T.P.11: Longitudinal assessment of Upper Limb function in DMD patients: 12 month changes

Abstract

While there have been considerable advances for outcome measures in ambulant children with Duchenne Muscular Dystrophy (DMD), no prospective longitudinal study has so far been devoted to the assessment of upper limbs. This information appears to be relevant for a better understanding of the disease progression in non ambulant patients. As a result of an international effort, a new tool, the Performance of Upper Limb (PUL) was specifically designed to assess upper limb function in DMD boys. The purpose of the PUL is to assess changes that occurs in motor performance of the upper limb over time both in ambulant and non-ambulant DMD boys and adults. A recent cross sectional study has demonstrated the ability of the PUL to assess a wide range of activities. The aim of the present study was to use the PUL in a cohort of DMD children and young adults to assess the range of findings at different ages and their changes over 12months. The results showed a progressive deterioration of scores with age, with early involvement of the proximal muscles that was more obvious after the age of 10years in the proximal domain and after the age of 15 in the more distal ones. The PUL Scale appears to be a useful tool for upper limb motor disease assessment in DMD ambulant and non-ambulant patients, that may be used in clinical trials and in clinical settings.