Upper limb function in ambulatory and non-ambulatory patients with Duchenne muscular dystrophy

ABSTRACT New treatments proposed for patients with Duchenne muscular dystrophy (DMD) have their efficacy evaluated by lower limb tests. However, upper limb function tests evaluate both ambulatory (A) and non-ambulatory (NA) people. Thus, this work aimed to compare the upper limb function of patients A and NA with DMD and to correlate disease staging and upper limb function. This is a cross-sectional study in which patients were divided into A and NA according to the Vignos scale. Subsequently, the upper limb function was evaluated by the performance of upper limb (PUL) scale. The independent t-test, chi-square test, Mann-Whitney test, linear regression and Spearman’s correlation test were performed by SPSS, version 22. The research dealt with 51 patients, 20 A and 31 NA. There were differences between the groups reference age (p=0.001), body mass index (BMI) (p=0.016), schooling (p=0.011), Vignos score (p<0.001) and upper limb function (p<0.001). The linear regression analysis showed that whether the patient was A or NA influenced the upper limb function in 18 points on the PUL scale. There was a strong correlation between disease staging and upper limb function (r2=−0.769, p<0.001). The functional condition of the upper limb depends on whether the patient is A or NA, being worse function in NA patients. We concluded that the upper limb function has a strong correlation with the staging of the disease.

摘要 针对杜氏肌营养不良症（Duchenne muscular dystrophy, DMD）患者的新型治疗方案，其疗效多通过下肢测试进行评估。然而上肢功能测试可同时评估能行走（ambulatory, A）与不能行走（non-ambulatory, NA）的DMD患者。本研究旨在对比两类DMD患者的上肢功能，并分析疾病分期与上肢功能的相关性。本研究为横断面研究，依据维戈斯量表（Vignos scale）将患者分为能行走组与不能行走组；随后采用上肢功能评定（Performance of Upper Limb, PUL）量表评估受试者的上肢功能。本研究使用SPSS 22.0软件完成统计学分析，涵盖独立样本t检验（independent t-test）、卡方检验（chi-square test）、曼-惠特尼U检验（Mann-Whitney test）、线性回归（linear regression）及斯皮尔曼相关性检验（Spearman’s correlation test）。本研究共纳入51例患者，其中能行走组20例，不能行走组31例。两组在年龄（p=0.001）、体重指数（body mass index, BMI）（p=0.016）、受教育程度（p=0.011）、维戈斯评分（p<0.001）及上肢功能评分（p<0.001）方面存在显著差异。线性回归分析显示，患者是否能行走可解释PUL量表18分的上肢功能差异。疾病分期与上肢功能间存在强相关性（决定系数r²=-0.769, p<0.001）。上肢功能状态与患者是否能行走密切相关，不能行走组患者的上肢功能更差。综上，上肢功能与疾病分期存在显著强相关性。