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Table 1 Demographic and disease characteristics

From: Cognitive performance of adult patients with SMA before and after treatment initiation with nusinersen

 

Total

(n = 23)

SMA type 2

(n = 9)

SMA type 3

(n = 14)

SMA type 2 vs. SMA type 3

Age at baseline, years

   

t(21) = − 2.29;p= 0.0331; d= 0.98

 Mean ± SD

 Median (Range)

38.1 ± 11.4

35.0 (18–57)

31.9 ± 10.8

30.0 (18–54)

42.1 ± 10.2

42.5 (22–57)

 

Sex, n (%)

   

p = 0.6692; OR = 2.00

 Female

10 (43.5)

3 (33.3)

7 (50.0)

 

 Male

13 (56.5)

6 (66.7)

7 (50.0)

 

SMN2 copy number, n (%)

   

U= 28.5;Z= − 2.464;p= 0.0283; r= 0.51

 2

2 (8.7)

1 (11.1)

1 (7.1)

 

 3

13 (56.5)

8 (88.9)

5 (35.7)

 

 4

8 (34.8)

0 (0.0)

8 (57.1)

 

Mobility, n (%)

   

p = 0.1162; OR = 1.56

 Ambulatory

5 (21.7)

0 (0.0)

5 (37.5)

 

 Non-ambulatory

18 (78.3)

9 (100.0)

9 (64.3)

 

Disease duration, years

   

U = 43.5; Z = − 1.231; p = 0.2243; r = 0.26

 Mean ± SD

 Median (Range)

34.6 ± 11.1

32.0 (16–54)

31.0 ± 11.0

29.3 (16–54)

36.9 ± 10.8

34.9 (20–49)

 

Education level, years

   

t(21) = 1.09; p = 0.2581; d = 0.48

 Mean ± SD

 Median (Range)

13.5 ± 3.1

13.0 (9–19)

14.4 ± 3.6

13.0 (9–19)

12.9 ± 2.7

12.5 (10–18)

 
  1. SMA: spinal muscular atrophy; SMN2: survival motor neuron 2 gene; SD: standard deviation; n: number; 1calculated by one-sample t-test; 2calculated by Fisher’s exact test; 3calculated by Mann-Whitney U-test; p values < 0.05 considered statistically significant; Cohen’s effect sizes of r = 0.10, r = 0.30 and r = 0.50 as threshold for small, medium and large effects respectively [15]