Session Information
Date: Sunday, October 7, 2018
Session Title: Huntington's Disease
Session Time: 1:45pm-3:15pm
Location: Hall 3FG
Objective: Evaluate insight of functional impairment in premanifest and early-manifest Huntington’s disease (HD).
Background: Following the guidelines of the US Food and Drug Administration Clinical Outcome Assessment Qualification Program we used standard rating scale development methodology to create the Functional Rating Scale (FuRST 2.0), a patient-reported outcome (PRO) designed to measure functional ability in premanifest and early-manifest HD. However, the ability of Huntington’s disease gene-expansion carriers (HDGECs) to accurately self-report has been questioned due to lack of insight (McCusker & Loy, 2014). To explore the prevalence of this problem and to determine if it affects participant responses we designed an adjunct study.
Methods: Within the context of a cognitive debriefing study for the FuRST 2.0 scale development we examined participant responses between HD and companion dyads to explore score discrepancies. Data of 35 HDGEC/companion dyads were evaluated: 15 premanifest HDGECs and 15 of their companions, 20 early-manifest HDGECs (Total Functional Capacity (TFC) score of 7-13) and 20 of their companions. Kolmogorov-Smirnov (K-S) tests and Lin’s Concordance Correlation Coefficients (CCC) were performed to assess whether the HDGECs and companions had similar distributions of FuRST 2.0 item scores.
Results: Results of the K-S and Lin’s CCC demonstrated acceptable score agreement between premanifest HDGECs and their companions, but less than ideal score agreement between the early-manifest HDGECs and their companions. Further sub-group analyses revealed adequate agreement between companions and early-manifest HDGECs with a TFC of 11-13, but not those with a TFC of 7-10.
Conclusions: Premanifest HDGECs have sufficient insight to complete a PRO measuring functional ability. There is evidence that early-manifest HDGECs with TFC scores of 11-13 retain the insight needed to complete a functional PRO, but not those with TFC scores less than 11. The small sample size must be acknowledged. Further analyses will be conducted in a large international cohort of study participants.
References: McCusker E, Loy CT. The many facets of unawareness in Huntington disease. Tremor and Other Hyperkinetic Movements. 2014;4.
To cite this abstract in AMA style:
R. Fuller, P. Feigenbaum, C. Fitzer-Attas, N. La Pelle, S. Luo, C. Sampaio, G. Stebbins. Exploration of insight competence in Huntington’s disease supplemental to a cognitive debriefing study for the FuRST2.0 rating scale [abstract]. Mov Disord. 2018; 33 (suppl 2). https://www.mdsabstracts.org/abstract/exploration-of-insight-competence-in-huntingtons-disease-supplemental-to-a-cognitive-debriefing-study-for-the-furst2-0-rating-scale/. Accessed June 14, 2025.« Back to 2018 International Congress
MDS Abstracts - https://www.mdsabstracts.org/abstract/exploration-of-insight-competence-in-huntingtons-disease-supplemental-to-a-cognitive-debriefing-study-for-the-furst2-0-rating-scale/