Clinical Outcomes: Rating Scales and Surveys
HD is a multifaceted disorder affecting cognitive, behavioral, and motor function, all of which play a role in functional disability, and the successful development of therapeutic interventions for HD will require novel, sensitive clinical assessments. CHDI, in collaboration with external consultants, provides recommendations on existing HD assessments after evaluation of their clinimetric and psychometric properties and, when necessary, collaborates to develop new or modified clinical assessments adhering to FDA/EMA guidance. In recent years we developed the Huntington’s Disease Cognitive Assessment Battery (HD-CAB) in collaboration with Julie Stout (Stout JC et al., 2014); a self-report functional rating scale, the Functional Rating Scale 2.0 (FuRST 2.0), with our collaborators Glenn Stebbins, Sheng Luo, and Nancy LaPelle; the HD-Structured Interview of Function (HD-SIF), a structured interview designed to standardize the information collected from the Unified Huntington’s Disease Rating Scale (UHDRS) functional rating scales; the Companion Information Form which assesses the nature of a knowledgeable informant’s relationship with a person with HD and other information critical to understanding HD companions and caregivers; and the HD-Charge Survey which evaluates the indirect and out of pocket costs of people with HD and their companions. Further scale reviews and scale development are underway.
Frequently Asked Questions
Which rating scales are used most frequently in HD?
Please see this systematic review of outcome measures used in HD research. The Unified Huntington’s Disease Rating Scale (UHDRS) was enhanced in 1999 and again in 2005 and is one of the most widely used rating scales.
Which rating scales are used currently in Enroll-HD?
Enroll-HD launched in 2012 and collects longitudinal clinical data and biosamples to accelerate the development of new therapeutics for HD. See the Enroll-HD protocol (page 11) for a list of the rating scales used in the study.
What information is available regarding rating scales in HD and their evaluation?
The National Institute of Neurological Disorders and Stroke (NINDS) provides a list of many of the HD rating scales. Also, the Movement Disorder Society Committee on Rating Scale Development has evaluated 27 HD rating scales to measure behavioral systems and provides recommendations.
What will be the outcome of your current rating scale evaluations?
CHDI and colleagues are currently evaluating the assessments used in Enroll-HD to determine whether there are floor and ceiling effects, whether they adequately cover all stages of HD progression, and to test the validity of these assessments in HD gene expansion carriers. Subsequently, development and/or adoption of alternative assessments may be considered.
When will the FuRST 2.0 be available to the HD research community?
The FuRST 2.0 Official Working Document (OWD) is available for use.
When will the FuRST 2.0 be validated?
We started collecting validation data on the FuRST 2.0 via FOCUS-HD, a series of studies evaluating the FuRST 2.0 on-line, in-person, and longitudinally. We will share the data with the HD research as it becomes available.
What is the validation process for the HD-CAB?
We are following the FDA guidance for the qualification of Clinical Outcome Assessments (COAs). We are currently at the stage of Longitudinal Evaluation of Measurement Properties/ Interpretation Methods. We evaluated 16 cognitive assessments and selected 6 of them based on sensitivity, reduced practice effects, reliability, domain coverage, feasibility, and tolerability that now comprise the HD-CAB.
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