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Table 5 Change from baseline in DAS-II GCA scores by baseline age and DAS-II GCA score subgroups

From: Neurodevelopmental status and adaptive behavior of pediatric patients with mucopolysaccharidosis II: a longitudinal observational study

 

Patient subgroup

 

Baseline age

< 7 years

(n = 42)

Baseline age

≥ 7 years

(n = 13)

Baseline DAS-II GCA score ≤ 70

(n = 18)

Baseline DAS-II GCA score > 70

(n = 26)

Baseline

    

 n

32

12

18

26

 Mean (SD)

78.3 (19.50)

78.7 (18.85)

59.3 (7.01)

91.7 (12.14)

Change from baseline at month 12

    

 n

19

8

7

20

 Mean (SD)

−2.3 (10.07)

2.4 (7.03)

−2.4 (9.83)

−0.4 (9.44)

Change from baseline at month 24

    

 n

14

6

7

13

 Mean (SD)

−4.8 (14.98)

−1.5 (4.59)

−12.1 (14.51)

0.7 (9.38)

  1. Changes from baseline were calculated using data only from patients with data available at both baseline and month 12 or at both baseline and month 24
  2. For DAS-II cluster and composite scores: mean = 100; standard scores of ≤ 69, 70–79, 80–89, and 90–109 indicate very low, low, below average, and average function, respectively
  3. DAS-II, Differential Ability Scales, Second Edition; GCA, General Conceptual Ability; SD, standard deviation
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Orphanet Journal of Rare Diseases

ISSN: 1750-1172