Résumé :
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Communication n° 196 Introduction : One of the major goals both in the general management of Neuromuscular Disorders (NMDs) and in therapeutic trials is increasingly recognized as the improvement or at least prevention of worsening of heath-related quality of life (HRQOL). However there are insufficient data regarding the outcome of these conditions and the correlation between functional impairment, health status and quality of patients' life, especially in children. There exist very few validated instruments to measure HRQOL in children; these methods must be child-centred and employ subjective self-reporting techniques. No such instrument, validated in an Italian paediatric population, currently exists. Objectives : This is a prospective multidimensional multicentric study aiming to develop and validate a new self-administered questionnaire for the study of HRQOL in children with NMDs. Method : We planned to validate the questionnaire in a population of almost 145 subjects aged 5-12 years and affected by genetic NMDs. They will be recruited from 6 different Italian centres (Pavia, Padova, Torino, Bologna, Roma Policlinico Gemelli, Bambin Gesù Hospital-Rome). The Mario Negri Institute (Milan) will be responsible for statistical analysis and data interpretation. Preliminary Results : The Coordinator Centre of the project created, using a multidisciplinary step by step approach, a first version of a new and original questionnaire, which measures QOL using a multidimensional, child-centred approach, language appropriate to the age of the patients investigated, and including a section for patients with NMDs. The questionnaire uses a series of pictures which illustrate moments previously identified as significative and specific for children affected by NMDs. In each item, the head is always drawn as a plain circle and the youngster has to pick a sticker showing the facial expression (pleasant, unpleasant or neutral) that best represents his feelings in the particular situation presented. We called this instrument Strips Of Life with Emoticons (SOLE) Questionnaire. The recruitment process has been going on from January 2005; an interim analysis will be undertaken after three months of the beginning. Conclusions : This study could provide the basis to create an ad-hoc instrument to measure QOL in children with NMDs.
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