Skip to main content

Table 4 Neurodevelopmental status assessment by age group (Wechsler preschool and primary scale of intelligence; ITTE population)

From: Long-term efficacy and safety of sapropterin in patients who initiated sapropterin at < 4 years of age with phenylketonuria: results of the 3-year extension of the SPARK open-label, multicentre, randomised phase IIIb trial

Timepoint

Statistics

‘sapropterin continuous’ (n=25)

‘sapropterin extension’ (n = 26)

Age

< 12 Months

12–< 24 Months

24–< 48 Months

< 12 Months

12–< 24 Months

24–< 48 Months

Full scale IQ (Wechsler scale of intelligence)

       

 Baseline

n (missing)

0 (7)

0 (9)

2 (7)

0 (8)

1 (9)

0 (8)

 

Mean (SD)

115.50 (17.68)

124.00

 Month 12

n (missing)

0 (7)

0 (9)

9 (0)

0 (8)

7 (3)

0 (8)

 

Mean (SD)

110.44 (17.58)

111.43 (26.22)

 Month 24

n (missing)

0 (7)

3 (6)

4 (5)

0 (8)

4 (6)

5 (3)

 

Mean (SD)

98.00 (20.07)

105.50 (12.12)

115.25 (7.27)

94.40 (19.69)

 EOS

n (missing)

6 (1)

1 (8)

4 (5)

5 (3)

5 (5)

4 (4)

 

Mean (SD)

120.67 (14.67)

94.00

95.25 (8.26)

108.60 (14.81)

108.20 (14.75)

88.25 (14.17)

Performance IQ (Wechsler scale of intelligence)

       

 Baseline

n (missing)

0 (7)

0 (9)

2 (7)

0 (8)

1 (9)

0 (8)

 

Mean (SD)

 

114.00

 Month 12

n (missing)

0 (7)

0 (9)

9 (0)

0 (8)

7 (3)

0 (8)

 

Mean (SD)

 

96.43 (20.26)

 Month 24

n (missing)

0 (7)

3 (6)

4 (5)

0 (8)

4 (6)

5 (3)

 

Mean (SD)

92.33 (22.01)

105.25 (4.99)

111.00 (12.19)

95.40 (22.79)

 EOS

n (missing)

6 (1)

1 (8)

4 (5)

5 (3)

5 (5)

4 (4)

 

Mean (SD)

116.33 (15.28)

90.00

93.75 (11.24)

96.80 (13.41)

103.40 (18.17)

89.75 (15.41)

  1. Treatment Groups from the Study Period. Both groups received sapropterin + Phe-restricted diet in the extension period. Age group at study entry used. Missing could be either have been due to missing analysis values or age out of range for assessment
  2. EOS end of study, IQ intelligence quotient, ITTE Intention-to-treat extension (population), SD standard deviation