P.2.4 Upper extremity reachable workspace evaluation in DMD using Kinect (original) (raw)
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AIM Clinician-reported outcome instruments such as the North Star Ambulatory Assessment (NSAA) need to be able to detect clinically important change to be suitable for clinical trials. However, in Duchenne muscular dystrophy (DMD), identifying changes in function is not straightforward. In this study, we use Rasch-transformed data to examine the responsiveness and minimal important difference (MID) of the NSAA in males with DMD receiving different corticosteroid regimes. METHOD NSAA data were examined from 198 males (mean age at assessment was 8y 6mo [SD 2y 6mo] range 4y–18y; 805 assessments). Responsiveness was assessed using mean score changes (using Rasch-transformed data) between adjacent pairs of age groups, pairwise squared t-values from paired samples t-tests, and an effect size calculation. The MID was assessed using the effect size calculation and 0.5 standard deviation (SD) of mean score differences. RESULTS Our findings revealed a difference in change scores over time between the two corticosteroid regimes. Mean NSAA person estimates were higher in the daily prednisolone group. The mean MID (0.5 SD) was 8.8 and 6.9 for the daily group and intermittent group respectively. INTERPRETATION This study, based on Rasch-transformed NSAA data, provides an initial basis for the interpretation of clinical change in DMD over time and between corticosteroid regimes. Our proposed MIDs can be mapped back to differences in specific item content across the range of the NSAA.
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PLOS ONE
The North Star ambulatory assessment (NSAA) is a functional motor outcome measure in Duchenne muscular dystrophy (DMD), widely used in clinical trials and natural history studies, as well as in clinical practice. However, little has been reported on the minimal clinically important difference (MCID) of the NSAA. The lack of established MCID estimates for NSAA presents challenges in interpreting the significance of the results of this outcome measure in clinical trials, natural history studies and clinical practice. Combining statistical approaches and patient perspectives, this study estimated MCID for NSAA using distribution-based estimates of 1/3 standard deviation (SD) and standard error of measurement (SEM), an anchor-based approach, with six-minute walk distance (6MWD) as the anchor, and evaluation of patient and parent perception using participant-tailored questionnaires. The MCID for NSAA in boys with DMD aged 7 to 10 years based on 1/3 SD ranged from 2.3–2.9 points, and that...
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