To collect patient-reported data in Huntington’s Disease (HD) and evaluate the ability of various disease-specific Patient-Reported Outcome (PRO) measures to track HD symptoms over time. 

HD clinical trials require outcome measures that are clinically relevant, reliable, and responsive to changes in disease burden. The Huntington’s Disease-Health Index (HD-HI) and the Huntington’s Disease-Patient Report of Problems (HD-PROP) are disease-specific PROs developed to measure how individuals with HD feel and function. LEAD-HD is implementing the HD-HI and the HD-PROP in a longitudinal study to generate performance metrics of these PROs and collect patient-reported natural history data. 

Adults with HD are participating in a remote, 2-year study via the myHDStory research platform. At baseline, participants complete demographic questions, the HD-HI, HD-PROP, HLS19-Q12 health literacy questionnaire, and self-reported Total Functional Capacity (TFC) scale. At 6, 12, 18, and 24 months, participants will complete the HD-HI, HD-PROP, and TFC scales. The primary analysis will examine longitudinal trajectories of PRO scores and explore associations between baseline demographic characteristics and disease progression.

To date, there are 50 participants with 31 providing complete baseline data. Current participants are 74.2% female, range in age from 24-73 years, and have a mean age at diagnosis of 39.5 +/- SD 13.1 years. Mean baseline total HD-HI scores were 20.8 +/- SD 21.2 points (scores range 0-100; higher score = more disease burden). On the HD-PROP, the most serious HD-related problems reported were balance issues, impaired memory, reduced enjoyment reading, difficulty with community interactions, and impaired executive function.

LEAD-HD enables patients to report their experiences with HD via the myHDstory research platform. Future data will determine the ability of the HD-HI and HD-PROP to track changes in HD symptoms over time, which may facilitate future HD clinical trial outcomes and design.