Health-related quality of life and functional changes in DMD: A 12-month longitudinal cohort study

• DOI: 10.1016/j.nmd.2016.01.003
• Publication Date: 2016-02-02T15:31:02Z
• Authors: Sonia Messina; Gian Luca Vita; Maria Sframeli; Stefania Mondello; Elena Mazzone; Adele D'Amico; Angela Berardinelli; Matteo La Rosa; Claudio Bruno; Maria Grazia Dist...; Giovanni Baranello; Costanza Barcellona; Marianna Scutifero; Sonia Marcato; Arianna Palmieri; Luisa Politano; Lucia Morandi; Tiziana Mongini; Elena Pegoraro; Maria Grazia D'An...; Marika Pane; Carmelo Rodolico; Carlo Minetti; Enrico Bertini; Giuseppe Vita; Eugenio Mercuri
• Keywords: Duchenne muscular dystrophy; Quality of life; Male; PedsQLTM; Duchenne muscular dystrophy; Outcome measures; PedsQL(TM); Quality of life; Adolescent; Child; Child, Preschool; Fatigue; Humans; Longitudinal Studies; Male; Muscular Dystrophy, Duchenne; Prospective Studies; Severity of Illness Index; Surveys and Questionnaires; Quality of Life; Adolescent; Duchenne muscular dystrophy; Outcome measures; PedsQLTM; Quality of life; Adolescent; Child; Child, Preschool; Fatigue; Humans; Longitudinal Studies; Male; Muscular Dystrophy, Duchenne; Prospective Studies; Severity of Illness Index; Surveys and Questionnaires; Quality of Life; Pediatrics, Perinatology and Child Health; Neurology; Neurology (clinical); Genetics (clinical); Clinical Neurology; Duchenne muscular dystrophy; Outcome measures; PedsQLTM; Quality of life; Neurology (clinical); Pediatrics, Perinatology and Child Health; Genetics (clinical); Neurology; Outcome measures; Severity of Illness Index; Article; 03 medical and health sciences; 0302 clinical medicine; Surveys and Questionnaires; Humans; Genetics(clinical); Pediatrics, Perinatology, and Child Health; Longitudinal Studies; Prospective Studies; Child; Fatigue; Duchenne muscular dystrophy; Outcome measures; PedsQL(TM); Quality of life; Muscular Dystrophy, Duchenne; Neurology; Child, Preschool; Duchenne muscular dystrophy; Outcome measures; PedsQLTM; Quality of life; Adolescent; Child; Child, Preschool; Fatigue; Humans; Longitudinal Studies; Male; Muscular Dystrophy, Duchenne; Prospective Studies; Severity of Illness Index; Surveys and Questionnaires; Quality of Life; Pediatrics, Perinatology and Child Health; Medicine (all); Neurology; Neurology (clinical); Genetics (clinical); Quality of Life

In Duchenne muscular dystrophy (DMD) little has been reported on the association between clinical outcome measures and patient health-related quality of life (HRQOL) tools. Our study evaluated the relationship between 12 month changes on the Generic Core Scales (GCS), the Multidimensional Fatigue Scale and the Neuromuscular Module of the PedsQL(TM) with several outcome measures (6 minute walk test, North Star Ambulatory Assessment and timed items) in ambulatory DMD. Ninety-eight ambulatory DMD in a multicentric setting were included in the study. At baseline, the PedsQL(TM) inventories correlated with almost all the functional measures On the Child Self-Report there was a significant decrease between baseline and 12 months on the PedsQL(TM) GCS and its first domain, in parallel with the decrement in the functional outcome measures. Correlation between the 12 month changes on the PedsQL(TM) inventories and functional measures were almost all negligible. Similar results were obtained on the Parent Proxy-Report. In conclusion, PedsQL(TM) correlates with the level of impairment at baseline, but this does not hold true when 12 month changes are considered. Further studies comparing different tools are needed to better elucidate the complexity of the relationship between HRQOL and functional performances.