Background

The Charcot-Marie-Tooth disease Pediatric Scale (CMTPedS) has been used to measure aspects of disability in children with all types of Charcot-Marie-Tooth disease (CMT).

Objective

To translate and cross-culturally adapt the CMTPedS into Brazilian–Portuguese and determine its reliability and validity.

Methods

The translation and cross-cultural adaptation followed international guidelines recommendations. Twenty individuals with CMT were assessed. Two examiners assessed the participants for inter-rater reliability. Face validity was assessed by eight physical therapists that judged the relevance of each test item. The Bland-Altman analysis (bias) and standard error of measurement (SEM) complemented the analysis. Furthermore, intraclass correlation coefficients (ICC), weighted kappa (k), and internal consistency (Cronbach’s alpha) was determined.

Results

The CMTPedS was successfully translated and cross-culturally adapted. Twenty children/youth were enrolled in the study. Of these, the majority (55%) were girls with a mean age of 13.9 (range: from 6 to 18) years. Regarding face validity, the CMTPedS-Br showed relevant items for assessing children and youth with CMT. The ICC for the total score showed excellent reliability (ICC_2.1 = 0.93, 95% CI = 0.84, 0.97). The most reliable items were grip, dorsiflexion and plantar flexion strength while the least reliable items were pinprick, vibration, and gait. The internal consistency was excellent (α = 0.96, 95% CI = 0.91, 0.99) and the agreement showed small variability (bias = 0.15, 95% CI= -4.28, 4.60).

Conclusion

The CMTPedS-Br showed adequate reliability and face validity to measure disability in individuals with CMT. This tool will allow Brazil to be part of multicentered studies on such a rare but debilitating condition.

中文翻译：

---

Charcot-Marie-Tooth 病儿科量表对巴西葡萄牙语的翻译和跨文化适应及其测量特性的确定

背景

Charcot-Marie-Tooth 病儿科量表 (CMTPedS) 已用于衡量所有类型的 Charcot-Marie-Tooth 病 (CMT) 儿童的残疾方面。

客观的

将 CMTPedS 翻译和跨文化改编成巴西-葡萄牙语，并确定其可靠性和有效性。

方法

翻译和跨文化改编遵循国际指南建议。评估了 20 名 CMT 患者。两名考官评估了参与者的评分者间可靠性。表面效度由八名物理治疗师评估，他们判断每个测试项目的相关性。Bland-Altman 分析（偏差）和测量标准误差 (SEM) 补充了分析。此外，还确定了组内相关系数 (ICC)、加权 kappa ( k ) 和内部一致性（Cronbach α）。

结果

CMTPedS 被成功翻译和跨文化改编。二十名儿童/青年参加了这项研究。其中，大多数 (55%) 是平均年龄为 13.9 岁（范围：6 至 18 岁）的女孩。关于表面效度，CMTPedS-Br 显示了评估儿童和青少年 CMT 的相关项目。总分的 ICC 显示出极好的可靠性（ICC _2.1 = 0.93, 95% CI = 0.84, 0.97）。最可靠的项目是抓地力、背屈和跖屈强度，而最不可靠的项目是针刺、振动和步态。内部一致性非常好（α = 0.96, 95% CI = 0.91, 0.99）并且一致性显示出小的变异性（偏差 = 0.15, 95% CI = -4.28, 4.60）。

结论

CMTedS-Br 显示出足够的可靠性和表面效度来衡量 CMT 个体的残疾。该工具将使巴西能够参与这种罕见但令人衰弱的疾病的多中心研究。