Table 2 Demographic and baseline characteristics (ITT population)
Characteristic | Sapropterin + Phe- restricted diet (n = 27) | Phe-restricted diet only (n = 29) |
|---|---|---|
Age, months | ||
mean±SD | 21.1±12.3 | 21.2±12.0 |
min; max | 2; 47 | 2; 44 |
Age group, n (%) | ||
<12 months | 7 (25.9) | 8 (27.6) |
12 − <24 months | 9 (33.3) | 9 (31.0) |
24 − <48 months | 11 (40.7) | 12 (41.4) |
Males, n (%) | 16 (59.3) | 14 (48.3) |
Height, cm | ||
mean±SD | 82.0±11.3 | 82.3±11.6 |
min; max | 59; 108 | 57; 105 |
Weight, kg | ||
mean±SD | 11.3±3.1 | 11.3±2.8 |
min; max | 5; 20 | 6; 16 |
BMI, kg/m2 | ||
mean±SD | 16.5±1.0 | 16.5±1.4 |
min; max | 14; 18 | 14; 20 |
Age at PKU diagnosis, days | ||
mean±SD | 27.2±79.8 | 32.6±72.2 |
min; max | 1; 425 | 4; 382 |
Blood Phe concentration at diagnosis, μmol/L | ||
mean±SD | 780.3±480.7 | 879.9 ± 596.5 |
min; max | 191; 2062 | 221; 2600 |
PKU severitya n (%) | ||
Classical PKU | 5 (18.5) | 7 (24.1) |
Mild PKU | 10 (37.0) | 8 (27.6) |
Mild HPA | 12 (44.4) | 14 (48.3) |