Résumé :
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There are several motivations for the increasing interest in assessing quality of life (QoL) in chronic long term conditions such as neuromuscular disease (NMD). Many NMDs are not treatable, and even those that are may have persistent morbidity which is not adequately captured by traditional disease measures. In clinical trials there has been the hope that QoL measures may allow participants to express their perspectives on such issues as the benefit-to-side-effect ratio for the treatment, or the importance of any change in trial outcome measure. In order to realise these expectations it is important to understand the relationship between QoL and NMD severity and how this may change, with or without interventions, over time. In adults with NMD QoL has been measured using generic QoL measures but there is now a NMD specific QoL measure (Individualised NM QoL (INQoL )) that may have advantages over the generic measures. In children with NMD generic QoL measures may soon be supplemented by a muscle disease specific add on module. QoL "physical" sub-domains are more affected by NMD than are "mental" sub-domains. Under-recognised symptoms such as pain or fatigue may be particularly important in determining physical aspect of QoL. Mental sub domains of QoL may be more influenced by psycho-social factors. While mental sub-domains of QoL may be less affected they still contribute to the overall reduction in QoL. The psycho-social factors that influence QoL may do so either directly or by mediating the effects of disease severity on QoL. The complexity of the relationship between NMD severity and QoL may hinder the use of QoL as a clinical trial measure. Despite this, even if we cannot treat the NMD itself, opportunity exists to improve QoL by treating hitherto under-recognised symptoms or by altering the psycho-social factors that influence QoL.
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