The Huntington's Disease Health Index: Initial Evaluation of a Disease-Specific Patient Reported Outcome Measure

Area of Science:

• Neurology
• Clinical Trials
• Patient-Reported Outcomes

Background:

• Patient-reported outcome measures (PROMs) are crucial for assessing therapeutic trial efficacy.
• The Huntington's Disease Health Index (HD-HI) was developed following FDA guidance for product development.
• HD-HI is a multifaceted, disease-specific PROM for Huntington's disease (HD).

Purpose of the Study:

• To evaluate the validity, reliability, clinical relevance, and patient understanding of the HD-HI.
• To prepare the HD-HI for use in upcoming clinical trials.
• To ensure the HD-HI accurately captures patient experience in HD.

Main Methods:

• Partnered with 389 individuals with HD and caregivers to identify relevant questions.
• Utilized factor analysis and cognitive interviews with 15 individuals with HD for refinement.
• Conducted test-retest reliability assessments with 25 individuals with HD.

Main Results:

• Identified 13 relevant symptomatic domains for HD-HI subscales.
• Achieved high internal consistency and reliability across all HD-HI subscales.
• HD-HI scores statistically differentiated between HD participants with varying disease burdens.

Conclusions:

• The HD-HI demonstrates initial validity and reliability as a PROM for HD.
• Supports the use of HD-HI for assessing patient-reported outcomes in Huntington's disease.
• The HD-HI is a promising tool for evaluating treatment effects in HD clinical trials.