The increasing pressure to include non ambulant Duchenne muscular dystrophy (DMD) boys in clinical trials has highlighted the need for outcome measures that could address the impact of upper limb function on activities of daily living. The aim of the present study was to establish the correlation between the recently developed Patient Reported Outcome Measure for the upper limb (PROM UL) and the observer rated functional scale Performance of Upper Limb (PUL 2.0) in a large cohort of DMD boys and young adults. As part of a larger natural history study, non ambulant DMD patients were assessed using PUL2.0 and PROM UL. One hundred and twenty-five concurrent PUL 2.0 and PROM UL evaluations from 60 non ambulant DMD boys were taken into consideration. The total PROM UL scores showed a strong correlation with both PUL 2.0 total scores and with PUL 2.0 entry item score. The strong correlation between the two tools confirms the clinical meaningfulness of the PUL2.0 and that the PROM UL can help to detect the gradient of progression of upper limb involvement.
Cicala, G., Pane, M., Coratti, G., Brogna, C., Fanelli, L., Norcia, G., Forcina, N., Mazzone, E. S., Stanca, G., Ferrante, A. M. R., Vento, A., Ferraroli, E., Ricci, M., Capasso, A., Leone, D., Palermo, F. C., Berti, B., Cutrona, C., Mahyew, A., Duong, T., Goemans, N., Vroom, E., Mercuri, E. M., Patient reported outcome measure for upper limb in Duchenne muscular dystrophy: correlation with PUL2.0, <<NEUROMUSCULAR DISORDERS>>, 2023; 33 (9): 69-73. [doi:10.1016/j.nmd.2023.07.003] [https://hdl.handle.net/10807/260287]
Patient reported outcome measure for upper limb in Duchenne muscular dystrophy: correlation with PUL2.0
Pane, Marika;Coratti, Giorgia;Brogna, Claudia;Mazzone, Elena Stacy;Ferrante, Angela Maria Rosaria;Ferraroli, Elisabetta;Capasso, Anna;Palermo, Francesco Cesare;Cutrona, Costanza;Mercuri, Eugenio Maria
2023
Abstract
The increasing pressure to include non ambulant Duchenne muscular dystrophy (DMD) boys in clinical trials has highlighted the need for outcome measures that could address the impact of upper limb function on activities of daily living. The aim of the present study was to establish the correlation between the recently developed Patient Reported Outcome Measure for the upper limb (PROM UL) and the observer rated functional scale Performance of Upper Limb (PUL 2.0) in a large cohort of DMD boys and young adults. As part of a larger natural history study, non ambulant DMD patients were assessed using PUL2.0 and PROM UL. One hundred and twenty-five concurrent PUL 2.0 and PROM UL evaluations from 60 non ambulant DMD boys were taken into consideration. The total PROM UL scores showed a strong correlation with both PUL 2.0 total scores and with PUL 2.0 entry item score. The strong correlation between the two tools confirms the clinical meaningfulness of the PUL2.0 and that the PROM UL can help to detect the gradient of progression of upper limb involvement.
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