Patient reported outcome measure for upper limb in Duchenne muscular dystrophy: correlation with PUL2.0.

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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Copyright © 2023. Published by Elsevier B.V.

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Declaration of Competing Interest Gianpaolo Cicala reports personal fees from BIOGEN S.R.L and ROCHE outside the submitted work; Giorgia Coratti reports personal fees from BIOGEN S.R.L., ROCHE, AVEXIS, NOVARTIS, GENESIS PHARMA and Biologix outside the submitted work; Marika Pane reports personal fees from BIOGEN S.R.L., ROCHE, AVEXIS and NOVARTIS outside the submitted work; Anna Capasso, Martina Ricci report personal fees from BIOGEN S.R.L., ROCHE and NOVARTIS outside the submitted work; Elena Mazzone, reports personal fees from BIOGEN S.R.L., ROCHE, NOVARTIS, DYNE outside the submitted work Nathalie Goemans has received personal fees outside the submitted work as member of data monitoring committee or advisory boards, from Biogen, Pfizer, Genethon,Roche, WAVE ther Eugenio Mercuri is part of advisory boards for BIOGEN S.R.L., ROCHE, AVEXIS and NOVARTIS, Scholar ROCK, EPIRIUM, CYTOKINETICS and NMD PHARMA. Eugenio Mercuri is funded by grant from the Italian Ministry of Health (RF-2019–12,370,334). All remaining authors have nothing to disclose. Funders had no role in the study design; in the collection, analysis, and interpretation of data; in the writing of the report; and in the decision to submit the paper for publication.