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 | |
| Publication status | Published - 2018 |
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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
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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 -