Summary of AEs based on treatment-emergent AE incidence of 15% or more and treatment-emergent SAE incidence of 5% or more
| All terms, n (%) . | Safety population (N = 84) . | |||
|---|---|---|---|---|
| Treatment-emergent AE . | Treatment-related AE . | |||
| Any grade . | Grade ≥3 . | Any grade . | Grade ≥3 . | |
| AE | 83 (98.8) | 77 (91.7) | 66 (78.6) | 50 (59.5) |
| AE (treatment emergent), incidence ≥15% | ||||
| QTcF prolongation | 36 (42.9) | 19 (22.6) | 34 (40.5) | 18 (21.4) |
| Vomiting | 31 (36.9) | 3 (3.6) | 17 (20.2) | 1 (1.2) |
| Febrile neutropenia | 29 (34.5) | 28 (33.3) | 12 (14.3) | 11 (13.1) |
| Hypokalemia | 27 (32.1) | 8 (9.5) | 7 (8.3) | 2 (2.4) |
| Nausea | 24 (28.6) | 5 (6.0) | 14 (16.7) | 2 (2.4) |
| Anemia | 23 (27.4) | 21 (25.0) | 13 (15.5) | 12 (14.3) |
| Diarrhea | 23 (27.4) | 5 (6.0) | 6 (7.1) | 1 (1.2) |
| Fatigue | 20 (23.8) | 4 (4.8) | 8 (9.5) | 1 (1.2) |
| Pyrexia | 20 (23.8) | 1 (1.2) | 3 (3.6) | 0 |
| Epistaxis | 18 (21.4) | 4 (4.8) | 3 (3.6) | 2 (2.4) |
| Peripheral edema | 18 (21.4) | 0 | 4 (4.8) | 0 |
| Differentiation syndrome | 16 (19.0) | 11 (13.1) | 15 (17.9) | 11 (13.1) |
| Dyspnea | 16 (19.0) | 3 (3.6) | 4 (4.8) | 1 (1.2) |
| Pneumonia | 16 (19.0) | 12 (14.3) | 3 (3.6) | 1 (1.2) |
| Dysgeusia | 14 (16.7) | 0 | 10 (11.9) | 0 |
| Platelet count decreased | 14 (16.7) | 14 (16.7) | 9 (10.7) | 9 (10.7) |
| Thrombocytopenia | 14 (16.7) | 12 (14.3) | 8 (9.5) | 8 (9.5) |
| Abdominal pain | 13 (15.5) | 2 (2.4) | 3 (3.6) | 0 |
| Arthralgia | 13 (15.5) | 1 (1.2) | 1 (1.2) | 0 |
| Constipation | 13 (15.5) | 0 | 1 (1.2) | 0 |
| Decreased appetite | 13 (15.5) | 1 (1.2) | 5 (6.0) | 1 (1.2) |
| Sepsis | 13 (15.5) | 13 (15.5) | 0 | 0 |
| SAE | 64 (76.2) | — | 31 (36.9) | — |
| SAE (treatment emergent), incidence ≥5% | ||||
| Febrile neutropenia | 18 (21.4) | — | 7 (8.3) | — |
| Differentiation syndrome | 11 (13.1) | — | 11 (13.1) | — |
| Sepsis | 11 (13.1) | — | 0 | — |
| Pneumonia | 7 (8.3) | — | 1 (1.2) | — |
| Anemia | 6 (7.1) | — | 3 (3.6) | — |
| QTcF prolongation | 6 (7.1) | — | 6 (7.1) | — |
| AE leading to dose reduction | 10 (11.9) | — | 10 (11.9) | — |
| AE leading to dose interruption∗ | 56 (66.7) | — | 42 (50.0) | — |
| AE leading to treatment discontinuation | 25 (29.8)† | — | 4 (4.8) | — |
| AE leading to death | 21 (25.0) | — | 1 (1.2)‡ | — |
| All terms, n (%) . | Safety population (N = 84) . | |||
|---|---|---|---|---|
| Treatment-emergent AE . | Treatment-related AE . | |||
| Any grade . | Grade ≥3 . | Any grade . | Grade ≥3 . | |
| AE | 83 (98.8) | 77 (91.7) | 66 (78.6) | 50 (59.5) |
| AE (treatment emergent), incidence ≥15% | ||||
| QTcF prolongation | 36 (42.9) | 19 (22.6) | 34 (40.5) | 18 (21.4) |
| Vomiting | 31 (36.9) | 3 (3.6) | 17 (20.2) | 1 (1.2) |
| Febrile neutropenia | 29 (34.5) | 28 (33.3) | 12 (14.3) | 11 (13.1) |
| Hypokalemia | 27 (32.1) | 8 (9.5) | 7 (8.3) | 2 (2.4) |
| Nausea | 24 (28.6) | 5 (6.0) | 14 (16.7) | 2 (2.4) |
| Anemia | 23 (27.4) | 21 (25.0) | 13 (15.5) | 12 (14.3) |
| Diarrhea | 23 (27.4) | 5 (6.0) | 6 (7.1) | 1 (1.2) |
| Fatigue | 20 (23.8) | 4 (4.8) | 8 (9.5) | 1 (1.2) |
| Pyrexia | 20 (23.8) | 1 (1.2) | 3 (3.6) | 0 |
| Epistaxis | 18 (21.4) | 4 (4.8) | 3 (3.6) | 2 (2.4) |
| Peripheral edema | 18 (21.4) | 0 | 4 (4.8) | 0 |
| Differentiation syndrome | 16 (19.0) | 11 (13.1) | 15 (17.9) | 11 (13.1) |
| Dyspnea | 16 (19.0) | 3 (3.6) | 4 (4.8) | 1 (1.2) |
| Pneumonia | 16 (19.0) | 12 (14.3) | 3 (3.6) | 1 (1.2) |
| Dysgeusia | 14 (16.7) | 0 | 10 (11.9) | 0 |
| Platelet count decreased | 14 (16.7) | 14 (16.7) | 9 (10.7) | 9 (10.7) |
| Thrombocytopenia | 14 (16.7) | 12 (14.3) | 8 (9.5) | 8 (9.5) |
| Abdominal pain | 13 (15.5) | 2 (2.4) | 3 (3.6) | 0 |
| Arthralgia | 13 (15.5) | 1 (1.2) | 1 (1.2) | 0 |
| Constipation | 13 (15.5) | 0 | 1 (1.2) | 0 |
| Decreased appetite | 13 (15.5) | 1 (1.2) | 5 (6.0) | 1 (1.2) |
| Sepsis | 13 (15.5) | 13 (15.5) | 0 | 0 |
| SAE | 64 (76.2) | — | 31 (36.9) | — |
| SAE (treatment emergent), incidence ≥5% | ||||
| Febrile neutropenia | 18 (21.4) | — | 7 (8.3) | — |
| Differentiation syndrome | 11 (13.1) | — | 11 (13.1) | — |
| Sepsis | 11 (13.1) | — | 0 | — |
| Pneumonia | 7 (8.3) | — | 1 (1.2) | — |
| Anemia | 6 (7.1) | — | 3 (3.6) | — |
| QTcF prolongation | 6 (7.1) | — | 6 (7.1) | — |
| AE leading to dose reduction | 10 (11.9) | — | 10 (11.9) | — |
| AE leading to dose interruption∗ | 56 (66.7) | — | 42 (50.0) | — |
| AE leading to treatment discontinuation | 25 (29.8)† | — | 4 (4.8) | — |
| AE leading to death | 21 (25.0) | — | 1 (1.2)‡ | — |
SAE, serious AE.
This includes patients who had interruptions and restarted treatment on the same day as directed by protocol for electrolyte management and/or QTcF prolongation.
Patients may have experienced >1 treatment-emergent AE leading to discontinuation. Of these 25 patients in the safety population who discontinued treatment due to an AE, 21 were in the efficacy-evaluable population (Figure 1).
The treatment-related AE leading to death was classified as cardiac arrest; however, the investigator reported 2 possible causes of death: intracranial hemorrhage (patient had profound thrombocytopenia since baseline [platelet count, 6 × 109 to 13 × 109/L]) or arrhythmia; an autopsy was not performed.