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

S. Messina, Gian Luca Vita, Maria Sframeli, Stefania Mondello, E. S. Mazzone, Adele D'Amico, Angela Lucia Berardinelli, Matteo La Rosa, Claudio Bruno, Maria Grazia Distefano, Giovanni Baranello, Costanza Barcellona, Marianna Scutifero, Sonia Marcato, Arianna Palmieri, L. Politano, Lucia Morandi, T. Mongini, E. Pegoraro, Maria Grazia D'AngeloM. Pane, C. Rodolico, Carlo Minetti, Enrico Silvio Bertini, G. Vita, E. Mercuri

Research output: Contribution to journalArticle

Abstract

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.

Original languageEnglish
Pages (from-to)189-196
Number of pages8
JournalNeuromuscular Disorders
Volume26
Issue number3
DOIs
Publication statusPublished - Mar 1 2016

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Keywords

  • Duchenne muscular dystrophy
  • Outcome measures
  • PedsQL™
  • Quality of life

ASJC Scopus subject areas

  • Clinical Neurology
  • Pediatrics, Perinatology, and Child Health
  • Genetics(clinical)
  • Neurology

Cite this

Messina, S., Vita, G. L., Sframeli, M., Mondello, S., Mazzone, E. S., D'Amico, A., Berardinelli, A. L., La Rosa, M., Bruno, C., Distefano, M. G., Baranello, G., Barcellona, C., Scutifero, M., Marcato, S., Palmieri, A., Politano, L., Morandi, L., Mongini, T., Pegoraro, E., ... Mercuri, E. (2016). Health-related quality of life and functional changes in DMD: A 12-month longitudinal cohort study. Neuromuscular Disorders, 26(3), 189-196. https://doi.org/10.1016/j.nmd.2016.01.003