Related Experiment Video
Updated: Jul 18, 2025
Measurements of Motor Function and Other Clinical Outcome Parameters in Ambulant Children with Duchenne Muscular Dystrophy
Published on: January 12, 2019
Patient reported outcome measure for upper limb in Duchenne muscular dystrophy: correlation with PUL2.0
Gianpaolo Cicala1, Marika Pane1, Giorgia Coratti1
1Paediatric Neurology, Catholic University, Rome, Italy; Centro Clinico Nemo, Fondazione Policlinico Gemelli IRCCS, Rome, Italy.
Insights
This study validated the Patient Reported Outcome Measure for the upper limb (PROM UL) against the Performance of Upper Limb (PUL 2.0) scale in Duchenne muscular dystrophy (DMD) patients. Strong correlations confirm PROM UL’s utility in assessing upper limb function in non-ambulant DMD individuals.
Area of Science:
- Neurology
- Clinical Trials
- Rehabilitation Medicine
Background:
- Clinical trials for Duchenne muscular dystrophy (DMD) increasingly require outcome measures for non-ambulant individuals.
- Assessing upper limb function is crucial for daily living activities in DMD patients.
- Existing measures may not fully capture the nuances of upper limb impairment in this population.
Purpose of the Study:
- To establish the correlation between the Patient Reported Outcome Measure for the upper limb (PROM UL) and the Performance of Upper Limb (PUL 2.0) scale.
- To validate PROM UL as a reliable tool for assessing upper limb function in non-ambulant DMD boys and young adults.
- To confirm the clinical meaningfulness of the PUL 2.0 scale.
Main Methods:
- A cohort of non-ambulant DMD patients was assessed as part of a larger natural history study.
- Concurrent evaluations using the PUL 2.0 (observer-rated) and PROM UL (patient-reported) were conducted.
- Statistical analysis focused on the correlation between total scores and specific item scores of the two instruments.
Main Results:
- One hundred twenty-five paired PUL 2.0 and PROM UL assessments from 60 non-ambulant DMD boys were analyzed.
- Total PROM UL scores demonstrated a strong correlation with both total PUL 2.0 scores and the PUL 2.0 entry item score.
- This indicates a significant agreement between patient-reported and observer-rated upper limb function assessments.
Conclusions:
- The strong correlation validates the clinical meaningfulness of the PUL 2.0 scale.
- The PROM UL is confirmed as a valuable tool for detecting the progression of upper limb involvement in DMD.
- These findings support the use of PROM UL in clinical trials for non-ambulant DMD populations.
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.
