Table 4.
Exposure to final treatment received during follow up period (exposed population)
| OTC (n = 57) | CPS1 (n = 15) | NAGS (n = 4) | ORNT1 (n = 3) | Total (N = 79) | |
|---|---|---|---|---|---|
| Last treatment received, n (%) | |||||
| Arginine | 10 (17.5) | 1 (6.7) | 2 (50.0) | 1 (33.3) | 14 (17.7) |
| Citrulline | 17 (29.8) | 4 (26.7) | 2 (50.0) | 1 (33.3) | 24 (30.4) |
| Arginine + citrulline | 30 (52.6) | 10 (66.7) | 0 | 1 (33.3) | 41 (51.9) |
| Duration of exposure to last treatment, years | |||||
| Arginine and/or citrulline | |||||
| Mean (SD) | 8.1 (6.3) | 7.1 (5.0) | 2.8 (3.0) | 10.2 (13.9) | 7.7 (6.3) |
| Median (range) | 6.8 (0.0–23.9) | 6.4 (0.2–15.7) | 2.1 (0.0–6.9) | 2.5 (1.9–26.1) | 6.3 (0.0–26.1) |
| Arginine | |||||
| Mean (SD) | 9.7 (7.2) | 6.9 (NA) | 2.1 (0.9) | 1.9 (NA) | 7.9 (6.9) |
| Median (range) | 9.8 (0.7–23.9) | 6.9 (6.9–6.9) | 2.1 (1.4–2.7) | 1.9 (1.9–1.9) | 7.1 (0.7–23.9) |
| Citrulline | |||||
| Mean (SD) | 6.3 (5.4) | 3.5 (2.1) | 3.4 (4.8) | 2.5 (NA) | 5.5 (4.9) |
| Median (range) | 5.3 (0.1–19.2) | 2.7 (2.0–6.4) | 3.4 (0.0–6.9) | 2.5 (2.5–2.5) | 4.6 (0.0–19.2) |
| Arginine + citrulline | |||||
| Mean (SD) | 8.5 (6.4) | 8.5 (5.4) | NA | 26.1 (NA) | 9.0 (6.6) |
| Median (range) | 7.0 (0.0–22.9) | 10.3 (0.2–15.7) | NA | 26.1 (26.1–26.1) | 7.9 (0.0–26.1) |
CPS1, carbamoyl phosphate synthetase 1; NAGS, N-acetylglutamate synthase; NA, not applicable; ORNT1, ornithine translocase; OTC, ornithine transcarbamylase; SD, standard deviation