Individualized exergame training improves postural control in advanced degenerative spinocerebellar ataxia: A rater-blinded, intra-individually controlled trial

Neurorehabilitation therapy in spinocerebellar ataxia type 2: A 24‐week, rater‐blinded, randomized, controlled trial

“…Nearly all of these studies included a small sample size with higher heterogeneity for the disease duration, ataxia severity, and molecular subtype, excepting 3 studies encompassing relatively large cohorts of patients with spinocerebellar degenerations . Only 2 studies have been performed using a controlled design . Thus, the heterogeneity in patient populations and study designs hampers the unbiased interpretation and comparison of existing findings …”

“…8,9 Nevertheless, there are few studies assessing the effect of this therapeutic alternative on SCAs. Nearly all of these studies included a small sample size with higher heterogeneity for the disease duration, ataxia severity, and molecular subtype, [9][10][11][12][13] excepting 3 studies encompassing relatively large cohorts of patients with spinocerebellar degenerations. [14][15][16] Only 2 studies have been performed using a controlled design.…”

“…[14][15][16] Only 2 studies have been performed using a controlled design. 13,16 Thus, the heterogeneity in patient populations and study designs hampers the unbiased interpretation and comparison of existing findings. 9 In view of the higher prevalence of SCA2 in Cuba as result of a founder effect, 17,18 the larger and homogenous population of affected patients provides a good opportunity to evaluate the effect of the rehabilitation using meaningful designs.…”

Towards ecologically valid biomarkers: real-life gait assessment in cerebellar ataxia

“…These ranges are motivated by previous analysis on the responsiveness of the SARA, showing that a change of 1 SARA point can be considered as a clinically important progression 40 . In addition, they are motivated by motor intervention studies demonstrating that current treatment interventions can yield an average improvement of 1.5-2 points on the SARA p&g subscore, and that these effects represent patient-relevant improvements 14,15,41,42 StrideT CV (p=0.02, d =0.86, ROC accuracy 0.72) allowed to distinguish between HC and CA (see Figure 1 and Supplement S2, Table 2). Effect sizes and discrimination performance were smaller than in LBW, as variability in gait measures was generally higher in these unconstrained walking conditions, which was observed in both healthy controls and patients.…”

“…To serve as progression and treatment outcome measures, measures of real-life walking should ideally be able to capture changes that correspond to clinically and everyday-living important differences as well as to treatment effects achievable by current and future ataxia treatment interventions. A change of one point in the SARA score has been shown to reflect a clinically important difference over one year disease course 40 , determined by the patients' by currently available motor rehabilitation interventions14,15,41,42 . Our measure SPcmp yields a strong effect size (Cohen's d=1.2) for differentiating movement patterns when patients differ by two SARA p&g points, and an at least moderate effect size (Cohen's d=0.67) when patients differ by one SARA p&g point, demonstrating that this measure is able to capture clinically important differences.…”