Patient-reported disease burden in the Accelerate Clinical Trials in Charcot-Marie-Tooth Disease Study.

IF 3.9 3区医学 Q1 CLINICAL NEUROLOGY Journal of the Peripheral Nervous System Pub Date : 2024-10-10 DOI:10.1111/jns.12662

T Rehbein, J Purks, N Dilek, S Behrens-Spraggins, J E Sowden, K J Eichinger, J Burns, D Pareyson, S S Scherer, M M Reilly, M E Shy, M P McDermott, C R Heatwole, D N Herrmann

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Abstract

Background and aims: The Charcot-Marie-Tooth Disease Health Index (CMT-HI) is a disease-specific, patient-reported disease burden measure. As part of an international clinical trial readiness study, individuals with CMT1A (ages 18-75 years) underwent clinical outcome assessments (COAs), including the CMT-HI, to capture their longitudinal perspective on the disease burden.

Methods: Two hundred and fifteen participants underwent serial COAs including the CMT-HI, CMT Functional Outcome Measure (CMT-FOM), CMT Neuropathy Score (CMTNSv2R), and CMT Exam Score (CMTES/CMTES-R). Correlations between the total and subscale scores for the CMT-HI and other COAs were determined. Changes in the CMT-HI scores over 12 months were assessed using paired t-tests. The minimum clinically important difference (MCID) for the CMT-HI and its subscales were calculated by anchoring to a participant global impression of change scale.

Results: At baseline, CMT1A participants were 44.5 ± 15 years old (range: 18-75) and 58% were women. The mean CMT-HI was 25.7 ± 18.8 (range: 0-91.9; 100 reflecting maximal disease burden). The CMT-HI correlated with the CMT-FOM (r = .54, p < .0001), CMTNSv2R (r = .48, p < .0001), and CMTES/CMTES-R (r = .52/r = .54, p < .0001). Disease burden was greater in women than in men (CMT-HI 29.1 ± 19.1 vs. 21.2 ± 17.3, p = .001). Over 12 months, there was a nonsignificant mean increase in CMT-HI of 0.40 ± 10.0 (n = 189, p = .89). The MCID for the CMT-HI total score was 3.8 points (95% CI: 1.7-5.9).

Discussion: Patient-reported disease burden in CMT1A as measured by the CMT-HI is associated with measures of neurologic impairment and physical functioning. Women reported a higher disease burden than men. These data will inform the design of clinical trials in CMT1A.

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Charcot-Marie-Tooth 病加速临床试验研究中患者报告的疾病负担。

背景和目的：Charcot-Marie-Tooth 疾病健康指数（CMT-HI）是一种针对特定疾病、由患者报告的疾病负担测量方法。作为国际临床试验准备研究的一部分，CMT1A 患者（18-75 岁）接受了包括 CMT-HI 在内的临床结果评估（COA），以了解他们对疾病负担的纵向看法：215 名参与者接受了包括 CMT-HI、CMT 功能结果测量（CMT-FOM）、CMT 神经病变评分（CMTNSv2R）和 CMT 检查评分（CMTES/CMTES-R）在内的连续 COA。确定了 CMT-HI 的总分和分量表得分与其他 COA 之间的相关性。采用配对 t 检验评估 12 个月内 CMT-HI 分数的变化。CMT-HI及其分量表的最小临床重要差异（MCID）通过锚定到参与者总体变化印象量表来计算：基线时，CMT1A 患者的年龄为 44.5 ± 15 岁（范围：18-75 岁），58% 为女性。CMT-HI 平均值为 25.7 ± 18.8（范围：0-91.9；100 代表最大疾病负担）。CMT-HI 与 CMT-FOM 相关（r = .54，p 讨论）：根据 CMT-HI 测量，患者报告的 CMT1A 疾病负担与神经功能损伤和身体功能的测量结果相关。女性报告的疾病负担高于男性。这些数据将为 CMT1A 临床试验的设计提供参考。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

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来源期刊

Journal of the Peripheral Nervous System 医学-临床神经学

CiteScore

6.10

自引率

7.90%

发文量

审稿时长

>12 weeks

期刊介绍： The Journal of the Peripheral Nervous System is the official journal of the Peripheral Nerve Society. Founded in 1996, it is the scientific journal of choice for clinicians, clinical scientists and basic neuroscientists interested in all aspects of biology and clinical research of peripheral nervous system disorders. The Journal of the Peripheral Nervous System is a peer-reviewed journal that publishes high quality articles on cell and molecular biology, genomics, neuropathic pain, clinical research, trials, and unique case reports on inherited and acquired peripheral neuropathies. Original articles are organized according to the topic in one of four specific areas: Mechanisms of Disease, Genetics, Clinical Research, and Clinical Trials. The journal also publishes regular review papers on hot topics and Special Issues on basic, clinical, or assembled research in the field of peripheral nervous system disorders. Authors interested in contributing a review-type article or a Special Issue should contact the Editorial Office to discuss the scope of the proposed article with the Editor-in-Chief.