Table 3.
Dynamics in CSF CHIT1 levels during 14 months of nusinersen treatment
| CSF CHIT1 (pg/mL) | 14-month analysis | ||||
|---|---|---|---|---|---|
| n | Median (IQR) | Difference versus baseline | p value | ||
| Median (range) | (%) | ||||
| Overall | 58 | 2963 (1726–4179) | 775 (− 2713 to 11,103) | + 43 | < 0.0001**** |
| Height < 131 cm | 8 | 6370 (3328–9980) | 4363 (826 to 11,103) | + 309 | 0.011719* |
| Height > 131 cm | 50 | 2759 (1699–4014) | 545 (− 2713 to 3900) | + 29 | 0.001747** |
| SMA type 1 | 6 | 2605 (1073–5731) | 1675 (− 114 to 7086) | + 180 | n.s |
| SMA type 2 | 24 | 3026 (1744–4335) | 897 (− 1154 to 11,103) | + 51 | 0.000829*** |
| SMA type 3 | 28 | 2847 (1710–4093) | 543 (− 2713 to 3087) | + 21 | n.s |
| < 4 SMN2 copies | 30 | 3087 (1992–4528) | 1066 (− 1536 to 8220) | + 58 | 0.000960*** |
| ≥ 4 SMN2 copies | 13 | 2103 (1696–3789) | 495 (− 2713 to 2303) | + 22 | n.s |
| Children | 19 | 2909 (1860–5091) | 984 (− 1536 to 8220) | + 76 | 0.001592** |
| Adults | 39 | 3016 (1702–4088) | 495 (− 2713 to 11,103) | + 24 | 0.007929** |
CHIT1 Chitotriosidase 1 concentration, CSF cerebrospinal fluid, IQR interquartile range, n.s. not significant
*p < 0.05; **p < 0.01; ***p < 0.001; ****p < 0.0001 calculated by Wilcoxon signed-rank test