TY - JOUR
T1 - Health-related quality of life and functional changes in DMD: A 12-month longitudinal cohort study
AU - Messina, Sonia
AU - Vita, Gian Luca
AU - Sframeli, Maria
AU - Mondello, Stefania
AU - Mazzone, Elena Stacy
AU - D'Amico, Adele
AU - Berardinelli, Angela
AU - La Rosa, Matteo
AU - Bruno, Claudio
AU - Distefano, Maria Grazia
AU - Baranello, Giovanni
AU - Barcellona, Costanza
AU - Scutifero, Marianna
AU - Marcato, Sonia
AU - Palmieri, Arianna
AU - Politano, Luisa
AU - Morandi, Lucia
AU - Mongini, Tiziana
AU - Pegoraro, Elena
AU - D'Angelo, Maria Grazia
AU - Pane, Marika
AU - Rodolico, Carmelo
AU - Minetti, Carlo
AU - Bertini, Enrico
AU - Bertini, Enrico Silvio
AU - Vita, Giuseppe
AU - Mercuri, Eugenio Maria
PY - 2016
Y1 - 2016
N2 - 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.
AB - 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.
KW - Adolescent
KW - Child
KW - Child, Preschool
KW - Duchenne muscular dystrophy
KW - Fatigue
KW - Genetics (clinical)
KW - Humans
KW - Longitudinal Studies
KW - Male
KW - Medicine (all)
KW - Muscular Dystrophy, Duchenne
KW - Neurology
KW - Neurology (clinical)
KW - Outcome measures
KW - Pediatrics, Perinatology and Child Health
KW - PedsQL™
KW - Prospective Studies
KW - Quality of Life
KW - Quality of life
KW - Severity of Illness Index
KW - Surveys and Questionnaires
KW - Adolescent
KW - Child
KW - Child, Preschool
KW - Duchenne muscular dystrophy
KW - Fatigue
KW - Genetics (clinical)
KW - Humans
KW - Longitudinal Studies
KW - Male
KW - Medicine (all)
KW - Muscular Dystrophy, Duchenne
KW - Neurology
KW - Neurology (clinical)
KW - Outcome measures
KW - Pediatrics, Perinatology and Child Health
KW - PedsQL™
KW - Prospective Studies
KW - Quality of Life
KW - Quality of life
KW - Severity of Illness Index
KW - Surveys and Questionnaires
UR - http://hdl.handle.net/10807/92051
UR - http://www.elsevier.com/locate/nmd
U2 - 10.1016/j.nmd.2016.01.003
DO - 10.1016/j.nmd.2016.01.003
M3 - Article
SN - 0960-8966
VL - 26
SP - 189
EP - 196
JO - Neuromuscular Disorders
JF - Neuromuscular Disorders
ER -