Table 1.
Age (Years) | Weight (kg) | SUM METAB (µmol/L) | Change SUM METAB % | cAKUSSI | mAKUSSI | OCH SC | |
---|---|---|---|---|---|---|---|
Control group | |||||||
V1 | 47.6 ± 10.1 | 74.1 ± 15.5 | 42,716 ± 15,212 | 80.4 ± 33.3 † | 54.0 ± 24.9 * | 18.0 ± 10.9 | |
V2 | 48.1 ± 10.0 | 74.7 ± 15.6 | 38,829 ± 19,257 | −9.1 ± 36.5 | |||
V3 | 48.8 ± 10.0 | 74.3 ± 15.6 | 36,485 ± 12,065 | −9.0 ± 37.0 | 79.2 ± 34.8 | 54.8 ± 25.6 | 17.8 ± 10.9 |
V4 | 49.8 ± 10.1 | 76.0 ± 15.7 | 37,503 ± 10,428 | −11.8 ± 28.2 | 83.1 ± 33.9 | 57.2 ± 26.1 | 17.8 ± 10.7 |
V5 | 50.6 ± 10.1 | 74.9 ± 14.6 | 40,699 ± 13,268 | 0.9 ± 47.3 | 90.1 ± 37.2 | 63.4 ± 30.3 | 19.4 ± 10.7 |
V6 | 51.6 ± 10.1 | 73.8 ± 15.0 | 41,020 ± 11,724 | 0.2 ± 38.6 | 95.7 ± 36.4 | 67.0 ± 29.9 | 20.3 ± 11.2 |
Nitisinone group | |||||||
V1 | 49.0 ± 11.3 | 74.8 ± 14.8 | 42,268 ± 14,177 **** | 86.9 ± 34.6 | 56.6 ± 27.2 | 21.4 ± 10.7 | |
V2 | 49.2 ± 11.3 | 75.7 ± 15.1 | 91,145 ± 29,629 | 131 ± 106 | |||
V3 | 49.8 ± 11.3 | 78.1 ± 15.0 | 82,204 ± 20,743 | 110 ± 86 | 84.2 ± 34.0 | 57.2 ± 27.3 | 20.9 ± 11.1 |
V4 | 50.8 ± 11.3 | 78.6 ± 15.3 | 82,343 ± 21,724 | 110 ± 85 | 91.1 ± 35.6 | 62.1 ± 28.3 | 21.1 ± 10.6 |
V5 | 51.3 ± 11.3 | 78.6 ± 15.9 | 84,610 ± 22,801 | 111 ± 64 | 88.8 ± 38.8 | 61.2 ± 31.1 | 20.2 ± 10.8 |
V6 | 52.6 ± 11.4 | 78.4 ± 16.1 | 80,874 ± 24,976 | 105 ± 65 | 94.8 ± 37.3 | 66.4 ± 31.2 | 20.3 ± 10.8 |
V1–V6 refers to visits to study sites at baseline, 3, 12, 24, 36 and 48 months; values are shown as Mean ± SD; statistical comparisons within control or nitisinone group denotes whether there were differences within the group across visits and to what degree indicated as: †—p = 0.058; * <0.05; ** <0.01; *** <0.001; **** <0.0001.