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) |
IVA Isovaleric aciduria, MMA Methylmalonic aciduria, NH3 Ammonia, OA Organic aciduria, PA Propionic aciduria
aHaemodialysis, haemofiltration and peritoneal dialysis. Full analysis set