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Table 3 Cognitive categoriesa at the end of year 1 and year 2

From: A post hoc analysis of Projected Retained Ability Scores (PRAS) for the longitudinal assessment of cognitive functioning in patients with neuronopathic mucopolysaccharidosis II receiving intrathecal idursulfase-IT

 

Overall population

Subpopulation

(aged < 6 years at baseline)

 

Idursulfase-IT

in RCT

(n = 34)

No idursulfase-IT

in RCT

(n = 15)

Idursulfase-IT

in RCT

(n = 28)

No idursulfase-IT

in RCT

(n = 12)

Cognitive category

Year 1 (n = 29)

Year 1 (n = 15)

Year 1 (n = 25)

Year 1 (n = 12)

Above-average cognitive growth rate

2/29 (6.9%)

0

2/25 (8.0%)

0

Average cognitive growth rate

19/29 (65.5%)

8/15 (53.3%)

17/25 (68.0%)

6/12 (50.0%)

Below-average cognitive growth rate

0

0

0

0

Plateauing cognitive functioning

6/29 (20.7%)

4/15 (26.7%)

5/25 (20.0%)

4/12 (33.3%)

Deteriorating cognitive functioning

2/29 (6.9%)

3/15 (20.0%)

1/25 (4.0%)

2/12 (16.7%)

Cognitive category

Year 2 (n = 27)

Year 2 (n = 12)

Year 2 (n = 25)

Year 2 (n = 11)

Above-average cognitive growth rate

1/27 (3.7%)

0

1/25 (4.0%)

0

Average cognitive growth rate

12/27 (44.4%)

5/12 (41.7%)

11/25 (44.0%)

4/11 (36.4%)

Below-average cognitive growth rate

1/27 (3.7%)

1/12 (8.25%)

1/25 (4.0%)

1/11 (9.1%)

Plateauing cognitive functioning

10/27 (37.0%)

5/12 (41.7%)

9/25 (36.0%)

5/11 (45.5%)

Deteriorating cognitive functioning

3/27 (11.1%)

1/12 (8.25%)

3/25 (12.0%)

1/11 (9.1%)

  1. aPatients with a missing cognitive development category were omitted. There was no statistically significant difference between treatment groups for cognitive category distribution at 1 year (p = 0.0731) or 2 years (p = 0.4426) in the overall population. In the subpopulation aged < 6 years at baseline, there was a marginally significant difference between treatment groups for cognitive category distribution at 1 year (p = 0.0480) but no statistically significant difference at 2 years (p = 0.8579)
  2. IT intrathecal; RCT randomized controlled trial