Trundell Dylan, Le Scouiller Stephanie, Gorni Ksenija, Seabrook Timothy, Vuillerot Carole
Roche Products Limited, Welwyn Garden City, UK.
Rare Diseases-Pharma Research and Early Development, F. Hoffmann-La Roche, Basel, Switzerland.
Neurol Ther. 2020 Dec;9(2):575-584. doi: 10.1007/s40120-020-00206-3. Epub 2020 Aug 27.
To investigate the validity and reliability of the 32-item Motor Function Measure (MFM32) in individuals with neuromuscular disorders (NMD), including spinal muscular atrophy (SMA), aged 2-5 years, and in non-ambulant individuals with Types 2 or 3 SMA, aged 2-25 years.
Test-retest reliability (intraclass correlation coefficient [ICC]), internal consistency (Cronbach's alpha [α]), convergent validity (Spearman rank-order correlations), and known-groups validity (analysis of covariance comparing groups defined by the Clinical Global Impression of Severity [CGI-S] scale and Vignos grade) were calculated. The analysis was performed on a dataset provided by Hospices Civils De Lyon, extracted from the multinational MFM32 database. A total of 165 individuals were included in the analyses, of whom 84 were in the NMD group (aged 2-5 years) and 81 were in the SMA group (aged 2-25 years).
Strong evidence of test-retest reliability (ICC: 2- to 5-years' population = 0.94-0.95; 2- to 25-years' population = 0.97), internal consistency (Cronbach's α: 2- to 5-years' population = 0.96; 2- to 25-years' population = 0.95), convergent validity (2- to 5-years' population: CGI-S rho = - 0.84, Vignos grade rho = - 0.79; 2- to 25-years' population: CGI-S rho = - 0.49), and known-groups validity (all P < 0.001) were demonstrated.
These analyses provide supportive evidence of the validity and reliability of the MFM32 in younger individuals with NMDs, aged 2-5 years, and in non-ambulant individuals with Types 2 or 3 SMA, aged 2-25 years, supporting the use of the MFM32 across a wide age range.
探讨32项运动功能量表(MFM32)在2至5岁患有神经肌肉疾病(NMD)(包括脊髓性肌萎缩症(SMA))的个体以及2至25岁非行走型2型或3型SMA个体中的有效性和可靠性。
计算重测信度(组内相关系数[ICC])、内部一致性(克朗巴哈系数[α])、收敛效度(斯皮尔曼等级相关)和已知群体效度(通过协方差分析比较由临床总体严重程度印象[CGI-S]量表和维格诺斯分级定义的组)。分析是在里昂公民医院提供的一个数据集上进行的,该数据集取自跨国MFM32数据库。共有165名个体纳入分析,其中84名在NMD组(2至5岁),81名在SMA组(2至25岁)。
有充分证据证明重测信度(ICC:2至5岁人群=0.94 - 0.95;2至25岁人群=0.97)、内部一致性(克朗巴哈α:2至5岁人群=0.96;2至25岁人群=0.95)、收敛效度(2至5岁人群:CGI-S相关系数=-0.84,维格诺斯分级相关系数=-0.79;2至25岁人群:CGI-S相关系数=-0.49)和已知群体效度(所有P<0.001)。
这些分析为MFM32在2至5岁患有NMD的较年轻个体以及2至25岁非行走型2型或3型SMA个体中的有效性和可靠性提供了支持性证据,支持在广泛年龄范围内使用MFM32。