Performance of Upper Limb module for Duchenne muscular dystrophy

Coratti, Giorgia; Mazzone, Elena; Klingels, Katrijn; James, M.; Pane, Marika; Straub, Volker; Goemans, N.; Ricotti, Valeria; Muntoni, Francesco; Ridout, Deborah; Selby, V.

doi:10.1111/dmcn.14361

Cited by 55 publications

(78 citation statements)

References 14 publications

(28 reference statements)

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“…Finally, the PUL module demonstrated to have inter-rater reliability, as demonstrated by ten independent assessors. The comparability and the possibility of using PUL 1.2 and 2.0 versions in longitudinal studies have been shown [37]. This study demonstrated the robustness and reliability of the PUL module and, as a consequence, this clinical scale can be suggested as a primary outcome measure in experimental clinical trials involving patients diagnosed with muscular dystrophy other than DMD, such as LGMD and BMD.…”

Section: Discussionmentioning

confidence: 63%

“…PUL module has the advantage of providing evidence on the functional evolution over time of the disease from distal to proximal levels. After the beginning of this study, a novel version of the PUL module (PUL 2.0) has been validated and shared with the scientific community [13,36,37]. PUL 2.0 is constituted by 22 items, and some items of the previous version were deleted (i.e., items B, I, K, U, and V).…”

Section: Discussionmentioning

confidence: 99%

“…In DMD, the PUL module has been widely studied and used to evaluate changes in the muscular function of upper limbs along with time [36,37]. However, in LGMD and BMD, only Artilhiero studied some patients to investigate the relationship between the PUL module and Jebsen-Taylor Test to assess and monitor upper limb function progression in patients with different MD types [21].…”

Section: Discussionmentioning

confidence: 99%

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Test-retest reliability of the Performance of Upper Limb (PUL) module for muscular dystrophy patients

et al. 2020

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The Performance of the Upper Limb (PUL) module is an externally-assessed clinical scale, initially designed for the Duchenne muscular dystrophy population. It provides an upper extremity functional score suitable for both weaker ambulatory and non-ambulatory phases up to the severely impaired patients. It is capable of characterizing overall progression and severity of disease and of tracking the stereotypical proximal-to-distal progressive loss of upper limb function in muscular dystrophy. Since the PUL module has been validated only with Duchenne patients, its use also for Becker and Limb-Girdle muscular dystrophy patients has been here evaluated, to verify its reliability and extend its use. In particular, two different assessors performed this scale on 32 dystrophic subjects in two consecutive days. The results showed that the PUL module has high reliability, both absolute and relative, based on the calculation of Pearson's r (0.9942), Intraclass Correlation Coefficient (0.9943), Standard Error of Measurement (1.36), Minimum Detectable Change (3.77), and Coefficient of Variation (3%). The Minimum Detectable Change, in particular, can be used in clinical trials to perform a comprehensive longitudinal evaluation of the effects of interventions with the lapse of time. According to this analysis, an intervention is effective if the difference in the PUL score between subsequent evaluation points is equal or higher than 4 points; otherwise, the observed effect is not relevant. Inter-rater reliability with ten different assessors was evaluated, and it has been demonstrated that deviation from the mean is lower than calculated Minimum Detectable Change. The present work provides evidence that the PUL module is a reliable and valid instrument for measuring upper limb ability in people with different forms of muscular dystrophy. Therefore, the PUL module might be extended to other pathologies and reliably used in multicenter settings.

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Section: Discussionmentioning

confidence: 63%

Section: Discussionmentioning

confidence: 99%

Section: Discussionmentioning

confidence: 99%

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Test-retest reliability of the Performance of Upper Limb (PUL) module for muscular dystrophy patients

et al. 2020

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“…The current PUL scale is of version 1.3. This year in 2019, an updated version 2.0 has been established for patients with DMD [10] and the study con rmed that both PUL versions measure the same construct so the current study ndings can be generalized to the use of PUL version 2.0 in addition we understand that the update version could be better in detecting the serial changes when checked in 12 months or 24 months interval.…”

Section: Limitations and Future Research Directionsmentioning

confidence: 57%

“…The development of Performance of the Upper Limb module (PUL) for Duchenne muscular dystrophy [8,9] was rst established in 2013. There is also an updated version of PUL 2.0 [10].…”

Section: Introductionmentioning

confidence: 99%

Performance of the Upper Limb Module—A Reliable and Valid Evaluation for Chinese Patients with Duchenne Muscular Dystrophy

Chiu

Hui

et al. 2020

Preprint

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Background Duchenne muscular dystrophy is a genetic disease leading to progressive muscle weakness and degeneration. Effective assessment tool is needed to allow monitoring of progress to guide the management. This study assessed the reliability and validity of the Performance of Upper Limb (PUL) Module when used in patients with Duchenne Muscular Dystrophy (DMD). MethodsTotal thirty-three Chinese DMD patients were included. Twenty-five video-recorded PUL Module version 1.3 assessments were performed for the recruited patients with three raters evaluated the same recorded video for inter-rater reliability and evaluated the same performance one month later for intra-rater reliability. Construct validity was assessed correlating the PUL Module scores with the patients’ age, their forced vital capacity (N=25) and their Hammersmith motor scale scores (N=25) performed on the same day. ResultsThe intra-rater and inter-rater reliability (ICC 0.92 - 0.99), internal consistency (Cronbach’s alpha 0.97 - 0.99) and known groups validity (AUC 0.97) of PUL module were excellent. PUL was negatively correlated with age (r = -0.912), and positively correlated with the forced vital capacity (r = 0.87) and the Hammersmith motor scale (r = 0.84). The findings confirm the high reliability and validity of PUL module, and its high clinical relevancy in monitoring the deteriorating upper limb motor performance that strongly correlated with the lung function and generalized motor performance as age increased in DMD. ConclusionThis first study of PUL module in Chinese patients with DMD confirmed that it is a reliable valid tool to monitor clinical progress and outcome for DMD.

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Upper limb disease evolution in exon 53 skipping eligible patients with Duchenne muscular dystrophy

Lilien

Reyngoudt

Seferian

et al. 2021

Ann Clin Transl Neurol

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Objective To understand the natural disease upper limb progression over 3 years of ambulatory and non‐ambulatory patients with Duchenne muscular dystrophy (DMD) using functional assessments and quantitative magnetic resonance imaging (MRI) and to exploratively identify prognostic factors. Methods Forty boys with DMD (22 non‐ambulatory and 18 ambulatory) with deletions in dystrophin that make them eligible for exon 53‐skipping therapy were included. Clinical assessments, including Brooke score, motor function measure (MFM), hand grip and key pinch strength, and upper limb distal coordination and endurance (MoviPlate), were performed every 6 months and quantitative MRI of fat fraction (FF) and lean muscle cross sectional area (flexor and extensor muscles) were performed yearly. Results In the whole population, there were strong nonlinear correlations between outcome measures. In non‐ambulatory patients, annual changes over the course of 3 years were detected with high sensitivity standard response mean (|SRM| ≥0.8) for quantitative MRI‐based FF, hand grip and key pinch, and MFM. Boys who presented with a FF<20% and a grip strength >27% were able to bring a glass to their mouth and retained this ability in the following 3 years. Ambulatory patients with grip strength >35% of predicted value and FF <10% retained ambulation 3 years later. Interpretation We demonstrate that continuous decline in upper limb strength, function, and MRI measured muscle structure can be reliably measured in ambulatory and non‐ambulatory boys with DMD with high SRM and strong correlations between outcomes. Our results suggest that a combination of grip strength and FF can be used to predict important motor milestones.

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Performance of Upper Limb module for Duchenne muscular dystrophy

Cited by 55 publications

References 14 publications

Test-retest reliability of the Performance of Upper Limb (PUL) module for muscular dystrophy patients

Test-retest reliability of the Performance of Upper Limb (PUL) module for muscular dystrophy patients

Performance of the Upper Limb Module—A Reliable and Valid Evaluation for Chinese Patients with Duchenne Muscular Dystrophy

Upper limb disease evolution in exon 53 skipping eligible patients with Duchenne muscular dystrophy

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