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

Abstract

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.

Instruments to assess upper‐limb function in children and adolescents with neuromuscular diseases: a systematic review

“…The instruments with the highest number of measurement properties tested were the MFM scale and PUL. 35,49,[66][67][68][69][70][71][72][73][74] In most instruments, measurement properties were classified as sufficient according to the criterion of good measurement. Content validity, reliability, structural validity, and hypothesis testing for construct validity were the properties that met the least quality criteria.…”

“…16,35,44,47,53,54,63,65,67,69,70 Hypothesis testing for construct and content validity was evaluated in seven instruments and structural validity was evaluated in six. 16,35,44,[47][48][49]53,54,57,63,69,[70][71][72] Responsiveness was measured in only three instruments 50,52,73,74 and examination of criterion validity was made in only two. 67,69 Measurement error was reported in only one instrument 69 and none of the five studies that translated and carried out cross-cultural adaptation of the instruments reported crosscultural validity and/or measurement invariance.…”

“…60,62 Thus, in the 10 instruments that were assessed, reliability was examined in nine instruments 16,27,35,44,48,49,54,63,[65][66][67][68][69] and internal consistency was tested in eight instruments. 16,35,44,47,53,54,63,65,67,69,70 Hypothesis testing for construct and content validity was evaluated in seven instruments and structural validity was evaluated in six. 16,35,44,[47][48][49]53,54,57,63,69,[70][71][72] Responsiveness was measured in only three instruments 50,52,73,74 and examination of criterion validity was made in only two.…”

“…23,24 Of the 12 instruments included in the review, the CUE and WBQ instruments lacked measurement properties specific to patients with NMDs. 60,62 Thus, in the 10 instruments that were assessed, reliability was examined in nine instruments 16,27,35,44,48,49,54,63,[65][66][67][68][69] and internal consistency was tested in eight instruments. 16,35,44,47,53,54,63,65,67,69,70 Hypothesis testing for construct and content validity was evaluated in seven instruments and structural validity was evaluated in six.…”

“…16,35,44,[47][48][49]53,54,57,63,69,[70][71][72] Responsiveness was measured in only three instruments 50,52,73,74 and examination of criterion validity was made in only two. 67,69 Measurement error was reported in only one instrument 69 and none of the five studies that translated and carried out cross-cultural adaptation of the instruments reported crosscultural validity and/or measurement invariance. The instruments with the highest number of measurement properties tested were the MFM scale and PUL.…”