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Table 2 Change from baseline over time in growth parameters by age group (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

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) –
  1. Treatment groups from the study period. Both groups received sapropterin + Phe-restricted diet in the extension period. Age group at study entry used
  2. EOS end of extension period (month 36), ITTE intention-to-treat extension (population), SD standard deviation, SDS standard deviation score
  3. *Measured as maximum occipital frontal head circumference
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Orphanet Journal of Rare Diseases

ISSN: 1750-1172