients’ clinical status. We constructed a motor function test that is easy and quick to use. The Quick Motor Function Test (QMFT) was constructed on the basis of the clinical expertise of several physicians involved in the care of Pompe patients; the Gross Motor Function Measure and the IPA/Erasmus MC Pompe survey. The test comprises 16 items. Validity and test reliability were determined in a cohort of 91 Pompe patients (5 to 76 years of age). In addition, responsiveness of the scale to changes in clinical condition over time was examined in a subgroup of 18 patients receiving treatment and 23 untreated patients. Interrater and intrarater reliabilities were good (intraclass correlation coefficients: 0.78 to 0.98 and 0.76 to 0.98). The test correlated strongly with proximal muscle strength assessed by hand held dynamometry and manual muscle testing (rs= 0.81, rs=0.89), and showed significant differences between patient groups with different disease severities. A clinical-empirical exploration to assess responsiveness showed promising results, albeit it should be repeated in a larger group of patients. In conclusion, the Quick Motor Function Test can reliably rate clinical severity and motor function in children and adults with Pompe disease. Electronic supplementary material The online version of this article (doi:10.1007/s10545-011-9388-3) contains supplementary material, which is available to authorized users. |
| Databáze: | OpenAIRE |
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