A critical review of functional assessment tools for upper limbs in Duchenne muscular dystrophy.

Department of Paediatric Neurology, Catholic University, Rome, Italy.  Dubowitz Neuromuscular Centre, Institute of Child Health, London
Developmental Medicine & Child Neurology (Impact Factor: 3.29). 06/2012; 54(10):879-885. DOI: 10.1111/j.1469-8749.2012.04345.x
Source: PubMed

ABSTRACT The recent development of therapeutic approaches for Duchenne muscular dystrophy (DMD) has highlighted the need to identify clinical outcome measures for planned efficacy studies. Although several studies have reported the value of functional scales, timed tests, and measures of endurance aimed at ambulant individuals, less has been done to identify reliable measures of function in individuals who have lost ambulation. The aim of this paper is to provide a critical review of the existing literature on functional measures assessing upper extremity function in DMD. Four observer-rated, performance-based measures and four self-reported scales have been previously used in DMD. Each scale provides useful information but none reflects all the different levels of functional ability in activities of daily living observed in individuals with DMD at different ages.

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Available from: Anna Mayhew, Oct 16, 2014
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    ABSTRACT: Upper limb evaluation of patients with Duchenne Muscular Dystrophy is crucially important to evaluations of efficacy of new treatments in non-ambulant patients. In patients who have lost ambulation, there are few validated and informative outcome measures. In addition, longitudinal data demonstrating sensitivity to clinical evolution of outcome measures over short-term periods are lacking. We report here the results of a one-year multicenter study using specifically designed tools to assess grip, pinch strength, and hand function in wheelchair-bound patients. Our study assessed 53 non-ambulant patients with Duchenne muscular dystrophy aged 17.1 ± 4.8 years (range: 9 - 28.1 years). The average Brooke functional score of these patients was 4.6 ± 1.1. The average forced vital capacity was 44.5% predicted and 19 patients used non-invasive ventilation. Patients were assessed at baseline, 6 months, and one year using the Motor Function Measure and innovative devices (namely the MyoSet composed of MyoGrip, MyoPinch, and MoviPlate). Our study confirmed preliminary data previously reported regarding feasibility of use and of reliability of the MyoSet and the correlation at baseline between distal strength and clinical outcomes such as FVC, Brooke score, age, and duration since loss of ambulation. A significant correlation was observed between the distal upper limb strength and clinical variables. The sensitive dynamometers (MyoGrip and MyoPinch) and MoviPlate captured a 12-month change in non-ambulant Duchenne muscular dystrophy patients of all ages. NCT00993161 NCT00993161.
    PLoS ONE 02/2015; 10(2):e0113999. DOI:10.1371/journal.pone.0113999 · 3.53 Impact Factor
  • Neuromuscular Disorders 07/2012; 22(11). DOI:10.1016/j.nmd.2012.06.006 · 3.13 Impact Factor
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    ABSTRACT: One of the major challenges in the neuromuscular field has been lack of upper extremity outcome measures that can be useful for clinical therapeutic efficacy studies. Using vision-based sensor system and customized software, 3-dimensional (3D) upper extremity motion analysis can reconstruct a reachable workspace as a valid, reliable and sensitive outcome measure in various neuromuscular conditions where proximal upper extremity range of motion and function is impaired. Using a stereo-camera sensor system, 3D reachable workspace envelope surface area normalized to an individual's arm length (relative surface area: RSA) to allow comparison between subjects was determined for 20 healthy controls and 9 individuals with varying degrees of upper extremity dysfunction due to neuromuscular conditions. All study subjects were classified based on Brooke upper extremity function scale. Right and left upper extremity reachable workspaces were determined based on three repeated measures. The RSAs for each frontal hemi-sphere quadrant and total reachable workspaces were determined with and without loading condition (500 gram wrist weight). Data were analyzed for assessment of the developed system and validity, reliability, and sensitivity to change of the reachable workspace outcome. The mean total RSAs of the reachable workspace for the healthy controls and individuals with NMD were significantly different (0.586 ± 0.085 and 0.299 ± 0.198 respectively; p<0.001). All quadrant RSAs were reduced for individuals with NMDs compared to the healthy controls and these reductions correlated with reduced upper limb function as measured by Brooke grade. The upper quadrants of reachable workspace (above the shoulder level) demonstrated greatest reductions in RSA among subjects with progressive severity in upper extremity impairment. Evaluation of the developed outcomes system with the Bland-Altman method demonstrated narrow 95% limits of agreement (LOA) around zero indicating high reliability. In addition, the intraclass correlation coefficient (ICC) was 0.97. Comparison of the reachable workspace with and without loading condition (wrist weight) showed significantly greater RSA reduction in the NMD group than the control group (p<0.012), with most of the workspace reduction occurring in the ipsilateral upper quadrant relative to the tested arm (p<0.001). Reduction in reachable workspace due to wrist weight was most notable in those subjects with NMD with marginal strength reserve and moderate degree of impairment (Brooke = 2) rather than individuals with mild upper extremity impairment (Brooke = 1) or individuals who were more severely impaired (Brooke =3). The developed reachable workspace evaluation method using scalable 3D vision technology appears promising as an outcome measure system for clinical studies. A rationally-designed combination of upper extremity outcome measures including a region-specific global upper extremity outcome measure, such as the reachable workspace, complemented by targeted disease- or function-specific endpoints, may be optimal for future clinical efficacy trials.
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