Table 2.
TEAEs
| Adverse events | All patients (N = 17) | |
|---|---|---|
| Summary of TEAEs, no. of patients (%) | ||
| At least 1 adverse event | 16 (94.1) | |
| At least 1 adverse event of grade ≥ 3 | 7 (41.2) | |
| At least 1 serious adverse event | 6 (35.3) | |
| At least 1 serious adverse event related to treatment | 2 (11.8) | |
| Most common TEAEs, no. of events (% of patients) | All grades (≥10%) | Grade ≥3 |
| Insomnia | 7 (41.2) | 0 (0) |
| Hyperglycemia | 5 (29.4) | 0 (0) |
| Pain | 5 (29.4) | 2 (11.8) |
| Vaso-occlusive crisis | 4 (23.5) | 4 (23.5) |
| Anemia | 3 (17.6) | 2 (11.8) |
| Arthralgia | 3 (17.6) | 0 (0) |
| AST increased | 3 (17.6) | 0 (0) |
| Creatinine phosphokinase (CPK) increased | 3 (17.6) | 0 (0) |
| Headache | 3 (17.6) | 0 (0) |
| Hypertension | 3 (17.6) | 1 (5.9) |
| Hyperuricemia | 3 (17.6) | 0 (0) |
| Hyponatremia | 3 (17.6) | 0 (0) |
| Back pain | 2 (11.8) | 0 (0) |
| Blood bicarbonate decreased | 2 (11.8) | 0 (0) |
| Blood bilirubin increased | 2 (11.8) | 0 (0) |
| Diarrhea | 2 (11.8) | 0 (0) |
| Dyspnea | 2 (11.8) | 0 (0) |
| Heart rate increased | 2 (11.8) | 0 (0) |
| Helicobacter pylori infection | 2 (11.8) | 0 (0) |
| Skin ulceration | 2 (11.8) | 0 (0) |
| Sore throat | 2 (11.8) | 0 (0) |
| Upper respiratory infection | 2 (11.8) | 0 (0) |
| Serious adverse events, no. of events (% of patients) | ||
| All | 6 (35.3) | |
| VOC† | 4 (23.5) | |
| Pain (shoulder) | 1∗ (5.9) | |
| PE | 1 (5.9) | |
∗
One episode of shoulder pain, and 1 preexisting PE.
†
VOC is defined as acute clinical events that has no evident cause other than SCD, including acute episodes of pain requiring treatment with parenteral opioids at a medical facility, acute chest syndrome, hepatic sequestration, splenic sequestration, and priapism. Two of 4 VOCs were possibly related to the study drug, both occurred during the drug taper.