Long-Term Effects of Annual Intensive Rehabilitation in Patients with Hereditary Pure Cerebellar Ataxia: A 7-year Follow-up Study

Abstract

Although intensive rehabilitation has achieved short-term benefits in patients with spinocerebellar degeneration, long-term outcomes of periodic intervention remain unclear, particularly in patients with pure spinocerebellar ataxia types 6 (SCA6) and 31 (SCA31). To investigate the longitudinal effects of annual intensive rehabilitation on ataxic symptoms and balance function in patients with pure cerebellar type SCA6 and SCA31. Seven patients with genetically confirmed SCA6 or SCA31 participated in annual 4-week intensive rehabilitation programmes. Each programme consisted of daily physical therapy, occupational/speech therapy, and self-directed balance training. The participants were assessed annually at pre-intervention, post-intervention, and the 6-month follow-up using the Scale for the Assessment and Rating of Ataxia (SARA) and Balance Evaluation Systems Test (BESTest). Changes were analysed using linear mixed-effect models. SARA scores were stable, indicating slower progression than the expected natural history, through year 6, with significant improvement observed post-intervention in year 2 (p = 0.04). Significant deterioration occurred at year 7 based on pre-intervention scores (p = 0.01), suggesting prolonged sustained benefits for coordination. The BESTest scores revealed an earlier decline, with significant deterioration from year 3 (p = 0.04), which progressed until year 7 (p < 0.01). Annual intensive rehabilitation effectively slowed the progression of ataxic symptoms (SARA) for up to six years, while balance function (BESTest) showed a significant decline from the third year. These findings indicate that an annual rehabilitation schedule is valuable for maintaining coordination but may be insufficient to prevent the progressive decline of balance function in patients with pure cerebellar ataxia.

Keywords: Ataxia; Longitudinal studies; Postural balance; Rehabilitation; Spinocerebellar ataxia.

Fig. 1

Individual longitudinal changes in SARA and BESTest scores over the 7-year follow-up. Note: Values are shown as percentile-transformed scores comparing SARA and BESTest scores (SARA: 0–40 points; BESTest: 0–108 points). The y-axis for SARA scores is inverted because lower scores indicate better function, whereas higher BESTest scores indicate better balance performance. Graphs should be interpreted such that a downward trend indicates worsening of symptoms. SARA, Scale for the Assessment and Rating of Ataxia; BESTest, Balance Evaluation Systems Test