|
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)
|
- Note that in small children, even common childhood illnesses can cause temporary loss of metabolic control, which may lead a physician to stop dietary adjustments or to stop drug treatment. Such patients were excluded from analysis in the PPE population. Six patients (> 10%) moved from the PPE to ITTE population during the extension period
- ITTE intention-to-treat extension (population), MHP mild hyperphenylalaninaemia, Phe phenylalanine, PPE per protocol extension (population), PKU phenylketonuria
- *Defined as: MHP, 120–599 µmol/L Phe; mild PKU, 600–1199 µmol/L Phe; classical PKU, ≥ 1200 µmol/L Phe
