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Table 1 Demographics and baseline characteristics (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

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)

  1. 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
  2. ITTE intention-to-treat extension (population), MHP mild hyperphenylalaninaemia, Phe phenylalanine, PPE per protocol extension (population), PKU phenylketonuria
  3. *Defined as: MHP, 120–599 µmol/L Phe; mild PKU, 600–1199 µmol/L Phe; classical PKU, ≥ 1200 µmol/L Phe