Table 3.
Response rates and duration
| FAS | N = 22 |
N = 22 |
Pooled (N = 22) | ||
|---|---|---|---|---|---|
| Monotherapy OLU (n = 6)∗ | Combination OLU+AZA (n = 16) | TN (n = 7)∗ | R/R (n = 15)∗ | ||
| Best overall response, n (%) | |||||
| ORR (CR + marrow CR + PR) | 2 (33) | 11 (69) | 6 (86) | 7 (47) | 13 (59) |
| CR | 1 (17) | 5 (31) | 5 (71) | 1 (7) | 6 (27) |
| Marrow CR | 1 (17) | 6 (38) | 1 (14) | 6 (40) | 7 (32) |
| Marrow CR with HI | 1 (17) | 3 (19) | 1 (14) | 3 (20) | 4 (18) |
| PR | 0 | 0 | 0 | 0 | 0 |
| SD | 2 (33) | 3 (19) | 1 (14) | 4 (27) | 5 (23) |
| SD with HI | 1 (17) | 1 (6) | 1 (14) | 1 (7) | 2 (9) |
| PD | 1 (17) | 0 | 0 | 1 (7) | 1 (5) |
| Not done | 1 (17) | 2 (13) | 0 | 3 (20) | 3 (14) |
| Composite response rate (CR + marrow CR + SD HI) | 3 (50) | 12 (75) | 7 (100) | 8 (53) | 15 (68) |
| DOR, median (95% CI), mo | |||||
| Duration of CR | NR (NR to NR)† | 14.2 (4.9 to NR) | 14.5 (4.9 to NR) | 20.5‡ | 20.5 (4.9 to NR) |
| Duration of CR + marrow CR | NR (6.7 to NR) | 14.6 (2.8 to NR) | 23 (5.8 to NR) | 14.6 (2.8 to NR) | 14.6 (5.8-32.8) |
| Time to response, median (range), mo | |||||
| Time to CR | 8.3 (8.3-8.3) | 5.1 (2.5-14.3) | 5.1 (2.5-8.3) | 14.3‡ | 5.7 (2.5-14.3) |
| Time to CR + marrow CR | 4.7 (1-8.3) | 2 (1-13) | 2.2 (1-8.3) | 2 (1-13) | 2 (1-13) |
| OS, median (95% CI), mo | |||||
| OS, total | 14 (4.5 to NR) | 27.5 (5-36.6) | NR (7.3 to NR) | 16.3 (3.1-36.6) | 27.2 (6.9-37) |
| CR responders | NR (NR to NR)† | 26.9 (7.3 to NR) | 26.9 (7.3 to NR) | 37‡ | 37 (7.3 to NR) |
| CR + marrow CR | NR (14 to NR) | 36.6 (7.3 to NR) | NR (7.3 to NR) | 36.6 (5 to NR) | 36.6 (14 to NR) |
| Nonresponders | NR (4.5 to NR) | 3.1 (1 to NR) | NR (NR to NR)§ | 5.7 (1 to NR) | 6.9 (1-30.8) |
| EFS, median (95% CI), mo | |||||
| EFS, total | 4.9 (1.9 to NR) | 10.2 (3.7-25.8) | 25.8 (7.3 to NR) | 20.3 (3.7 to NR) | 7.6 (3.7-25.8) |
| CR responders | NR (NR to NR)† | 19.9 (7.3 to NR) | 19.9 (7.3 to NR) | 34.8‡ | 25.8 (7.3 to NR) |
| CR + marrow CR | NR (7.6 to NR) | 20.3 (7.3 to NR) | 25.8 (7.3 to NR) | 20.3 (3.7 to NR) | 20.3 (7.3-34.8) |
| Nonresponders | 4.3 (1.9 to NR) | 1.9 (1 to NR) | NR (NR to NR)§ | 2.8 (1 to 4.9) | 3.7 (1-4.9) |
AZA added to the regimen and achieved a CR.
∗
Three patients from the phase 1 dose-escalation portion of the study (all received monotherapy OLU) received a suboptimal dose that was ≤50% of the approved dose of OLU. One patient (R/R) received 100 mg daily, and 2 patients (1 TN and 1 R/R) received 150 mg daily.
†
There were no patients with an event, and 1 patient was censored at study end but continued to receive study drug/transitioned to commercial use.
‡
Range and 95% CI not applicable (n = 1)
§
The patient was categorized as SD while receiving suboptimal OLU monotherapy (150 mg, daily) but at investigator’s decision had AZA added to the regimen and achieved a CR.