Résumé :
|
The EK scale was developed as a clinical tool to assess functional ability and to determine the need for and impact of intervention in the non-ambulatory stages of Duchenne muscular dystrophy (DMD) and in spinal muscular atrophy (SMA). EK is a composite scale consisting of 10 items that are chosen to be of clinically relevance to the non-ambulatory stages of the diseases. Each item is divided into four grades, scored 0-3 and the sum of scores of the items is the overall functional level. In studies of reliability, validity and sensitivity published in the years 1995-2002 the EK scale was shown to be able to: o discriminate between individuals o predict the need of assisted ventilation in DMD o detect change of function as a result of a) the natural history of the two diseases or b) scoliosis surgery and spinal bracing However, EK was not as sensitive to change over time in individuals with SMA as in individuals with DMD and advances in drug treatments mean there is greater need for robust sensitive tools that measure life events relevant to individuals with neuromuscular disease. The purpose of the present study was to o Improve the discriminatory power of EK in the non-ambulatory stages of SMA o Increase the number of clinically meaningful items Methods and results An international focus group of 10 experts identified 10 new items representing clinically meaningful aspects which were characteristic for the non-ambulatory stages of SMA. After having evaluated these 10 new items in 58 patients from Denmark, Italy and the UK they were reduced to seven and the manual revised. In a second test of 81 non-ambulatory patients from the three countries, aged 2-70 yrs the seven new items were tested for discriminatory and content validity in conjunction with the original scale. Preliminary data from this international, multi-centre study supported by Treat NMD will be presented.
|