University of Sydney, School of Health Sciences & The Children's Hospital at Westmead, Sydney, Australia.
Department of Occupational Therapy, The Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, USA.
J Peripher Nerv Syst. 2020 Sep;25(3):288-291. doi: 10.1111/jns.12406. Epub 2020 Aug 26.
The CMT-FOM is a 13-item clinical outcome assessment (COA) that measures physical ability in adults with Charcot-Marie-Tooth disease (CMT). Test-retest reliability, internal consistency and convergent validity have been established for the CMT-FOM. This current study sought to establish inter-rater reliability. Following an in-person training of six international clinical evaluators we recruited 10 participants with genetically diagnosed CMT1A, (aged 18-74 years, 6 female). Participants were evaluated using the CMT-FOM over 2 days. Participants were given at least a 3 hour rest between evaluations, and were assessed twice each day. Following the provision of training by master trainers, all 13 items of the CMT-FOM exhibited excellent inter-rater reliability for raw scores (ICC 0.825-0.989) and z-scores (ICC 0.762-0.969). Reliability of the CMT-FOM total score was excellent (ICC 0.983, 95% CI 0.958-0.995). The CMT-FOM is a reliable COA used by clinical evaluators internationally. The next steps are to establish further validation through psychometric evaluation of the CMT-FOM in the Accelerate Clinical Trials in CMT (ACT-CMT) study.
CMT-FOM 是一种 13 项的临床结局评估（COA），用于评估成年型腓骨肌萎缩症（CMT）患者的身体机能。CMT-FOM 的测试-重测信度、内部一致性和聚合效度已经得到了验证。本研究旨在建立其评分者间信度。在对 6 名国际临床评估者进行了现场培训后，我们招募了 10 名经基因诊断的 CMT1A 患者（年龄 18-74 岁，女性 6 名）参与研究。参与者在 2 天内使用 CMT-FOM 进行评估。两次评估之间至少间隔 3 小时，每天评估两次。在接受了主培训师的培训后，CMT-FOM 的 13 项原始评分（ICC 0.825-0.989）和 z 评分（ICC 0.762-0.969）均具有极好的评分者间信度。CMT-FOM 总分的信度极好（ICC 0.983，95%CI 0.958-0.995）。CMT-FOM 是一种可靠的 COA，在国际上由临床评估者使用。下一步将通过在 CMT 加速临床试验（ACT-CMT）中对 CMT-FOM 进行心理测量学评估来进一步验证其有效性。
