title: Health-related quality of life and functional changes in DMD: A 12-month longitudinal cohort study.
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.
authors.affiliation: Department of Neurosciences, University of Messina, Messina, Italy; Nemo Sud Clinical Center for Neuromuscular Diseases, Messina, Italy.
authors.forename: Sonia
authors.initials: S
authors.lastname: Messina
authors.affiliation: Nemo Sud Clinical Center for Neuromuscular Diseases, Messina, Italy.
authors.forename: Gian Luca
authors.initials: GL
authors.lastname: Vita
authors.affiliation: Nemo Sud Clinical Center for Neuromuscular Diseases, Messina, Italy.
authors.forename: Maria
authors.initials: M
authors.lastname: Sframeli
authors.affiliation: Department of Neurosciences, University of Messina, Messina, Italy.
authors.forename: Stefania
authors.initials: S
authors.lastname: Mondello
authors.affiliation: Department of Paediatric Neurology, Catholic University, Rome, Italy.
authors.forename: Elena
authors.initials: E
authors.lastname: Mazzone
authors.affiliation: Unit of Neuromuscular and Neurodegenerative Disorders, Laboratory of Molecular Medicine, Bambino Gesu' Children's Research Hospital, Rome, Italy.
authors.forename: Adele
authors.initials: A
authors.lastname: D'Amico
authors.affiliation: IRCCS "C.Mondino" Institute, Unit of Child Neuropsychiatry, University of Pavia, Pavia, Italy.
authors.forename: Angela
authors.initials: A
authors.lastname: Berardinelli
authors.affiliation: Department of Neurosciences, University of Messina, Messina, Italy.
authors.forename: Matteo
authors.initials: M
authors.lastname: La Rosa
authors.affiliation: Neuromuscular Disease Unit, G. Gaslini Institute, Genoa, Italy.
authors.forename: Claudio
authors.initials: C
authors.lastname: Bruno
authors.affiliation: Department of Neurosciences, University of Messina, Messina, Italy.
authors.forename: Maria Grazia
authors.initials: MG
authors.lastname: Distefano
authors.affiliation: Muscle Pathology and Neuroimmunology and Developmental Neurology Unit, Neurological Institute "Carlo Besta", Milan, Italy.
authors.forename: Giovanni
authors.initials: G
authors.lastname: Baranello
authors.affiliation: Department of Neurosciences, University of Messina, Messina, Italy.
authors.forename: Costanza
authors.initials: C
authors.lastname: Barcellona
authors.affiliation: Department of Experimental Medicine, Cardiomyology and Medical Genetics, Second University of Naples, Naples, Italy.
authors.forename: Marianna
authors.initials: M
authors.lastname: Scutifero
authors.affiliation: Department of Neurosciences, University of Padua, Padua, Italy.
authors.forename: Sonia
authors.initials: S
authors.lastname: Marcato
authors.affiliation: Department of Neurosciences, University of Padua, Padua, Italy.
authors.forename: Arianna
authors.initials: A
authors.lastname: Palmieri
authors.affiliation: Department of Experimental Medicine, Cardiomyology and Medical Genetics, Second University of Naples, Naples, Italy.
authors.forename: Luisa
authors.initials: L
authors.lastname: Politano
authors.affiliation: Muscle Pathology and Neuroimmunology and Developmental Neurology Unit, Neurological Institute "Carlo Besta", Milan, Italy.
authors.forename: Lucia
authors.initials: L
authors.lastname: Morandi
authors.affiliation: Neuromuscular Center, SG. Battista Hospital, University of Turin, Turin, Italy.
authors.forename: Tiziana
authors.initials: T
authors.lastname: Mongini
authors.affiliation: Department of Neurosciences, University of Padua, Padua, Italy.
authors.forename: Elena
authors.initials: E
authors.lastname: Pegoraro
authors.affiliation: IRCCS E Medea Bosisio Parini Neuromuscular Unit, Department of Neurorehabilitation, Bosisio Parini, Italy.
authors.forename: Maria Grazia
authors.initials: MG
authors.lastname: D'Angelo
authors.affiliation: Department of Paediatric Neurology, Catholic University, Rome, Italy.
authors.forename: Marika
authors.initials: M
authors.lastname: Pane
authors.affiliation: Department of Neurosciences, University of Messina, Messina, Italy.
authors.forename: Carmelo
authors.initials: C
authors.lastname: Rodolico
authors.affiliation: Neuromuscular Disease Unit, G. Gaslini Institute, Genoa, Italy.
authors.forename: Carlo
authors.initials: C
authors.lastname: Minetti
authors.affiliation: Unit of Neuromuscular and Neurodegenerative Disorders, Laboratory of Molecular Medicine, Bambino Gesu' Children's Research Hospital, Rome, Italy.
authors.forename: Enrico
authors.initials: E
authors.lastname: Bertini
authors.affiliation: Department of Neurosciences, University of Messina, Messina, Italy; Nemo Sud Clinical Center for Neuromuscular Diseases, Messina, Italy.
authors.forename: Giuseppe
authors.initials: G
authors.lastname: Vita
authors.affiliation: Department of Paediatric Neurology, Catholic University, Rome, Italy. Electronic address: mercuri@rm.unicatt.it.
authors.forename: Eugenio
authors.initials: E
authors.lastname: Mercuri
mesh_terms: D000293:Adolescent; D002648:Child; D002675:Child, Preschool; D005221:Fatigue / Q000175:diagnosis / Q000209:etiology; D006801:Humans; D008137:Longitudinal Studies; D008297:Male; D020388:Muscular Dystrophy, Duchenne / Q000150:complications / Q000175:diagnosis* / Q000523:psychology*; D011446:Prospective Studies; D011788:Quality of Life; D012720:Severity of Illness Index; D011795:Surveys and Questionnaires
keywords: Duchenne muscular dystrophy; Outcome measures; PedsQL(TM); Quality of life