title: Benefits of glucocorticoids in non-ambulant boys/men with Duchenne muscular dystrophy: A multicentric longitudinal study using the Performance of Upper Limb test.
abstract: The aim of this study was to establish the possible effect of glucocorticoid treatment on upper limb function in a cohort of 91 non-ambulant DMD boys and adults of age between 11 and 26 years. All 91 were assessed using the Performance of Upper Limb test. Forty-eight were still on glucocorticoid after loss of ambulation, 25 stopped steroids at the time they lost ambulation and 18 were GC naïve or had steroids while ambulant for less than a year. At baseline the total scores ranged between 0 and 74 (mean 41.20). The mean total scores were 47.92 in the glucocorticoid group, 36 in those who stopped at loss of ambulation and 30.5 in the naïve group (p < 0.001). The 12-month changes ranged between -20 and 4 (mean -4.4). The mean changes were -3.79 in the glucocorticoid group, -5.52 in those who stopped at loss of ambulation and -4.44 in the naïve group. This was more obvious in the patients between 12 and 18 years and at shoulder and elbow levels. Our findings suggest that continuing glucocorticoids throughout teenage years and adulthood after loss of ambulation appears to have a beneficial effect on upper limb function.
authors.affiliation: Child Neurology and Psychiatry Unit, Catholic University, Rome, Italy.
authors.forename: Marika
authors.initials: M
authors.lastname: Pane
authors.affiliation: Child Neurology and Psychiatry Unit, Catholic University, Rome, Italy.
authors.forename: Lavinia
authors.initials: L
authors.lastname: Fanelli
authors.affiliation: Child Neurology and Psychiatry Unit, Catholic University, Rome, Italy.
authors.forename: Elena Stacy
authors.initials: ES
authors.lastname: Mazzone
authors.affiliation: Child Neurology and Psychiatry Unit, Catholic University, Rome, Italy.
authors.forename: Giorgia
authors.initials: G
authors.lastname: Olivieri
authors.affiliation: Unit of Neuromuscular and Neurodegenerative Disorders, Bambino Gesù Children's Hospital, Rome, Italy.
authors.forename: Adele
authors.initials: A
authors.lastname: D'Amico
authors.affiliation: Department of Neurosciences and Nemo Sud Clinical Center, University of Messina, Messina, Italy.
authors.forename: Sonia
authors.initials: S
authors.lastname: Messina
authors.affiliation: Cardiomiology and Medical Genetics, Department of Experimental Medicine, Second University of Naples, Naples, Italy.
authors.forename: Marianna
authors.initials: M
authors.lastname: Scutifero
authors.affiliation: Department of Developmental Neuroscience, IRCCS Stella Maris, Pisa, Italy.
authors.forename: Roberta
authors.initials: R
authors.lastname: Battini
authors.affiliation: Cardiomiology and Medical Genetics, Department of Experimental Medicine, Second University of Naples, Naples, Italy.
authors.forename: Roberta
authors.initials: R
authors.lastname: Petillo
authors.affiliation: Department of Developmental Neuroscience, IRCCS Stella Maris, Pisa, Italy.
authors.forename: Silvia
authors.initials: S
authors.lastname: Frosini
authors.affiliation: Child Neurology and Psychiatry Unit, Catholic University, Rome, Italy.
authors.forename: Serena
authors.initials: S
authors.lastname: Sivo
authors.affiliation: Department of Neurosciences and Nemo Sud Clinical Center, University of Messina, Messina, Italy.
authors.forename: Gian Luca
authors.initials: GL
authors.lastname: Vita
authors.affiliation: Center of Myology and Neurodegenerative Disorders and Physical and Rehabilitation Medicine Unit, Istituto Giannina Gaslini, Genova, Italy.
authors.forename: Claudio
authors.initials: C
authors.lastname: Bruno
authors.affiliation: Neuromuscular Center, AOU Città della Salute e della Scienza, University of Torino, 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: Child Neurology and Psychiatry Unit, Catholic University, Rome, Italy.
authors.forename: Roberto
authors.initials: R
authors.lastname: De Sanctis
authors.affiliation: IRCCS Casimiro Mondino Foundation, Pavia, Italy.
authors.forename: Alice
authors.initials: A
authors.lastname: Gardani
authors.affiliation: IRCCS Casimiro Mondino Foundation, Pavia, Italy.
authors.forename: Angela
authors.initials: A
authors.lastname: Berardinelli
authors.affiliation: Center of Myology and Neurodegenerative Disorders and Physical and Rehabilitation Medicine Unit, Istituto Giannina Gaslini, Genova, Italy.
authors.forename: Valentina
authors.initials: V
authors.lastname: Lanzillotta
authors.affiliation: Unit of Neuromuscular and Neurodegenerative Disorders, Bambino Gesù Children's Hospital, Rome, Italy.
authors.forename: Adelina
authors.initials: A
authors.lastname: Carlesi
authors.affiliation: Cardiomiology and Medical Genetics, Department of Experimental Medicine, Second University of Naples, Naples, Italy.
authors.forename: Emanuela
authors.initials: E
authors.lastname: Viggiano
authors.affiliation: Department of Neurosciences and Nemo Sud Clinical Center, University of Messina, Messina, Italy.
authors.forename: Filippo
authors.initials: F
authors.lastname: Cavallaro
authors.affiliation: Department of Neurosciences and Nemo Sud Clinical Center, University of Messina, Messina, Italy.
authors.forename: Maria
authors.initials: M
authors.lastname: Sframeli
authors.affiliation: Department of Neurosciences, University of Padua, Padua, Italy.
authors.forename: Luca
authors.initials: L
authors.lastname: Bello
authors.affiliation: Department of Neurosciences, University of Padua, Padua, Italy.
authors.forename: Andrea
authors.initials: A
authors.lastname: Barp
authors.affiliation: Child Neurology and Psychiatry Unit, Catholic University, Rome, Italy.
authors.forename: Flaviana
authors.initials: F
authors.lastname: Bianco
authors.affiliation: Child Neurology and Psychiatry Unit, IRCCS Institute of Neurological Sciences, Bellaria Hospital, Bologna, Italy.
authors.forename: Serena
authors.initials: S
authors.lastname: Bonfiglio
authors.affiliation: Neuromuscular Center, AOU Città della Salute e della Scienza, University of Torino, Turin, Italy.
authors.forename: Enrica
authors.initials: E
authors.lastname: Rolle
authors.affiliation: Child Neurology and Psychiatry Unit, Catholic University, Rome, Italy.
authors.forename: Concetta
authors.initials: C
authors.lastname: Palermo
authors.affiliation: IRCCS Eugenio Medea, Bosisio Parini, Italy.
authors.forename: Grazia
authors.initials: G
authors.lastname: D'Angelo
authors.affiliation: Child Neurology and Psychiatry Unit, IRCCS Institute of Neurological Sciences, Bellaria Hospital, Bologna, Italy.
authors.forename: Antonella
authors.initials: A
authors.lastname: Pini
authors.affiliation: Pediatric Neurology and Myopathology Units, Neurological Institute Carlo Besta, Milan, Italy.
authors.forename: Elena
authors.initials: E
authors.lastname: Iotti
authors.affiliation: Centro Clinico Nemo, Milan, Italy.
authors.forename: Ksenija
authors.initials: K
authors.lastname: Gorni
authors.affiliation: Pediatric Neurology and Myopathology Units, Neurological Institute Carlo Besta, Milan, Italy.
authors.forename: Giovanni
authors.initials: G
authors.lastname: Baranello
authors.affiliation: Unit of Neuromuscular and Neurodegenerative Disorders, Bambino Gesù Children's Hospital, Rome, Italy.
authors.forename: Enrico
authors.initials: E
authors.lastname: Bertini
authors.affiliation: Cardiomiology and Medical Genetics, Department of Experimental Medicine, Second University of Naples, Naples, Italy.
authors.forename: Luisa
authors.initials: L
authors.lastname: Politano
authors.affiliation: Biostatistics Unit, Department of Health Sciences, University of Genoa, Genoa, Italy.
authors.forename: Maria Pia
authors.initials: MP
authors.lastname: Sormani
authors.affiliation: Child Neurology and Psychiatry Unit, Catholic University, Rome, Italy. Electronic address: mercuri@rm.unicatt.it.
authors.forename: Eugenio
authors.initials: E
authors.lastname: Mercuri
mesh_terms: D000293:Adolescent; D000328:Adult; D002648:Child; D005938:Glucocorticoids / Q000627:therapeutic use*; D006801:Humans; D008137:Longitudinal Studies; D008297:Male; D020388:Muscular Dystrophy, Duchenne / Q000188:drug therapy* / Q000503:physiopathology; D020127:Recovery of Function; D016896:Treatment Outcome; D034941:Upper Extremity / Q000503:physiopathology*; D055815:Young Adult
chemical_list: D005938:Glucocorticoids
keywords: Duchenne muscular dystrophy; Glucocorticoids; Non ambulant; PUL; Upper limb