Table 2.
Outcomes across disability stages and ataxia severity.
| FARS disability stages |
p-valueb | Effect sizec (η2, Cramér's V) | |||||
|---|---|---|---|---|---|---|---|
| 1: No disability (n = 5) | 2: Minimal disability (n = 30) | 3: Mild disability (n = 10) | 4: Moderate disability (n = 31) | 5: Severe disability (n = 24) | |||
| Clinical characteristics | |||||||
| Age of onset, mean (SD) | 14.0 (9.0) | 20.6 (13.5) | 20.6 (13.2) | 21.3 (12.2) | 15.6 (5.8) | 0.295 | 0.050 small |
| Ataxia duration (years), mean (SD) | 9.4 (4.3) | 12.8 (8.7) | 9.8 (4.0) | 19.1 (8.3) | 19.1 (8.0) | 0.001 | 0.194 large |
| Ataxia severity (SARA), mean (SD) | 8.5 (3.5) | 13.0 (3.4) | 15.1 (3.2) | 19.8 (4.1) | 23.4 (4.5) | 0.001 | 0.589 large |
| INAS, mean (SD) | 2.6 (0.9) | 3.8 (1.5) | 3.5 (0.7) | 5.1 (1.6) | 5.0 (1.5) | 0.001 | 0.224 large |
| CCAS, mean (SD) | 97.4 (7.4) | 95.4 (12.1) | 96.4 (11.9) | 90.4 (15.5) | 85.8 (12.5) | 0.063 | 0.094 medium |
| Hearing problemsa, n (%) | 0 (0.0) | 8 (26.7) | 4 (44.4) | 11 (36.7) | 17 (73.9) | 0.013 | 0.316 medium |
| Patient-reported outcomes | |||||||
| EQ-5D-5L, mean (SD) | 0.96 (0.06) | 0.84 (0.12) | 0.58 (0.31) | 0.57 (0.26) | 0.59 (0.12) | 0.001 | 0.360 large |
| PROM-Ataxia, mean (SD) | 6.2 (3.8) | 14.8 (7.4) | 20.3 (6.7) | 24.6 (6.9) | 24.6 (4.4) | 0.001 | 0.428 large |
| FARS-ADL, mean (SD) | 6.2 (3.0) | 9.2 (3.6) | 10.8 (2.3) | 14.6 (3.6) | 16.7 (3.6) | 0.001 | 0.490 large |
| WEMWBS, mean (SD) | 40.0 (7.7) | 38.8 (9.3) | 30.8 (12.2) | 34.7 (7.4) | 34.4 (9.1) | 0.126 | 0.086 medium |
| COMATAX, mean (SD) | 8.0 (7.9) | 18.5 (11.7) | 19.4 (6.9) | 27.7 (12.2) | 29.1 (10.3) | 0.001 | 0.242 large |
| Health service utilization | |||||||
| Social care accessa, n (%) | 1 (25.0) | 3 (15.0) | 0 (0.0) | 5 (26.3) | 3 (18.8) | 0.530 | 0.216 small |
| Hospitalizeda, n (%) | 0 (0.0) | 1 (5.0) | 0 (0.0) | 1 (5.3) | 2 (12.5) | 0.714 | 0.177 small |
| Formal care supporta, n (%) | 1 (25.0) | 2 (10.0) | 2 (22.2) | 7 (36.8) | 11 (68.8) | 0.006 | 0.463 medium |
| Omaveloxolone intakea, n (%) | 3 (60.0) | 8 (26.7) | 0 (0.0) | 13 (41.9) | 9 (37.5) | 0.076 | 0.291 small |
| Informal care | |||||||
| Informal care provisiona, n (%) | 0 (0.0) | 9 (45.0) | 6 (66.7) | 15 (79.0) | 10 (71.4) | 0.020 | 0.421 medium |
| Hours per week, mean (SD) | 0.0 (0.0) | 5.4 (12.3) | 14.1 (25.7) | 17.0 (28.9) | 20.2 (19.6) | 0.199 | 0.092 medium |
| Caregiver employment reduction, n (%) | 1 (25.0) | 3 (15.0) | 0 (0.0) | 4 (21.1) | 3 (21.4) | 0.639 | 0.196 small |
| Health-care costs in € values | |||||||
| Payer perspective, mean (SD) | 4526 (3088) | 10,394 (21,424) | 4318 (3457) | 5702 (8803) | 30,820 (46,674) | 0.042 | 0.143 large |
| Societal perspective, mean (SD) | 6088 (4429) | 18,698 (27,770) | 26,134 (38,598) | 32,084 (46,813) | 58,448 (50,030) | 0.039 | 0.146 large |
Abbreviations: FA, Friedreich's Ataxia; SD, standard deviation; CCAS, Cerebellar Cognitive Affective Syndrome Scale; FARS-ADL, Friedreich's Ataxia Rating Scale–Activities of Daily Living Subscale; WEMWBS, Warwick–Edinburgh Mental Well-being Scale; COMATAX, scale for the psychosocial impact of communication disabilities; INAS, Inventory of Non-Ataxia Signs; SARA, Scale for the Assessment and Rating of Ataxia.
During the last 2 months;a HearWho test, summarizing possible and likely hearing problems.
One-way ANOVA (with η2 as effect size) were used for continuous variables, Pearson χ2 tests (with Cramér's V as effect size) for categorical variables, bold numbers indicate a statistically significant difference (p ≤ 0.05).
Cramers V were used for percentage values, Eta-square (η2) for numeric values.