Table 1. Treatment-emergent adverse events (AE).
| AE, n (%) | Schedule Aa (n=27) | Schedule B (n=26) | Schedule C (n=2) | Schedule D (n=1) | Schedule E (n=16) | Total (n=72) |
|---|---|---|---|---|---|---|
| Any AEb | 27 (100) | 26 (100) | 2 (100) | 1 (100) | 16 (100) | 72 (100) |
| Pyrexia | 12 (44) | 16 (62) | 0 | 0 | 7 (44) | 35 (49) |
| Diarrhea | 14 (52) | 9 (35) | 2 (100) | 0 | 6 (38) | 31 (43) |
| Anorexia | 11 (41) | 7 (27) | 1 (50) | 0 | 6 (38) | 25 (35) |
| Peripheral edema | 11 (41) | 6 (23) | 1 (50) | 0 | 6 (38) | 24 (33) |
| Fatigue | 9 (33) | 9 (35) | 0 | 0 | 5 (31) | 23 (32) |
| Dyspnea | 6 (22) | 10 (38) | 0 | 1 (100) | 6 (38) | 23 (32) |
| FN | 11 (41) | 8 (31) | 0 | 0 | 3 (19) | 22 (31) |
| Nausea | 10 (37) | 7 (27) | 0 | 0 | 5 (31) | 22 (31) |
| Chills | 8 (30) | 11 (42) | 0 | 0 | 1 (6) | 20 (28) |
| Dizziness | 11 (41) | 4 (15) | 1 (50) | 0 | 4 (25) | 20 (28) |
| Myalgia | 8 (30) | 7 (27) | 1 (50) | 0 | 4 (25) | 20 (28) |
| Increased AST | 9 (33) | 3 (12) | 1 (50) | 0 | 5 (31) | 18 (25) |
| Increased ALT | 8 (30) | 4 (15) | 1 (50) | 0 | 4 (25) | 17 (24) |
| Headache | 5 (19) | 7 (27) | 1 (50) | 0 | 4 (25) | 17 (24) |
| Epistaxis | 6 (22) | 6 (23) | 0 | 0 | 5 (31) | 17 (24) |
| Vomiting | 8 (30) | 5 (19) | 1 (50) | 0 | 2 (13) | 16 (22) |
| Cough | 7 (26) | 6 (23) | 0 | 0 | 2 (13) | 15 (21) |
| Pneumonia | 5 (19) | 4 (15) | 2 (100) | 0 | 4 (25) | 15 (21) |
| Any treatment-related grade ⩾3 AEc | 16 (59) | 20 (77) | 2 (100) | 1 (100) | 12 (75) | 51 (71) |
| Low platelets | 2 (7) | 2 (8) | 0 | 0 | 0 | 4 (6) |
| MI | 0 | 1 (4) | 0 | 1 (100) | 0 | 2 (3) |
| FNd | 0 | 2 (8) | 0 | 0 | 0 | 2 (3) |
| Increased AST | 2 (7) | 0 | 0 | 0 | 0 | 2 (3) |
| Increased ALT | 1 (4) | 0 | 0 | 0 | 1 (6) | 2 (3) |
| Increased bilirubin | 0 | 1 (4) | 0 | 0 | 1 (6) | 2 (3) |
| Hypoxia | 1 (4) | 1 (4) | 0 | 0 | 0 | 2 (3) |
| Hypotension | 1 (4) | 1 (4) | 0 | 0 | 0 | 2 (3) |
| MOF | 0 | 2 (8) | 0 | 0 | 0 | 2 (3) |
| Fatigue | 2 (7) | 0 | 0 | 0 | 0 | 2 (3) |
Abbreviations: ALT, alanine transaminase; AST, aspartate aminotransferase; FN, febrile neutropenial; MI, myocardial infarction; MOF, multiorgan failure.
a
Schdeule A (dose escalation cohort, Days 1, 3, 5); Schedule B (dose escalation cohort, Days 1, 4, 8, 11); Schedule C (weekly dosing schedule); Schedule D (combination with azacitidine); Schedule E (dose expansion cohort, Days 1, 3, 5).
b
Any AE occurring in >20% of patients overall.
c
Any treatment-related grade ⩾3 AE occurring in >1 patient overall.
d
Bacteremia occurred in a total of 3 patients on study (1 on Schedule A and 2 on Schedule E) (4%) and septic shock in 1 patient on Schedule C (1%); these events were reported as not related to pevonedistat.