Rate of functional decline in Huntington’s disease

Abstract

The comparable rates of decline on the TFC and the IS scores with other published studies suggest that these estimates of functional decline are representative of HD patients who are evaluated at HSG research sites. In longitudinal analysis, longer disease duration and better neuropsychological performance at baseline were associated with a less rapid rate of decline in TFC score, whereas depressive symptomatology at baseline was associated with a more rapid decline in the IS score. These rates of functional decline and the covariates that modify them should be considered in estimating statistical power and designing future therapeutic trials involving HD patients with early or moderately severe disease.

Verbal fluency in Huntington’s disease: a longitudinal analysis of phonemic and semantic clustering and switching

“…The assumption of linear decline is relevant and has been previously used for this population [23].There were at least three data points (yearly assessments) for any patient, hence yielding a robust regression line to depict the picture of possible deterioration, improvement or stable performance over time. The effect sizes for the mean of patients' regression scores for each measure was then tested for significance with α at 0.05 (two-tailed).…”

Exploring the Validity of the Short Version of the Problem Behaviours Assessment (PBA-s) for Huntington’s disease: A Rasch Analysis

“…Participants with a positive HD genotype who had not yet met the motor-defined diagnostic criteria (premanifest HD), as judged by a qualified interviewer, were included alongside participants with motor signs (manifest HD). The premanifest HD group were included as neuropsychiatric symptoms can occur several years before the onset of motor signs [7,8]. Total Functional Capacity (TFC) score (0-13) was used to classify manifest HD participants into five categories, lower scores signifying greater functional impairment [25].…”

“…Behavioural (neuropsychiatric) symptoms in HD have been reported to have a greater impact on quality of life [5] and functional disability [6] than either cognitive or motor symptoms. These manifestations may develop many years before the onset of distinctive motor signs and are increasingly recognised as the main reason for institutionalisation, due to the disabling and distressing impact they have on both patients and their carers [6][7][8], making their early recognition vital.…”

Neuroprotection for Huntington’s Disease: Ready, Set, Slow

“…Regional cerebral cortical neurodegeneration in HD is heterogeneous in time and location, which may explain some of the differences between patients, as well as the evolution of symptoms in time, despite the early and stereotypical decimation of the striatum. 20 Despite the great day-to-day variability in symptoms and their uneven evolution over time, the overall clinical progression of HD is slow, as assessed by an integrated measure of functional capacity (e.g., the Total Functional Capacity scale, or TFC 45 ). Clinical trials in early symptomatic HD include subjects with a TFC of about 7 or greater.…”