Table 3.
Hyperammonemic Crises During GPB Treatment as Compared with Pre-Enrollment
| 12-month pre-enrollment (NaPBA) (N=49) |
12 months on study (GPB) (N=49) |
|
|---|---|---|
| Patients with HA Crises: n (%) | 21 (42.9) | 12 (24.5) |
| Age 29 days- <2 years (N=7) | 5 (71.4) | 2 (28.6) |
| Age 2–5 years (N=16) | 10 (62.5) | 5 (31.3) |
| Age 6–11 years (N=17) | 2 (11.8) | 4 (23.5) |
| Age 12–17 years (N=9) | 4 (44.4) | 1 (11.1) |
| Total number of HA Crises | 38 | 17 |
| Age 29 days- <2 years (N=7) | 6 | 2 |
| Age 2–5 years (N=16) | 23 | 10 |
| Age 6–11 years (N=17) | 3 | 4 |
| Age 12–17 years (N=9) | 6 | 1 |
| Number of HA Crises per Patient: mean (SD) | 0.78 (1.279) | 0.35 (0.830) |
| Age 29 days- <2 years (N=7) | 0.86 (0.690) | 0.29 (0.488) |
| Age 2–5 years (N=16) | 1.44 (1.825) | 0.63 (1.310) |
| Age 6–11 years (N=17) | 0.18 (0.529) | 0.24 (0.437) |
| Age 12–17 years (N=9) | 0.67 (1.000) | 0.11 (0.333) |
| Precipitating Factors (% of all HACs) | ||
| Infection | 6 (15.8) | 2 (11.8) |
| Intercurrent illness | 10 (26.3) | 6 (35.3) |
| Non-compliance with medication | 4 (10.5) | 0 |
| Non-compliance with diet | 4 (10.5) | 0 |
| Other | 14 (36.8) | 3 (17.6) |
| None | 7 (18.4) | 7 (41.2) |
| Clinical Symptoms: n (% of all HACs) | ||
| Abnormal neurology | 0 | 2 (11.8) |
| Brain edema | 0 | 1 (5.9) |
| Chronic migraine headaches | 2 (5.3) | 0 |
| Episodic lethargy | 14 (36.8) | 5 (29.4) |
| Protein intolerance | 2 (5.3) | 4 (23.5) |
| Psychosis (episodic) | 1 (2.6) | 0 |
| Recurrent vomiting | 24 (63.2) | 13 (76.5) |
| Other | 18 (47.4) | 4 (23.5) |
| None | 1 (2.6) | 1 (5.9) |