A longitudinal study of CMT1A using Rasch analysis based CMT neuropathy and examination scores
A longitudinal study of CMT1A using Rasch analysis based CMT neuropathy and examination scores
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DOI：
10.1212/wnl.0000000000009035
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发表时间：
2020-03-03
期刊：
影响因子：
9.9
通讯作者：
Shy, Michael E.
中科院分区：
文献类型：
--
作者：
Fridman, Vera;Sillau, Stefan;Shy, Michael E.
目的评价基于Rasch分析的加权Charcot-Marie-Tooth神经病变和检查评分（CMTNS-R和CMTES-R）对1A型Charcot-Marie-Tooth病（CMT1A）患者临床进展的敏感性。加权CMTNS和CMTES修改的类别反应与Rasch分析的标准分数。从基线CMTNS-R和CMTES-R的变化估计与纵向regression models.ResultsBaseline CMTNS-R和CMTES-R分数分别为517和1,177参与者。平均SD年龄的参与者与可用的CMTES-R评分为41 - 18（范围4 - 87）岁，56%是女性。377、321和244例患者在1、2和3年时进行了随访CMTES-R评估。混合回归模型显示，第2年至第6年的CMTES-R评分与基线相比发生显著变化（第2年较基线的平均变化为0.59分，p = 0.0004，n = 321）。与原始CMTES相比，CMTES-R显示2年时标准化反应平均值（平均变化/SD变化）改善了55%（0.17 vs 0.11）。CMTES-R在2年时的变化在轻度至中度受影响的患者中最大（1.48点平均变化，95%置信区间0.99 - 1.97，p <0.0001，对于基线CMTES-R评分0 - 9）. ConclusionThe CMTES-R demonstrates change over time in patients with CMT1A and is more sensitive than original CMTES. CMTES-R对基线疾病严重程度为轻度至中度的患者的变化最敏感，但未能捕获重度患者的进展。
ObjectiveTo evaluate the sensitivity of Rasch analysis-based, weighted Charcot-Marie-Tooth Neuropathy and Examination Scores (CMTNS-R and CMTES-R) to clinical progression in patients with Charcot-Marie-Tooth disease type 1A (CMT1A).MethodsPatients with CMT1A from 18 sites of the Inherited Neuropathies Consortium were evaluated between 2009 and 2018. Weighted CMTNS and CMTES modified category responses were developed with Rasch analysis of the standard scores. Change from baseline for CMTNS-R and CMTES-R was estimated with longitudinal regression models.ResultsBaseline CMTNS-R and CMTES-R scores were available for 517 and 1,177 participants, respectively. Mean SD age of participants with available CMTES-R scores was 41 18 (range 4-87) years, and 56% were female. Follow-up CMTES-R assessments at 1, 2, and 3 years were available for 377, 321, and 244 patients. A mixed regression model showed significant change in CMTES-R score at years 2 through 6 compared to baseline (mean change from baseline 0.59 points at 2 years, p = 0.0004, n = 321). Compared to the original CMTES, the CMTES-R revealed a 55% improvement in the standardized response mean (mean change/SD change) at 2 years (0.17 vs 0.11). Change in CMTES-R at 2 years was greatest in mildly to moderately affected patients (1.48-point mean change, 95% confidence interval 0.99-1.97, p < 0.0001, for baseline CMTES-R score 0-9).ConclusionThe CMTES-R demonstrates change over time in patients with CMT1A and is more sensitive than the original CMTES. The CMTES-R was most sensitive to change in patients with mild to moderate baseline disease severity and failed to capture progression in patients with severe CMT1A.ClinicalTrials.gov identifierNCT01193075.