Upper limb function in Duchenne muscular dystrophy: 24 month longitudinal data

Marika Pane, Giorgia Coratti, Claudia Brogna, Elena Stacy Mazzone, Anna Mayhew, Lavinia Fanelli, Sonia Messina, Adele D'Amico, Michela Catteruccia, Marianna Scutifero, Silvia Frosini, Valentina Lanzillotta, Giulia Colia, Filippo Cavallaro, Enrica Rolle, Roberto De Sanctis, Nicola Forcina, Roberta Petillo, Andrea Barp, Alice GardaniAntonella Pini, Giulia Monaco, Maria Grazia D'Angelo, Riccardo Zanin, Gian Luca Vita, Claudio Bruno, Tiziana Mongini, Federica Ricci, Elena Pegoraro, Luca Bello, Angela Berardinelli, Roberta Battini, Valeria Sansone, Emilio Albamonte, Giovanni Baranello, Enrico Bertini, Luisa Politano, Maria Pia Sormani, Eugenio Mercuri

Research output: Contribution to journalArticle

Abstract

The aim of the study was to establish 24 month changes in upper limb function using a revised version of the performance of upper limb test (PUL 2.0) in a large cohort of ambulant and non-ambulant boys with Duchenne muscular dystrophy and to identify possible trajectories of progression. Of the 187 patients studied, 87 were ambulant (age range: 7-15.8 years), and 90 non-ambulant (age range: 9.08-24.78). The total scores changed significantly over time (p<0.001). Non-ambulant patients had lower total scores at baseline (mean 19.7) when compared to the ambulant ones (mean 38.4). They also had also a bigger decrease in total scores over 24 months compared to the ambulant boys (4.36 vs 2.07 points). Multivariate model analysis showed that the Performance of Upper Limb changes reflected the entry level and ambulation status, that were independently associated to the slope of Performance of Upper Limb changes. This information will be of help both in clinical practice and at the time of designing clinical trials.

Original languageEnglish
Pages (from-to)e0199223
JournalPLoS One
Volume13
Issue number6
DOIs
Publication statusPublished - 2018

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muscular dystrophy
Duchenne Muscular Dystrophy
Upper Extremity
Trajectories
trajectories
clinical trials
Walking
Multivariate Analysis
testing
Clinical Trials

Cite this

Pane, M., Coratti, G., Brogna, C., Mazzone, E. S., Mayhew, A., Fanelli, L., ... Mercuri, E. (2018). Upper limb function in Duchenne muscular dystrophy: 24 month longitudinal data. PLoS One, 13(6), e0199223. https://doi.org/10.1371/journal.pone.0199223

Upper limb function in Duchenne muscular dystrophy : 24 month longitudinal data. / Pane, Marika; Coratti, Giorgia; Brogna, Claudia; Mazzone, Elena Stacy; Mayhew, Anna; Fanelli, Lavinia; Messina, Sonia; D'Amico, Adele; Catteruccia, Michela; Scutifero, Marianna; Frosini, Silvia; Lanzillotta, Valentina; Colia, Giulia; Cavallaro, Filippo; Rolle, Enrica; De Sanctis, Roberto; Forcina, Nicola; Petillo, Roberta; Barp, Andrea; Gardani, Alice; Pini, Antonella; Monaco, Giulia; D'Angelo, Maria Grazia; Zanin, Riccardo; Vita, Gian Luca; Bruno, Claudio; Mongini, Tiziana; Ricci, Federica; Pegoraro, Elena; Bello, Luca; Berardinelli, Angela; Battini, Roberta; Sansone, Valeria; Albamonte, Emilio; Baranello, Giovanni; Bertini, Enrico; Politano, Luisa; Sormani, Maria Pia; Mercuri, Eugenio.

In: PLoS One, Vol. 13, No. 6, 2018, p. e0199223.

Research output: Contribution to journalArticle

Pane, M, Coratti, G, Brogna, C, Mazzone, ES, Mayhew, A, Fanelli, L, Messina, S, D'Amico, A, Catteruccia, M, Scutifero, M, Frosini, S, Lanzillotta, V, Colia, G, Cavallaro, F, Rolle, E, De Sanctis, R, Forcina, N, Petillo, R, Barp, A, Gardani, A, Pini, A, Monaco, G, D'Angelo, MG, Zanin, R, Vita, GL, Bruno, C, Mongini, T, Ricci, F, Pegoraro, E, Bello, L, Berardinelli, A, Battini, R, Sansone, V, Albamonte, E, Baranello, G, Bertini, E, Politano, L, Sormani, MP & Mercuri, E 2018, 'Upper limb function in Duchenne muscular dystrophy: 24 month longitudinal data', PLoS One, vol. 13, no. 6, pp. e0199223. https://doi.org/10.1371/journal.pone.0199223
Pane M, Coratti G, Brogna C, Mazzone ES, Mayhew A, Fanelli L et al. Upper limb function in Duchenne muscular dystrophy: 24 month longitudinal data. PLoS One. 2018;13(6):e0199223. https://doi.org/10.1371/journal.pone.0199223
Pane, Marika ; Coratti, Giorgia ; Brogna, Claudia ; Mazzone, Elena Stacy ; Mayhew, Anna ; Fanelli, Lavinia ; Messina, Sonia ; D'Amico, Adele ; Catteruccia, Michela ; Scutifero, Marianna ; Frosini, Silvia ; Lanzillotta, Valentina ; Colia, Giulia ; Cavallaro, Filippo ; Rolle, Enrica ; De Sanctis, Roberto ; Forcina, Nicola ; Petillo, Roberta ; Barp, Andrea ; Gardani, Alice ; Pini, Antonella ; Monaco, Giulia ; D'Angelo, Maria Grazia ; Zanin, Riccardo ; Vita, Gian Luca ; Bruno, Claudio ; Mongini, Tiziana ; Ricci, Federica ; Pegoraro, Elena ; Bello, Luca ; Berardinelli, Angela ; Battini, Roberta ; Sansone, Valeria ; Albamonte, Emilio ; Baranello, Giovanni ; Bertini, Enrico ; Politano, Luisa ; Sormani, Maria Pia ; Mercuri, Eugenio. / Upper limb function in Duchenne muscular dystrophy : 24 month longitudinal data. In: PLoS One. 2018 ; Vol. 13, No. 6. pp. e0199223.
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abstract = "The aim of the study was to establish 24 month changes in upper limb function using a revised version of the performance of upper limb test (PUL 2.0) in a large cohort of ambulant and non-ambulant boys with Duchenne muscular dystrophy and to identify possible trajectories of progression. Of the 187 patients studied, 87 were ambulant (age range: 7-15.8 years), and 90 non-ambulant (age range: 9.08-24.78). The total scores changed significantly over time (p<0.001). Non-ambulant patients had lower total scores at baseline (mean 19.7) when compared to the ambulant ones (mean 38.4). They also had also a bigger decrease in total scores over 24 months compared to the ambulant boys (4.36 vs 2.07 points). Multivariate model analysis showed that the Performance of Upper Limb changes reflected the entry level and ambulation status, that were independently associated to the slope of Performance of Upper Limb changes. This information will be of help both in clinical practice and at the time of designing clinical trials.",
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AU - Coratti, Giorgia

AU - Brogna, Claudia

AU - Mazzone, Elena Stacy

AU - Mayhew, Anna

AU - Fanelli, Lavinia

AU - Messina, Sonia

AU - D'Amico, Adele

AU - Catteruccia, Michela

AU - Scutifero, Marianna

AU - Frosini, Silvia

AU - Lanzillotta, Valentina

AU - Colia, Giulia

AU - Cavallaro, Filippo

AU - Rolle, Enrica

AU - De Sanctis, Roberto

AU - Forcina, Nicola

AU - Petillo, Roberta

AU - Barp, Andrea

AU - Gardani, Alice

AU - Pini, Antonella

AU - Monaco, Giulia

AU - D'Angelo, Maria Grazia

AU - Zanin, Riccardo

AU - Vita, Gian Luca

AU - Bruno, Claudio

AU - Mongini, Tiziana

AU - Ricci, Federica

AU - Pegoraro, Elena

AU - Bello, Luca

AU - Berardinelli, Angela

AU - Battini, Roberta

AU - Sansone, Valeria

AU - Albamonte, Emilio

AU - Baranello, Giovanni

AU - Bertini, Enrico

AU - Politano, Luisa

AU - Sormani, Maria Pia

AU - Mercuri, Eugenio

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