Authors (including presenting author) :
Chiu AYY(1), Choi SWC(1), Hui CCK (1), Chan SHS (2), Lee SL (2)
Affiliation :
(1)Physiotherapy Department , The Duchess of Kent Children's Hospital at Sandy Bay
(2) Department of Paediatrics and Aldolescent Medicine, the University of Hong Kong , Queen Mary Hospital , HKSAR
Introduction :
Duchenne Muscular Dystrophy (DMD) due to gene mutation has progressive weakness affecting the skeletal, heart and respiratory muscles. The recent rapid development of approved disease-modifying therapies and ongoing clinical trials highlight the importance of using a reliable and valid outcome measure to monitor treatment response. The Performance of the Upper Limb Module (PUL) for DMD was first introduced to assess Chinese DMD patients in Duchess of Kent Children’s Hospital at Sandy Bay since 2016.
Objectives :
To examine the effectives of PUL for DMD, we assessed its inter-and -intra-rater reliability, group validity and construct validity with its correlation with the pulmonary function and motor scales performance.
Methodology :
Total 35 DMD patients under the care of Paediatric Neuromuscular Disorder Program and Pulmonary Rehabilitation in DKCH were included in our study from 2016 to 2019. Reliability assessment were done on 23 DMD patients by three raters rating one session of video-recorded performance, and also at one-month interval. Known group validity was assessed comparing performance of patients of ambulatory and non-ambulatory status. Construct validity was assessed in 25 DMD patients correlated their PUL performance with their lung function and motor scales respectively.
Result & Outcome :
We found PUL was easy to administer with no expensive equipment required and could be completed within 30 minutes. There were excellent intra-andinter-rater reliability for all PUL scores (Intra-correlation coefficient (ICC) >0.75 and good internal consistency for all level scores with Cronbach’s alpha >0.8.) The known group’s validity was good (ROC >0.7). The PUL of DMD was also found significant correlated with the pulmonary forced vital capacity (FVC % predicted) (r=0.87) and Hammersmith motor scale (r=0.84) performance. In conclusion, we found PUL for DMD is reliable, valid and easy-to-use, and recommend its use in disease monitoring and as an outcome measure for both ambulatory and non-ambulatory DMD patients.
