Table 1 Comparison between the subjects with or without clinical improvement Clinical, and cerebrospinal fluid profiles of the patients.
From: Nilotinib treatment outcomes in autosomal dominant spinocerebellar ataxia over one year
Total (N = 32) | Responsive (N = 20) | Non-reponsive (N = 12) | P | |
|---|---|---|---|---|
Sex (men, %) | 14 (43.8%) | 10 (50.0%) | 4 (33.3%) | 0.581 |
Age (years) | 42 [30.5–49.5] | 39.5 [30.5–46] | 48.5 [30.5–59] | 0.267 |
Type | 0.389 | |||
SCA2 (%) | 8 (25.0%) | 5 (25.0%) | 3 (25.0%) | |
SCA3 (%) | 10 (31.2%) | 4 (20.0%) | 6 (50.0%) | |
SCA6 (%) | 8 (25.0%) | 6 (30.0%) | 2 (16.7%) | |
SCA7 (%) | 3 (9.4%) | 3 (15.0%) | 0 (0.0%) | |
SCA17 (%) | 2 (6.2%) | 1 (5.0%) | 1 (8.3%) | |
SCA8 (%) | 1 (3.1%) | 1 (5.0%) | 0 (0.0%) | |
Trinucleotide repeat length (long allele) | 45 [39–70.5] | 45 [32–67.5] | 66 [40.5–70.5] | 0.447 |
SCA2 (n = 8) | 43 [41.25–45] | 45 [41. 5–45.5] | 42 [39–44] | 0.292 |
SCA3 (n = 10) | 71.5 [67.0–73.25] | 72.5 [72.0–73.75] | 68.5 [66.5–72.25] | 0.089 |
SCA6 (n = 8) | 23.5 [22.25–25.0] | 24 [22.75–28.5] | 23.0 | 0.301 |
SCA7 (n = 3) | 45 [42–53] | 45 [42–53] | – | – |
SCA8 (n = 2) | 94.5 | 81 | 108 | – |
SCA17 (n = 1) | 63 | 63 | – | – |
Years from symptom onset (years) | 4.5 [2–8] | 3 [2–5.5] | 5.5 [2.5–9] | 0.169 |
Dosage of nilotinib | 0.329 | |||
150 mg/day (%) | 6 (18.8%) | 5 (25.0%) | 1 (8.3%) | |
200 mg/day (%) | 3 (9.4%) | 1 (5.0%) | 2 (16.7%) | |
300 mg/day (%) | 23 (71.9%) | 14 (70.0%) | 9 (75.0%) | |
Baseline severity | ||||
SARA score | 10.5 [8.8–13.2] | 10.8 [9.5–14.5] | 9.8 [8–11.8] | 0.283 |
FARS I score | 2 [2–2] | 2 [2–2] | 2 [2–2.5] | 0.311 |
FARSII score | 9.5 [6.5–13] | 8.5 [5.5–13.5] | 10.5 [9–12] | 0.482 |
Barthel index | 1.5 [0–3] | 1 [0–4] | 2 [0–3] | 0.734 |
Follow-up evaluations | ||||
SARA score change-1 M | 0.5 [− 1–0.8] | 0.5 [− 0.5–2] | -0.5 [− 1–− 0.2] | 0.017* |
SARA score change-3 M | 1 [0–1.5] | 1.5 [1–3] | − 0.5 [− 1–0.5] | < 0.001** |
SARA score change-6 M | 1 [− 0.8–2] | 2 [1.2–2.8] | − 0.8 [− 1.5–− 0.5] | < 0.001** |
SARA score change-12 M | 1.5 [− 0.5–2.2] | 2 [1.5–3] | − 0.8 [− 2–− 0.2] | < 0.001** |
FARS I score change-1 M | 0 [0–0] | 0 [0–0] | 0 [0–0] | 1.000 |
FARS I score change-3 M | 0 [0–0] | 0 [0–1] | 0 [0–0] | 0.737 |
FARS I score change-6 M | 0 [0–1 | 0 [0–1] | 0 [0–1] | 0.851 |
FARS I score change-12 M | 0 [0–0] | 0 [0–0] | 0 [0–1] | 0.442 |
FARS I score improvement (%) | 8 (25.0%) | 5 (25.0%) | 3 (25.0%) | 1.000 |
FARS II score change-1 M | 1 [0–2] | 1 [0–2] | 0.5 [− 0.5–2] | 0.553 |
FARS II score change-3 M | 1 [0–2.5] | 1.5 [0–3] | 0.5 [− 0.5–2] | 0.237 |
FARS II score change-6 M | 1 [0–3] | 1 [0–3.5] | 0 [− 0.5–2] | 0.333 |
FARS II score change-12 M | 1 [0–3] | 1 [− 1–2] | 1.5 [0–3.5] | 0.399 |
FARS II score improvement (%) | 20 (62.5%) | 12 (60.0%) | 8 (66.7%) | 1.000 |
Barthel index change-1 M | 0 [0–1] | 0 [0–1] | 0 [0–0] | 0.207 |
Barthel index change-3 M | 0 [0–1] | 0 [0–1] | 0 [− 0.5–0] | 0.017* |
Barthel index change-6 M | 0 [0–0] | 0 [0–0.5] | 0 [− 1–0] | 0.098 |
Barthel index change-12 M | 0 [0–0.5] | 0 [0–1] | 0 [− 0.5–0] | 0.411 |
Barthel index improvement (%) | 8 (25.0%) | 6 (30.0%) | 2 (16.7%) | 0.673 |
