Daily variation in symptoms and functioning in Huntington disease: Feasibility and variability

Area of Science:

• Neurology
• Clinical Research
• Patient-Reported Outcomes

Background:

• Huntington disease (HD) daily symptom and functioning experiences are understudied.
• Real-world data capture is crucial for developing future HD outcome measures.

Purpose of the Study:

• Assess the feasibility of an intensive weeklong study design for HD.
• Understand daily symptom and functioning variations in premanifest and manifest HD.

Main Methods:

• 52 HD participants completed real-time symptom reports, wore a PRO-Diary device, and daily diaries over 8 days.
• Data included chorea, sleep, fatigue, anxiety, physical activity, speech, and social participation.
• Feasibility assessed via missing data rates; variability analyzed using multilevel models.

Main Results:

• Missing data rates ranged from 19-52%, with higher missingness for real-time reports.
• PRO-Diary wear compliance was 64-78%.
• Greater symptom and functioning variability occurred day-to-day compared to within a single day.

Conclusions:

• The intensive study design is feasible for HD research, comparable to other populations.
• Real-time symptom reporting had the highest missing data rates.
• Day-to-day symptom and functioning variability suggests once-daily assessments may be adequate for HD outcome measures.