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Development of a patient-reported outcome measure for upper limb function in Duchenne muscular dystrophy : DMD Upper Limb PROM

AIM: To develop a patient-reported outcome measure (PROM) assessing upper limb
function related to activities of daily living (ADL) that cannot be observed in a
clinical setting, specifically for patients with Duchenne muscular dystrophy
(DMD) across a wide age range, applicable in the different stages of the disease.
METHOD: The developmental process was based on US Food and Drug Administration
guidelines. This included item generation from a systematic review of existing
tools and expert opinion on task difficulty and relevance, involving individuals
with DMD. Cultural aspects affecting ADL were taken into consideration to make
this tool applicable to the broad DMD community. Items were selected in relation
to a conceptual framework reflecting disease progression covering the full range
of upper limb function across different ADL domains. RESULTS: After pilot testing
and iterative Rasch analyses, redundant or clinically irrelevant items were
removed. The final questionnaire consists of 32 items covering four domains of
ADL (food, self-care, household and environment, leisure and communication).
Test-retest reliability was excellent. INTERPRETATION:
A DMD-specific upper limb
PROM was developed on the basis of clinical relevance and psychometric
robustness. Its main purpose is to document the patient self-reported natural
history of DMD and assess the efficacy of interventions.
CI - (c) 2016 Mac Keith Press.

Langue : ANGLAIS

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