Measurement of the Functional Status of Patients with Different Types of Muscular Dystrophy

肌肉失養症(muscular dystrophy)是一群漸進性肌肉無力疾病，並造成功能持續退步。使用設計良好的評估表來正確評估功能狀態，為治療重要的基礎。本研究目的為檢測評估功能工具，使用於不同類型肌肉失養症功能狀態其合適情形。以Brooke和Vignos評估表分別評估上肢及下肢功能等級，以巴歇爾指數(Barthel index)評估日常活動的功能。統計檢測各種評估工具對不同類型肌肉失養症的接受度(acceptability)，並討論不同類型的表現。病患來自台灣多個醫療院所，共有179位，46.9%為裘馨型肌肉失養症(Duchenne muscular dystrophy)，屬於嚴重進展的肌肉失養症；其餘53.1%為貝克型肌肉失養症(Becker muscular dystrophy)、肢帶型肌肉失養症(limb girdle muscular dystrophy)與顏肩肱型肌肉失養症(fascioscapulohumeral muscular dystrophy)，屬於緩慢進展的肌肉失養症。結果顯示Brooke 評估表適用於裘馨型肌肉失養症，但不易分辨緩慢進展的肌肉失養症，其地板效應(floor effect)都較大（範圍為20.0%至61.9%），尤其是貝克型肌肉失養症。Vignos評估表方面，同樣的貝克型(23.8%)與顏肩肱型(50.0%)肌肉失養症有較大的地板效應，除此以外其評估等級6到8，有一些病患並不適用，因這些等級為使用長腿支架(long leg brace)來行走或站立，而他們並沒有使用。巴歇爾指數天花板效應(ceiling effect)在緩慢進展的肌肉失養症病患非常顯著，而地板效應(flooreffect)出現在裘馨型肌肉失養症病患。緩慢進展的肌肉失養症中以顏肩肱型肌肉失養症病患功能最佳，有比較好的下肢功能，日常生活功能也較好。本研究提供使用功能評估表於不同類型之肌肉失養症病患時之接受度，這些功能評估表出現的一些限制，在臨床上使用時應特別留意，尤其是使用於緩慢進展的肌肉失養症，建議使用時可考慮合併其他評估或使用題型結構較完整的評估量表。

關鍵字

巴歇爾指數；功能狀態；肌肉失養症

並列摘要

Muscular dystrophy (MD) comprises a group of diseases characterized by progressive muscle weakness that induces functional deterioration. Clinical management requires the use of a well-designed scale to measure patients' functional status. This study aimed to investigate the quality of the functional scales used to assess patients with different types of MD. The Brooke scale and the Vignos scale were used to grade arm and leg function, respectively. The Barthel Index was used to evaluate the function of daily living activity. We performed tests to assess the acceptability of these scales. The characteristics of the different types of MD are discussed. This was a multicenter study and included patients diagnosed with Duchenne muscular dystrophy (DMD) (classified as severely progressive MD), Becker muscular dystrophy (BMD), limb girdle muscular dystrophy (LGMD) and facioscapulohumeral muscular dystrophy (FSHD). BMD, LGMD, and FSHD were classified as slowly progressive MD. The results demonstrated that the Brooke scale was acceptable for grading arm function in DMD, but was unable to discriminate between differing levels of severity in slowly progressive MD. The floor effect was large for all types of slowly progressive MD (range, 20.0-61.9), and was especially high for BMD. The floor effect was also large for BMD (23.8%) and FSHD (50.0%) using the Vignos scale. Grades 6-8 of the Vignos scale were inapplicable because they included items involving the use of long leg braces for walking or standing, and some patients did not use long leg braces. In the Barthel Index, a ceiling effect was prominent for slowly progressive MD (58.9%), while a floor effect existed for DMD (17.9°o). Among the slowly progressive MDs, FSHD patients had the best level of functioning; they had better leg function and their daily living activities were less affected than patients with other forms of slowly progressive MD. The results of this study demonstrate the acceptability of the different applications used for measuring functional status in patients with different types of MD. Some of the limitations of these measures as applied to MD should be carefully considered, especially in patients with slowly progressive MD. We suggest that these applications be used in combination with other measures, or that a complicated instrument capable of evaluating the various levels of functional status be used.

並列關鍵字

Barthel Index ； functional status ； muscular dystrophy

國際替代計量

Measurement of the Functional Status of Patients with Different Types of Muscular Dystrophy

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