期刊
JOURNAL OF THE PERIPHERAL NERVOUS SYSTEM
卷 18, 期 2, 页码 177-180出版社
WILEY-BLACKWELL
DOI: 10.1111/jns5.12024
关键词
clinical trial; inherited peripheral neuropathy; natural history; neuromuscular diseases; outcome assessment
资金
- Department of Health's National Institute for Health Research Biomedical Research Centres funding scheme
- NHMRC (National Health and Medical Research Council of Australia) [1007569]
- NHMRC (Centre of Research Excellence) [1031893]
- NIH (National Institutes of Neurological Disorders and Stroke and Office of Rare Diseases) [U54NS065712]
- Muscular Dystrophy Association
- Charcot Marie Tooth Association
- CMT Association of Australia
- MRC [MR/K000608/1] Funding Source: UKRI
- Medical Research Council [MR/K000608/1] Funding Source: researchfish
Long-term studies of Charcot-Marie-Tooth (CMT) disease across the entire lifespan require stable endpoints that measure the same underlying construct (e.g., disability). The aim of this study was to assess the relationship between the CMT Pediatric Scale (CMTPedS) and the adult CMT Neuropathy Score (CMTNSv2) in 203 children, adolescents, and young adults with CMT. There was a moderate curvilinear correlation between the CMTPedS and the CMTNSv2 (Spearman's rho =0.716, p<0.0001), although there appears to be a floor effect of the CMTNSv2 in patients with a milder CMT phenotype. Univariate analyses indicate that the relationship between the CMTPedS and CMTNSv2 scores improves with worsening disease severity and advancing age. Although one universal scale throughout life would be ideal, our data supports the transition from the CMTPedS in childhood to the CMTNSv2 in adulthood as a continuum of measuring lifelong disability in patients with CMT.
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