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Table 3 Dynamics in CSF CHIT1 levels during 14 months of nusinersen treatment

From: Increased chitotriosidase 1 concentration following nusinersen treatment in spinal muscular atrophy

 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**

  1. CHIT1 Chitotriosidase 1 concentration, CSF cerebrospinal fluid, IQR interquartile range, n.s. not significant
  2. *p < 0.05; **p < 0.01; ***p < 0.001; ****p < 0.0001 calculated by Wilcoxon signed-rank test