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

Sonia Messina, Gian Luca Vita, Maria Sframeli, Stefania Mondello, Elena Stacy Mazzone, Adele D'Amico, Angela Berardinelli, Matteo La Rosa, Claudio Bruno, Maria Grazia Distefano, Giovanni Baranello, Costanza Barcellona, Marianna Scutifero, Sonia Marcato, Arianna Palmieri, Luisa Politano, Lucia Morandi, Tiziana Mongini, Elena Pegoraro, Maria Grazia D'AngeloMarika Pane, Carmelo Rodolico, Carlo Minetti, Enrico Bertini, Enrico Silvio Bertini, Giuseppe Vita, Eugenio Maria Mercuri*

*Autore corrispondente per questo lavoro

Risultato della ricerca: Contributo in rivistaArticolo in rivista

21 Citazioni (Scopus)


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™ 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™ 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™ GCS and its first domain, in parallel with the decrement in the functional outcome measures. Correlation between the 12 month changes on the PedsQL™ inventories and functional measures were almost all negligible. Similar results were obtained on the Parent Proxy-Report.In conclusion, PedsQL™ 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.
Lingua originaleEnglish
pagine (da-a)189-196
Numero di pagine8
RivistaNeuromuscular Disorders
Stato di pubblicazionePubblicato - 2016


  • Adolescent
  • Child
  • Child, Preschool
  • Duchenne muscular dystrophy
  • Fatigue
  • Genetics (clinical)
  • Humans
  • Longitudinal Studies
  • Male
  • Medicine (all)
  • Muscular Dystrophy, Duchenne
  • Neurology
  • Neurology (clinical)
  • Outcome measures
  • Pediatrics, Perinatology and Child Health
  • PedsQL™
  • Prospective Studies
  • Quality of Life
  • Quality of life
  • Severity of Illness Index
  • Surveys and Questionnaires


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