Confirmatory validation of the french version of the Duchenne Muscular Dystrophy module of the pediatric quality of life inventory (PedsQLTM3.0DMDfv)

Abstract Duchenne Muscular Dystrophy (DMD) is a neuromuscular disease that inevitably leads to total loss of autonomy. The new therapeutic strategies aim to both improve survival and optimise quality of life. Evaluating quality of life is nevertheless a major challenge. No DMD-specific quality of li...

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Main Authors: Elisabeth Wallach (Author), Virginie Ehlinger (Author), Maelle Biotteau (Author), Ulrike Walther-Louvier (Author), Yann Péréon (Author), Carole Vuillerot (Author), Stephanie Fontaine (Author), Pascal Sabouraud (Author), Caroline Espil-Taris (Author), Jean-Marie Cuisset (Author), Vincent Laugel (Author), Eloïse Baudou (Author), Catherine Arnaud (Author), Claude Cances (Author)
Format: Book
Published: BMC, 2023-11-01T00:00:00Z.
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Summary:Abstract Duchenne Muscular Dystrophy (DMD) is a neuromuscular disease that inevitably leads to total loss of autonomy. The new therapeutic strategies aim to both improve survival and optimise quality of life. Evaluating quality of life is nevertheless a major challenge. No DMD-specific quality of life scale to exists in French. We therefore produced a French translation of the English Duchenne Muscular Dystrophy module of the Pediatric Quality of Life Inventory (PedsQLTMDMD) following international recommendations. The study objective was to carry out a confirmatory validation of the French version of the PedsQLTMDMD for paediatric patients with DMD, using French multicentre descriptive cross-sectional data. The sample consisted of 107 patients. Internal consistency was acceptable for proxy-assessments, with Cronbach's alpha coefficients above 0.70, except for the Treatment dimension. For self-assessments, internal consistency was acceptable only for the Daily Activities dimension. Our results showed poor metric qualities for the French version of the PedsQLTMDMD based on a sample of about 100 children, but these results remained consistent with those of the original validation. This confirms the interest of its use in clinical practice.
Item Description:10.1186/s12887-023-04153-4
1471-2431