Responsiveness of Upper Limb Scales and Trunk Control for the Evolution of Patients With Duchenne Muscular Dystrophy

Abstract

Objective: To verify the interval of responsiveness to the scales Segmental Assessment of Trunk Control (SATCo-BR), Performance of Upper Limbs (PUL), and Jebsen Taylor Test (JTT) in patients with Duchenne Muscular Dystrophy (DMD). Methods: We assessed patients with DMD aged 6 to 19 years old and with mini-mental (MMSE) score above 10 points. The assessments were performed individually, in a single session. The upper limb function was performed by PUL and JTT, and trunk control by SATCo-BR. Assessments were repeated six and 12 months after the initial assessment. The repeated-measures analysis of variance model and Bonferroni’s multiple comparison method were employed as post hoc analysis; when the ANOVA assumptions were not met, the Friedman test was applied. Results: The sample consisted of 28 patients evaluated in three moments (initial, and six and 12 months after the beginning). There was a time effect for the Upper Limb function performance in the total JTT, and for the subtests, except for subtests 1 and 6, which did not show a difference between the different moments. There was also a time effect for the score of total PUL, proximal PUL, intermediate PUL, and distal PUL. In the SATCo-BR, this effect was observed between the initial and 6 months, and between the initial and 12 months. Conclusions: The JTT, PUL, and SATCo-BR scales can detect changes over time, and they showed responsiveness to detect the evolution of the disease in the 6-month interval.

Patient-led development of digital endpoints and the use of computer vision analysis in assessment of motor function in rare diseases

“…Understanding, characterising and accurately measuring compensatory movements to make up for the loss of motor function in DMD patients is important, as they may indicate disease progression and trigger the start of use of assistive devices and other aids ( Sá et al, 2016 ). Additionally, increased trunk movement can be observed over a 6-month period despite the fact essential motor tasks are preserved ( Maciel et al, 2021a ; Maciel et al, 2021b ), indicating the importance of the early detection and quantification of changes in compensatory movements. We assessed trunk movement by measuring the location of the shoulders over the course of the hands-to-head while seated task, which remained quite stationary in participant 1 (ambulant) compared to participant 10 (non-ambulant).…”