Table 5.
Treatment initiation, dosage and treatment duration in the efficacy population
| Isovaleric aciduria (n = 4) | Methylmalonic aciduria (n = 25) | Propionic aciduria (n = 19) | Neonates(n = 29) | Non-neonates(n = 19) | Total(n = 48) | |
|---|---|---|---|---|---|---|
| Time to treatment initiation | ||||||
| Mean, days (SD) | 0.8 (1.0) | 2.5 (5.5) | 4.2 (9.5) | 1.5 (2.8) | 5.3 (10.6) | 3.0 (7.2) |
| Median (range) | 0.5 (0–2) | 0.0 (0–24) | 2.0 (0–41) | 1.0 (0–14) | 1.0 (0–41) | 1.0 (0–41) |
| First dose | ||||||
| Mean, mg/kg (SD) | 99.0 (63.3) | 78.2 (64.9) | 119.5 (83.2) | 106.7 (75.3) | 80.4 (70.5) | 96.3 (73.8) |
| Median, mg/kg (range) | 81.6 (50.0–182.7) | 51.0 (13.7–236.2) | 106.0 (13.3–303.0) | 100.0 (13.7–303.0) | 50.0 (13.3–256.4) | 75.5 (13.3–303.0) |
| Initial 24-h dose | ||||||
| Mean, mg/kg (SD) | 216.0 (217.9) | 160.6 (108.3) | 209.3 (186.9) | 214.1 (179.1) | 139.2 (81.7) | 184.5 (151.8) |
| Median, mg/kg (range) | 131.6 (62.5–538.5) | 129.0 (41.1–470.8) | 190.2 (53.1–909.1) | 170.2 (41.1–909.1) | 100 (45.3–333.3) | 156.4 (41.0–909.1) |
| Treatment duration | ||||||
| Mean, days (SD) | 3.5 (1.7) | 6.1 (4.6) | 5.2 (5.0) | 4.9 (3.5) | 6.5 (5.9) | 5.5 (4.6) |
| Median, days (range) | 3.5 (2–5) | 5.0 (1–15) | 3.0 (1–15) | 4.0 (1–15) | 3.0 (1–15) | 4.0 (1–15) |
SD, standard deviation