Table 2.
Demographic characteristics of study patients
| Characteristic | Study PKU-015 (N = 80)a | Study PKU-004 (N = 78)a | Pooled studies PKU-015 and PKU-004 (N = 156)a |
|---|---|---|---|
| Age (years) | 3.28 (2.01) [0.107–6.98] | 21.1 (9.64) [9–50] | 12 (11.3) [0.107–50] |
| Age category [n (%)] | |||
| <1 year | 10 (12.5) | 0 | 10 (6.4) |
| 1 to <2 years | 14 (17.5) | 0 | 14 (9.0) |
| 2 to <4 years | 28 (35.0) | 0 | 28 (17.9) |
| 4 to <7 years | 28 (35.0) | 0 | 28 (17.9) |
| 7 to <12 years | 0 | 10 | 10 (6.4) |
| ≥12 years | 0 | 68 | 66 (42.3) |
| Sex [n (%)] | |||
| Females | 49 (61.3) | 33 (42.3) | 80 (51.3) |
| Males | 31 (38.7) | 45 (57.7) | 76 (48.7) |
| Ethnicity [n (%)] | |||
| Non-Hispanic | 78 (97.5) | 76 | 152 (97.4) |
| Hispanic | 2 (2.5) | 2 | 4 (2.6) |
| Body weight (kg) | 15.9 (6.36) [4.5–41.9] | 67.2 (21.8) [28.2–144] | 40.9 (30.3) [4.5–144] |
| Height (cm) | 95.6 (18.3) [56–128] | 165 (13.3) [126–191] | 129 (38.2) [56–191] |
| Body surface area (m2) | 0.635 (0.19) [0.259–1.18] | 1.72 (0.31) [1.05–2.65] | 1.17 (0.604) [0.259–2.65] |
| ALT (IU/L) | 22.4 (8.15) [13–63] | 28.4 (18.3) [11–127] | 25.4 (14.5) [11–127] |
| AST (IU/L) | 38.1 (8.75) [23–63] | 25.7 (5.8) [14–43] | 32.2 (9.65) [14–63] |
| Bilirubin (mg/dL) | 7.51 (4.4) [1.71–34.2] | 0.55 (0.33) [0.1–1.9] | 4.12 (4.7) [0.1–34.2] |
| Albumin (g/dL) | 4.19 (0.274) [3.6–4.9] | 4.49 (0.23) [4–5] | 4.33 (0.29) [3.6–5] |
| CLCR (mL/min)b | 57.1 (22.2) [9.39–120] | 114 (26) [48–231] | 87.8 (45.1) [9.39–276] |
| Baseline phenylalanine (μmol/L) | 324 (140) [57.5–768] | 811 (393) [53–2190] | 562 (378) [53–2190] |
ALT alanine aminotransferase, AST aspartate aminotransferase, CL CR creatinine clearance
aValues are presented as mean (standard deviation) [range], unless specified otherwise
bCalculated from plasma creatinine