期刊
JOURNAL OF THE PERIPHERAL NERVOUS SYSTEM
卷 25, 期 3, 页码 292-296出版社
WILEY
DOI: 10.1111/jns.12397
关键词
Charcot-Marie-Tooth disease; clinical trials; patient-reported outcome measures; quality of life; translation and cultural adaptation
资金
- Foundation for the National Institutes of Health [NIH 1 U01 NS109403-01]
TheCharcot-Marie-ToothHealth Index (CMT-HI) is a disease-specific patient-reported outcome measure measuring overall disease burden inCharcot-Marie-Tooth (CMT) patients, designed for natural history studies and clinical trials in English-speaking affected individuals. We developed and validated its ItalianCharcot-Marie-ToothHealth Index (I-CMT-HI) version. The questionnaire was translated and culturally adapted from source into Italian by two neurologists experienced in CMT and neuromuscular disorders (NMDs). The two translations were reviewed by a panel of seven experts in CMT and NMD. The provisional version was back-translated into English by a professional translator. The definitive Italian version was developed during a consensus teleconference by the panel and a patient representative from ACMT-Rete. A series of clinically and genetically characterized CMT patients completed the final questionnaire; 11 participated in a test-retest reliability assessment of the instrument. The I-CMT-HI was administered to 30 CMT patients (13 CMT1A, eight CMTX1, two CMT1B, two CMT1E, two CMT2I, one CMT2A, one CMT2N, one distal Hereditary Motor Neuropathy), with test-rest in 11:14 females and 16 males, aged (mean +/- SD) 48.0 +/- 16.4 years (range 18-81), with CMT Examination Score (CMTES) = 10.0 +/- 4.4 (range 2-18). The I-CMT-HI mean total score was 29.4 +/- 21.2 (range 0.1-60.3). The I-CMT-HI showed a high test-retest reliability: intraclass correlation coefficient = 0.95 (95% confidence interval, 0.84-0.99). No patient had difficulty in completing the questionnaire and none reported any problem with the questions' formulation. The total CMT-HI score was positively correlated with age and CMTES, with higher disease burden with increasing age and disease severity according to the CMTES. The I-CMT-HI is now ready for use in clinical studies in the Italian population.
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