Table 2 Change from baseline over time in growth parameters by age group (ITTE population)
Timepoint | ‘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 | |
Body mass index SDS, mean (SD) | ||||||
Baseline value | − 0.39 (0.90) | 0.56 (0.49) | 0.78 (0.55) | 0.26 (0.87) | 0.70 (0.91) | 0.30 (0.69) |
Baseline—month 12 | 0.96 (0.91) | 0.21 (0.94) | 0.17 (0.42) | 0.21 (0.68) | 0.23 (0.80) | − 0.02 (0.50) |
Baseline—month 24 | 0.46 (0.56) | − 0.03 (0.74) | − 0.16 (0.46) | 0.47 (0.75) | − 0.11 (0.41) | − 0.23 (0.51) |
Baseline—EOS | 0.45 (0.91) | 0.47 (0.74) | – | 0.53 (0.89) | − 0.37 (0.07) | – |
Head circumference (cm), mean (SD)* | ||||||
Baseline value | 42.56 (2.52) | 47.83 (2.25) | 49.33 (2.42) | 45.75 (1.89) | 48.75 (2.92) | 48.88 (1.65) |
Baseline—month 12 | 6.09 (2.06) | 1.66 (1.23) | 1.33 (0.98) | 2.96 (1.59) | 1.31 (1.39) | 0.93 (2.62) |
Baseline—month 24 | 6.77 (2.44) | 2.86 (0.80) | 1.67 (1.03) | 3.59 (1.01) | 1.80 (1.10) | 2.18 (1.61) |
Baseline—EOS | 7.95 (2.06) | 3.54 (1.09) | 2.68 (1.64) | 4.47 (1.23) | 1.36 (0.84) | 2.63 (1.41) |
Height SDS, mean (SD) | ||||||
Baseline value | 0.43 (0.94) | − 0.28 (1.42) | − 0.49 (1.13) | 0.11 (1.03) | − 0.19 (0.66) | − 0.35 (1.03) |
Baseline—month 12 | − 0.38 (0.58) | − 0.09 (0.80) | 0.12 (0.66) | 0.01 (0.65) | 0.11 (0.48) | 0.10 (0.57) |
Baseline—month 24 | − 0.46 (0.67) | 0.28 (0.53) | − 0.37 (0.92) | − 0.16 (0.75) | 0.19 (0.74) | 0.11 (0.52) |
Baseline—EOS | − 0.39 (0.81) | − 0.12 (0.62) | – | − 0.18 (0.55) | 0.31 (0.62) | – |
Weight SDS, mean (SD) | ||||||
Baseline value | − 0.04 (0.74) | 0.21 (0.95) | 0.22 (0.73) | 0.25 (0.71) | 0.38 (0.63) | − 0.01 (0.67) |
Baseline—month 12 | 0.48 (0.48) | 0.06 (0.60) | 0.19 (0.31) | 0.14 (0.36) | 0.21 (0.38) | 0.03 (0.25) |
Baseline—month 24 | 0.10 (0.50) | − 0.01 (0.67) | − 0.34 (0.65) | 0.22 (0.61) | 0.01 (0.33) | − 0.10 (0.11) |
Baseline—EOS | 0.12 (0.34) | 0.15 (0.83) | – | 0.22 (0.66) | − 0.10 (0.39) | – |