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 Gardani; Antonella 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

doi:10.1371/journal.pone.0199223

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 journal › Article › peer-review

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 language	English
Pages (from-to)	e0199223
Journal	PLoS One
Volume	13
Issue number	6
DOIs	https://doi.org/10.1371/journal.pone.0199223
Publication status	Published - 2018

Access to Document

10.1371/journal.pone.0199223

Cite this

Pane, M., Coratti, G., Brogna, C., Mazzone, E. S., 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., ... 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 journal › Article › peer-review

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, 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.

@article{70d2a8458c3f414b8f82456423acd573,

title = "Upper limb function in Duchenne muscular dystrophy: 24 month longitudinal data",

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.",

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

year = "2018",

doi = "10.1371/journal.pone.0199223",

language = "English",

volume = "13",

pages = "e0199223",

journal = "PLoS One",

issn = "1932-6203",

publisher = "Public Library of Science",

number = "6",

}

TY - JOUR

T1 - Upper limb function in Duchenne muscular dystrophy

T2 - 24 month longitudinal data

AU - Pane, Marika

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

PY - 2018

Y1 - 2018

N2 - 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.

AB - 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.

U2 - 10.1371/journal.pone.0199223

DO - 10.1371/journal.pone.0199223

M3 - Article

C2 - 29924848

VL - 13

SP - e0199223

JO - PLoS One

JF - PLoS One

SN - 1932-6203

IS - 6

ER -

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

Abstract

Access to Document

Fingerprint

Cite this