Understanding the relationship between patient-reported outcome measures, clinician-rated assessments, and performance-based measures (cognitive tests and device-based estimates) of symptoms and functioning in people with Huntington's disease

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.