Table 2.
Objective response rates by IRC assessment, time to response, and DOR in patients with higher-risk MDS, CMML, or AML with 20-30% blasts
| Pevonedistat + azacitidine | Azacitidine alone | |
|---|---|---|
| Higher-risk MDS | n = 161 | n = 163 |
| Objective response (CR+PR) | 39 (24) | 52 (32) |
| Objective response 2 (CR+PR+HI) | 69 (43) | 70 (43) |
| CR | 39 (24) | 52 (32) |
| mCR | 37 (23) | 32 (20) |
| PR | 0 | 0 |
| HI | 30 (19) | 18 (11) |
| Median time to first CR/PR/HI, months (range) | 2.89 (1.0-20.3) | 2.92 (1.0-13.5) |
| Median duration of ORR, months (95% CI) | 17.1 (10.71-NE) | 14.1 (7.16-29.08) |
| Median duration of ORR2, months (95% CI) | 18.9 (14.98-NE) | 18.3 (11.17-30.92) |
| Median duration of CR, months (95% CI) | 17.1 (10.71-NE) | 14.1 (6.93-29.08) |
| Patients with TP53 mutation/17p deletion/adverse-risk cytogenetics | 52 (32) | 50 (31) |
| ORR in patients with TP53 mutation/17p deletion/adverse-risk cytogenetics | 13 (25) | 14 (28) |
| Higher-risk CMML | n = 16 | n = 11 |
| Objective response (CR+PR) | 7 (44) | 4 (36) |
| Objective response 2 (CR+PR+HI) | 7 (44) | 7 (64) |
| CR | 7 (44) | 4 (36) |
| PR | 0 | 0 |
| HI | 0 | 3 (27) |
| Median time to first CR/PR/HI, months (range) | 2.63 (0.9-12.2) | 1.74 (0.9-10.4) |
| Median duration of ORR, months (95% CI) | 22.6 (3.94-NE) | NE (1.18-NE) |
| Median duration of ORR2, months (95% CI) | 29.0 (4.86-NE) | NE (1.18-NE) |
| Median duration of CR, months (95% CI) | 22.6 (3.94-NE) | NE (1.18-NE) |
| AML with 20-30% blasts | n = 50 | n = 53 |
| Objective response (CR+CRi+PR) | 18 (36) | 17 (32) |
| CR | 11 (22) | 12 (23) |
| CRi | 6 (12) | 3 (6) |
| PR | 1 (2) | 2 (4) |
| Median time to first CR/CRi/PR, months (95% CI) | 24.6 (4.80-NE) | NE (8.25-NE) |
| Median duration of CR, months (95% CI) | 15.3 (10.18-NE) | 8.0 (1.74-NE) |
| Median duration of CR/CRi, months (95% CI) | 15.0 (3.94-NE) | 8.5 (3.02-NE) |
Data are expressed as n (%), unless stated otherwise.
ORR was defined as CR+PR in higher-risk MDS/CMML and as CR+CRi+PR in AML with 20% to 30% blasts. ORR2 was defined as CR+PR+HI in higher-risk MDS/CMML and as CR+CRi+PR in patients with AML with 20% to 30% blasts.
DOR, duration of response; HI, hematologic improvement; mCR, marrow CR; NE, not estimable.