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 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'Angelo, Marika Pane, Carmelo Rodolico, Carlo Minetti, Enrico Bertini, Giuseppe Vita, Eugenio Mercuri, Sonia Messina, Gian Luca Vita, Maria Sframeli, Stefania Mondello, Elena 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'Angelo, Marika Pane, Carmelo Rodolico, Carlo Minetti, Enrico Bertini, Giuseppe Vita, Eugenio Mercuri

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(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.

Keywords: Duchenne muscular dystrophy; Outcome measures; PedsQL(TM); Quality of life.

Copyright © 2016 The Authors. Published by Elsevier B.V. All rights reserved.

Figures

Fig. 1
Fig. 1
Distribution of individual 12 month changes of PedsQLTM Child Self-Report Generic Core Scales scores versus 6MWT (A), NSAA (B), 10 meter timed walk/run test (10 meter) (C) and Gowers test (D) according to age (≤ or >7 years).

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