학술논문
Longitudinal Analysis of PUL 2.0 Domains in Ambulant and Non-Ambulant Duchenne Muscular Dystrophy Patients: How do they Change in Relation to Functional Ability?
Document Type
Academic Journal
Author
Pane M; Pediatric Neurology, Università Cattolica delSacro Cuore, Rome, Italy.; Centro Clinico Nemo, Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome, Italy.; Coratti G; Centro Clinico Nemo, Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome, Italy.; Brogna C; Centro Clinico Nemo, Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome, Italy.; Bovis F; Department of Health Sciences (DISSAL), University of Genova, Genova, Italy.; D'Amico A; Unit of Neuromuscular and Neurodegenerative Disorders, Bambino Gesù Children'sHospital, Rome, Italy.; Pegoraro E; Department of Neurosciences, University of Padua, Padua, Italy.; Bello L; Department of Neurosciences, University of Padua, Padua, Italy.; Sansone V; The NEMO Clinical Center in Milan, Neurorehabilitation Unit, University of Milan, ASST Niguarda Hospital, Milan, Italy.; Albamonte E; The NEMO Clinical Center in Milan, Neurorehabilitation Unit, University of Milan, ASST Niguarda Hospital, Milan, Italy.; Ferraroli E; Pediatric Neurology, Università Cattolica delSacro Cuore, Rome, Italy.; Mazzone ES; Pediatric Neurology, Università Cattolica delSacro Cuore, Rome, Italy.; Fanelli L; Centro Clinico Nemo, Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome, Italy.; Messina S; Department of Clinical and Experimental Medicine, University of Messina, Messina, Italy.; Catteruccia M; Unit of Neuromuscular and Neurodegenerative Disorders, Bambino Gesù Children'sHospital, Rome, Italy.; Cicala G; Pediatric Neurology, Università Cattolica delSacro Cuore, Rome, Italy.; Centro Clinico Nemo, Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome, Italy.; Ricci M; Pediatric Neurology, Università Cattolica delSacro Cuore, Rome, Italy.; Centro Clinico Nemo, Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome, Italy.; Frosini S; Department of Developmental Neuroscience, IRCCS Stella Maris, Pisa, Italy.; De Luca G; Unit of Neuromuscular and Neurodegenerative Disorders, Bambino Gesù Children'sHospital, Rome, Italy.; Rolle E; Neuromuscular Center, AOU Città della Salute e della Scienza, University of Torino, Turin, Italy.; De Sanctis R; Centro Clinico Nemo, Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome, Italy.; Forcina N; Centro Clinico Nemo, Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome, Italy.; Norcia G; Centro Clinico Nemo, Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome, Italy.; Passamano L; Department of Experimental Medicine, Cardiomiology and Medical Genetics, Second University of Naples, Naples, Italy.; Gardani A; Child and Adolescence NeurologicalUnit, National Neurological Institute Casimiro MondinoFoundation, IRCCS, Pavia, Italy.; Pini A; Child Neurologyand Psychiatry Unit, IRCCS Institute of Neurological Sciences, Bellaria Hospital, Bologna, Italy.; Monaco G; Child Neurologyand Psychiatry Unit, IRCCS Institute of Neurological Sciences, Bellaria Hospital, Bologna, Italy.; D'Angelo MG; Neuro Muscular Unit IRCCS Eugenio Medea, Bosisio Parini, Italy.; Capasso A; Pediatric Neurology, Università Cattolica delSacro Cuore, Rome, Italy.; Centro Clinico Nemo, Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome, Italy.; Leone D; Centro Clinico Nemo, Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome, Italy.; Zanin R; Developmental Neurology Unit, Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan, Italy.; Vita GL; Unit of Neurology, IRCCS Centro Neurolesi Bonino-Pulejo - P.O. Piemonte, Messina, Italy.; Panicucci C; Center of Translational and Experimental Myology, IRCCS Istituto Giannina Gaslini, and Department of Neuroscience, Rehabilitation, Ophtalmology, Genetics, Maternal and ChildHealth-DINOGMI, University of Genova, Genova, Italy.; Bruno C; Center of Translational and Experimental Myology, IRCCS Istituto Giannina Gaslini, and Department of Neuroscience, Rehabilitation, Ophtalmology, Genetics, Maternal and ChildHealth-DINOGMI, University of Genova, Genova, Italy.; Mongini T; Neuromuscular Center, AOU Città della Salute e della Scienza, University of Torino, Turin, Italy.; Ricci F; Neuromuscular Center, AOU Città della Salute e della Scienza, University of Torino, Turin, Italy.; Berardinelli A; Child and Adolescence NeurologicalUnit, National Neurological Institute Casimiro MondinoFoundation, IRCCS, Pavia, Italy.; Battini R; Department of Developmental Neuroscience, IRCCS Stella Maris, Pisa, Italy.; Department of Clinical and Experimental Medicine, University of Pisa, Italy.; Masson R; Developmental Neurology Unit, Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan, Italy.; Baranello G; Developmental Neurology Unit, Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan, Italy.; Dosi C; Developmental Neurology Unit, Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan, Italy.; Bertini E; Unit of Neuromuscular and Neurodegenerative Disorders, Bambino Gesù Children'sHospital, Rome, Italy.; Politano L; Cardiomyology and Medical Genetics Unit, Università degli Studi della CampaniaLuigi Vanvitelli Scuola di Medicina e Chirurgia, Napoli, Italy.; Mercuri E; Pediatric Neurology, Università Cattolica delSacro Cuore, Rome, Italy.
Source
Publisher: IOS Press Country of Publication: Netherlands NLM ID: 101649948 Publication Model: Print Cited Medium: Internet ISSN: 2214-3602 (Electronic) NLM ISO Abbreviation: J Neuromuscul Dis Subsets: MEDLINE
Subject
Language
English
Abstract
Background: The performance of upper limb 2.0 (PUL) is widely used to assess upper limb function in DMD patients. The aim of the study was to assess 24 month PUL changes in a large cohort of DMD patients and to establish whether domains changes occur more frequently in specific functional subgroups.
Methods: The PUL was performed in 311 patients who had at least one pair of assessments at 24 months, for a total of 808 paired assessments. Ambulant patients were subdivided according to the ability to walk: >350, 250-350, ≤250 meters. Non ambulant patients were subdivided according to the time since they lost ambulation: <1, 1-2, 2-5 or >5 years.
Results: At 12 months, the mean PUL 2.0 change on all the paired assessments was -1.30 (-1.51--1.05) for the total score, -0.5 (-0.66--0.39) for the shoulder domain, -0.6 (-0.74--0.5) for the elbow domain and -0.1 (-0.20--0.06) for the distal domain.At 24 months, the mean PUL 2.0 change on all the paired assessments was -2.9 (-3.29--2.60) for the total score, -1.30 (-1.47--1.09) for the shoulder domain, -1.30 (-1.45--1.11) for the elbow domain and -0.4 (-1.48--1.29) for the distal domain.Changes at 12 and 24 months were statistically significant between subgroups with different functional abilities for the total score and each domain (p < 0.001).
Conclusion: There were different patterns of changes among the functional subgroups in the individual domains. The time of transition, including the year before and after loss of ambulation, show the peak of negative changes in PUL total scores that reflect not only loss of shoulder but also of elbow activities. These results suggest that patterns of changes should be considered at the time of designing clinical trials.
Methods: The PUL was performed in 311 patients who had at least one pair of assessments at 24 months, for a total of 808 paired assessments. Ambulant patients were subdivided according to the ability to walk: >350, 250-350, ≤250 meters. Non ambulant patients were subdivided according to the time since they lost ambulation: <1, 1-2, 2-5 or >5 years.
Results: At 12 months, the mean PUL 2.0 change on all the paired assessments was -1.30 (-1.51--1.05) for the total score, -0.5 (-0.66--0.39) for the shoulder domain, -0.6 (-0.74--0.5) for the elbow domain and -0.1 (-0.20--0.06) for the distal domain.At 24 months, the mean PUL 2.0 change on all the paired assessments was -2.9 (-3.29--2.60) for the total score, -1.30 (-1.47--1.09) for the shoulder domain, -1.30 (-1.45--1.11) for the elbow domain and -0.4 (-1.48--1.29) for the distal domain.Changes at 12 and 24 months were statistically significant between subgroups with different functional abilities for the total score and each domain (p < 0.001).
Conclusion: There were different patterns of changes among the functional subgroups in the individual domains. The time of transition, including the year before and after loss of ambulation, show the peak of negative changes in PUL total scores that reflect not only loss of shoulder but also of elbow activities. These results suggest that patterns of changes should be considered at the time of designing clinical trials.