Measuring Duchenne muscular dystrophy impact: development of a proxy-reported measure derived from PROMIS item banks.
Carolyn Emily SchwartzRoland B StarkDavid CellaKatrina BorowiecKatherine L GoochIvana F AudhyaPublished in: Orphanet journal of rare diseases (2021)
The present study derived a DMD-specific proxy-report measure from PROMIS item banks and supplemental items that could potentially be utilized in caregiver research across all stages of the care recipient's DMD. Future research will focus on assessing the responsiveness and validity of the measure over time and its comparison to DMD patient self-report.