Duchenne Muscular Dystrophy Clinical Trial
Official title:
The Validation Process for Confirmation of the French Version of the Pediatric Quality of Life Inventory (PedsQLTM) 3.0 Duchenne Muscular Dystrophy Module.
There isn't specific Health related quality of life measure for children with DMD in French. The aim of this study is to validate the French version of the Pediatric Quality of Life Inventory 3.0 Duchenne Muscular Dystrophy module with a multicentric study. The investigators will evaluate the following psychometric properties : convergent validity, internal validity, inter-rater reliability. The investigators would like to be able to use this scientific tool in future clinical trials.
The Duchenne Muscular Dystrophy, the commonest form of dystrophy, is an X-linked, recessive
neuromuscular disease, in which there is an absence of the protein dystrophin. This chronic
and progressive disease leads to an inevitable loss of autonomy (muscle weakness, respiratory
and cardiac failure). With better multidisciplinary care, life expectancy has increased but
also morbidity. From now one, the evaluation of the quality of life of children with DMD is
necessary in therapeutic trials.
Given the specificities of the disease, it seems appropriate to have a specific scale. In the
literature there isn't quality of life scale specific to Duchenne Muscular Dystrophy in
French version. The only specific scale that exists is the specific module PedsQLTM DMD that
was validated in English version in 2012. This scale is relevant for assessing the quality of
life in clinical trials and in daily clinical practice given its psychometric properties
(good internal consistency close to 0.8).The main hypothesis that we formulate is to validate
the French translation of the pediatric module of Duchenne Muscular Dystrophy of the PedsQL ™
3.0 scale.
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