Understanding the relationship between patient-reported outcome measures, clinician-rated assessments, and...

BackgroundGiven the progressive cognitive decline in Huntington's disease (HD), most research in this population relies solely on objective assessments of symptoms and function, rather than on patient-reported outcome (PRO) measures.ObjectiveThe purpose of this paper is to understand how PRO measures relate to clinician-rated assessments and performance-based measures (cognitive tests and device-based estimates) of symptoms and functioning in people with HD.MethodsWe enrolled N = 52 participants with HD in this study. Participants completed several self-reported PROs as well as clinician-administered assessments and performance-based measures (cognitive tests and device-based estimates) of related concepts. Pearson correlations and linear regression models were used to examine the concordance among PROs, clinician-rated assessments, and performance-based measures.ResultsThere were strong relationships among PROs that assessed related concepts. There were also strong relationships between PROs and associated clinician-rated assessments of physical functioning (chorea, sleep/fatigue) and mental health, and slightly less robust relationships between the PROs and associated clinician-rated assessments for speech/swallowing and cognition. Relationships between PROs and associated performance-based measures were moderate for chorea/motor functioning, but negligible for sleep/fatigue and cognition.ConclusionsFindings from this study support the construct validity of PROs that assess motor functioning and mental health among individuals with HD and indicate that PROs and clinician-rated assessments of these constructs (i.e., motor functioning and mental health) provide complementary information. On the other hand, the negligible relationships between PROs and associated performance-based cognitive tests and between PROs and associated wearable device-based estimates of sleep and physical activity indicate that reliance solely on HD patients' self-report for these concepts might be misleading.