VALIDATION OF A NEUROMUSCULAR INDIVIDUALIZED QUALITY OF LIFE MEASURE IN ITALY (INQoL ITALIA)

A specific and adequate tool to measure the effects of a specific intervention is missing in adult muscle diseases. This has been a limiting factor in many clinical trials conducted so far because the cost-benefits and relative side-effects have not been clearly weighed in the patients' perspective of the impact on the quality of life. Ad adequate tool in this sense would provide the necessary information to monitor patient management in the daily clinical setting of in- and outpatients but it would also provide a good instrument in clinical trials. Moreover, the identification of specific areas rated as important for the patients would allow to allocate human and financial resources in a more targeted way. Over the past year the Applicants have collaborated with Dr Michael Rose from King's College, London at the translation of an individualized quality of life measure, called INQoL, which has been validated in the UK and which has been demonstrated to be reproducible and reliable. The speficic aims of this project are to validate INQoL in Italy, to further extend this instrument to other muscle disorders like muscle channelopathies and spinal muscular atrophies and to explore the sensitivity to change of this tool by applying the questionnaire to a subset of patients who will enter a planned clinical trial (eg periodic paralysis treatment trial). The proposed study is a 3-year study involving 7 Center all over Italy and also involving external collaborators.