Table 2.
Treatment-related adverse events that were considered possibly, probably, or definitely related to study therapy for children treated on NCT02992964 (n = 11).
| Number of patients | |||
|---|---|---|---|
| Adverse event | All grades | Grade 3 | Grade 4 |
| Headache | 3 | 1 | 0 |
| Fatigue | 3 | 0 | 0 |
| Hydrocephalus | 1 | 0 | 1 |
| Papilledema | 1 | 0 | 0 |
| Other neurologica | 2 | 0 | 0 |
| Lipase increased | 1 | 1 | 1 |
| Pancreatitis | 1 | 1 | 0 |
| Other gastrointestinalb | 3 | 0 | 0 |
| Other skinc | 2 | 0 | 0 |
| Otherd | 3 | 0 | 0 |
| Lymphopenia | 1 | 1 | 0 |
| Other laboratorye | 1 | 0 | 0 |
aGait disturbance, dizziness, tremor, seizure.
bVomiting, nausea, diarrhea, blood in stool, gastritis.
cAlopecia, dry skin.
dDyspnea, flu-like symptoms, noninfective cystitis, arthralgia.
eAlanine aminotransferase increased, hypokalemia, hyponatremia, anemia.