Objective: To improve collection of outcomes meaningful to patients with Huntington’s disease (HD) and their families, through the development of novel assessments of daily functional ability, irritability, and speech difficulty.

Background: With an increasing focus on clinical research, there are a number of recently developed outcome measures in HD, particularly patient-reported outcome measures, notably the HD-PRO-TRIAD, HD-Health Index and the FuRST 2.0. Despite these valuable additions, there is still need for a clinician-reported outcome (ClinRO) measure of daily function that can assess change in a more granular fashion than existing ClinROs. With multidimensional diseases, such as HD, there is a need to measure a broad range of symptoms and impacts. Gaps were identified while evaluating the holistic measurement strategy for GENERATION HD1 against qualitative research (indicating the core symptoms and impacts from the perspectives of patients and their families). To balance patient burden with the desire for comprehensive measurement, it was determined that in addition to a novel ClinRO assessing daily functional ability, brief measures of patient-reported speech difficulty, and companion-reported irritability were also required.

Method: Qualitative research was reviewed and, in collaboration with three HD clinical experts, an iterative process was used to develop items and response options to assess clinician-reported daily functional ability. Additionally, a single-item patient-reported assessment of speech difficulty and three draft companion-reported items to assess observed irritability and angry outbursts were developed. The draft companion-reported measure was shared with the international HD-COPE board of patients and care companions via an interactive online platform. Feedback was used to revise the content.

Results: Three novel scales were developed: the HD-Daily Activities Scale (HD-DAS), a 25-item measure of daily activities; the HD-Companion-reported Irritability and Angry Outbursts Scale (HD-CIAOS), a 2–3 item measure of irritability and angry outbursts; and the HD-Speaking Difficulty Item (HD-SDI), a single-item measure of speech difficulty.

Conclusion: Three novel outcome scales are being used in the GENERATION HD1 study as exploratory measures: the HD-DAS, the HD-CIAOS and the HD-SDI. Future assessment of their measurement properties (e.g., validity and reliability) is required to support their use in longitudinal research.

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MDS Abstracts - https://www.mdsabstracts.org/abstract/novel-exploratory-outcome-assessments-in-generation-hd1/