Table 1 Episode characteristics at baseline
Carglumic acid | NH3 scavengers | Carglumic acid + NH3 scavengers | |
|---|---|---|---|
Episodes, n | 38 | 33 | 27 |
Episodes occurring in males, n (%) | 22 (57.9) | 16 (48.5) | 12 (44.4) |
Episodes occurring in neonates, n (%) | 17 (44.7) | 15 (45.5) | 20 (74.1) |
Episodes with OA diagnosis, n (%) | |||
PA | 19 (50.0) | 27 (81.8) | 14 (51.9) |
MMA | 15 (39.5) | 5 (15.2) | 12 (44.4) |
IVA | 4 (10.5) | 1 (3.0) | 1 (3.7) |
Median (Q1, Q3) age at diagnosis, days | 13.0 (5.0, 33.0) | 9.5 (5.0, 19.5) | 8.0 (5.0, 15.0) |
Median (Q1, Q3) NH3 levels, μmol/L | 199.0 (125.0, 295.0) | 122.0 (91.0, 191.0) | 270.9 (160.0, 429.0) |
Median (Q1, Q3) age at start of episode, days | 50.5 (4.0, 1190.0) | 68.0 (4.0, 1477.0) | 5.0 (4.0, 61.0) |
Median (Q1, Q3) duration of ended episode, days | 6.0 (3.5, 8.0) | 8.5 (5.0, 12.0) | 6.0 (4.0, 7.0) |
Episodes requiring extracorporeal detoxification,a n (%) | 5 (13.2) | 10 (30.3) | 3 (11.1) |