Characteristics, statistics | ‘sapropterin continuous’ (n = 25) | ‘sapropterin extension’ (n = 26) |
---|---|---|
Age (months), mean (SD) | 20.1 (12.1) | 19.8 (11.5) |
Age group, n (%) | ||
< 12 months | 7 (28.0) | 8 (30.8) |
12 to < 24 months | 9 (36.0) | 8 (30.8) |
24 to < 48 months | 9 (36.0) | 10 (38.5) |
Female, n (%) | 10 (40.0) | 12 (46.2) |
Race, n (%) | ||
White | 24 (96.0) | 25 (96.2) |
Asian | 0 | 1 (3.8) |
Other | 1 (4.0) | 0 |
Height (cm), mean (SD) | 81.21 (11.37) | 80.73 (10.92) |
Weight (kg), mean (SD) | 11.14 (3.17) | 11.09 (2.83) |
Age at PKU diagnosis, days | ||
Mean (SD) | 28.2 (83.0) | 31.9 (75.5) |
Min; Max | 1; 425 | 4; 382 |
Blood Phe level at diagnosis (µmol/L), mean (SD) | 738.9 (421.4) | 788.4 (508.3) |
Disease severity* | ||
Classical PKU, n (%) | 4 (16.0) | 5 (19.2) |
Mild PKU, n (%) | 9 (36.0) | 7 (26.9) |
MHP, n (%) | 12 (48.0) | 14 (53.8) |