Department of Neurology, University of Iowa Carver College of Medicine, Iowa City, Iowa, USA.
Neuromuscular Omnicentre (NEMO)-Fondazione Serena Onlus, Genoa, Liguria, Italy.
J Peripher Nerv Syst. 2020 Jun;25(2):138-142. doi: 10.1111/jns.12383. Epub 2020 May 26.
The Charcot-Marie-Tooth disease Pediatric Scale (CMTPedS) is a Rasch-built clinical outcome measure of disease severity. It is valid, reliable, and responsive to change for children and adolescents aged 3 to 20 years. The aim of this study was to translate and validate an Italian version of the CMTPedS using a validated framework of transcultural adaptation. The CMTPedS (Italian) was translated and culturally adapted from source into Italian by two experts in CMT with good English language proficiency. The two translations were reviewed by a panel of experts in CMT. The agreed provisional version was back translated into English by a professional translator. The definitive Italian version was developed during a consensus teleconference by the same panel. CMT patients were assessed with the final version of the outcome measure and a subset had a second assessment after 2 weeks to evaluate test-retest reliability. Seventeen patients with CMT aged 5 to 20 years (eight female) were evaluated with the CMTPedS (Italian), and test-retest was performed in three patients. The CMTPedS (Italian) showed a high test-retest reliability. No patient had difficulty in completing the scale. The instructions for the different items were clearly understood by clinicians and therefore the administration of the outcome measure was straight forward and easily understood by the children assessed. The CMTPedS (Italian) will be used for clinical follow-up and in clinical research studies in the Italian population. The data is fully comparable to that obtained from the English language version.
Charcot-Marie-Tooth 疾病儿科量表（CMTPedS）是一种基于 Rasch 理论构建的疾病严重程度临床结局测量工具。它在 3 至 20 岁的儿童和青少年中具有有效性、可靠性和对变化的反应能力。本研究旨在使用经过验证的跨文化适应框架，将 CMTPedS 翻译成意大利语并进行验证。CMTPedS（意大利语）由两名具有良好英语语言能力的 CMT 专家从源语言翻译成意大利语，并进行文化适应性调整。这两个翻译版本由 CMT 专家小组进行了审查。经专家组同意的暂定版本由专业翻译人员翻译成英语。最终的意大利语版本是由同一专家组通过电话会议达成共识制定的。使用最终版本的结局测量工具对 17 名年龄在 5 至 20 岁的 CMT 患者（8 名女性）进行了评估，其中 3 名患者在 2 周后进行了第二次评估，以评估测试-重测信度。CMTPedS（意大利语）显示出较高的测试-重测信度。没有患者在完成量表方面有困难。临床医生清楚地理解了不同项目的说明，因此，该结局测量工具的管理对接受评估的儿童来说是直接和易于理解的。CMTPedS（意大利语）将用于意大利人群的临床随访和临床研究。数据与从英语版本获得的数据完全可比。
