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    The pediatric Charcot-Marie-Tooth (CMT) specific quality of life (QOL) outcome measure (pCMT-QOL) is a recently developed and validated patient-reported measure of health QOL for children with CMT. The aim of this study was to provide and validate an Italian version of the pCMT-QOL. The original English version was translated and adapted into Italian using standard procedures. pCMT-QOL was administered to patients genetically diagnosed with CMT, aged 8 to 18 years. A retest was given 2 weeks later to assess reliability in all patients. A total of 22 patients (median age 14 years, DS 2.5; M:F 1:1) affected with CMT (19 CMT1A, 2 CMT2A, 1 CMT2K) were assessed as part of their clinical visit. The Italian-pCMT-QOL demonstrate a high test-retest reliability. None of the patients experienced difficulty in completing the questionnaire, no further corrections were needed after administration in patients. The Italian-pCMT-QOL is a reliable, culturally adapted and comparable version of the original English pCMT-QOL. This questionnaire is expected to be valuable in monitoring disease progression and useful for future clinical trials in Italian-speaking children with CMT. © 2022 The Authors. Journal of the Peripheral Nervous System published by Wiley Periodicals LLC on behalf of Peripheral Nerve Society.

    Citation

    Isabella Moroni, Federica Rachele Danti, Davide Pareyson, Emanuela Pagliano, Giuseppe Piscosquito, Maria Foscan, Alessia Marchi, Anna Ardissone, Silvia Genitrini, Tong Tong Wu, Michael E Shy, Sindhu Ramchandren. Validation of the Italian version of the pediatric CMT quality of life outcome measure. Journal of the peripheral nervous system : JPNS. 2022 Jun;27(2):127-130

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    PMID: 35416371

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