Investigator-reported hematologic response, time to response, and response duration among patients with relapsed/refractory AML treated with enasidenib
| Response . | Relapsed or refractory AML . | |||||||||
|---|---|---|---|---|---|---|---|---|---|---|
| Enasidenib 100 mg per day (n = 109) . | All doses (N = 176) . | |||||||||
| No. . | % . | 95% CI . | Median . | Range . | No. . | % . | 95% CI . | Median . | Range . | |
| ORR*† | 42 | 38.5 | 29.4-48.3 | 71 | 40.3 | 33.0-48.0 | ||||
| Best response | ||||||||||
| CR | 22 | 20.2 | 13.1-28.9 | 34 | 19.3 | 13.8-25.9 | ||||
| CR with incomplete hematologic recovery/CR with incomplete platelet recovery | 7 | 6.4 | 12 | 6.8 | ||||||
| Partial remission | 3 | 2.8 | 11 | 6.3 | ||||||
| Morphologic leukemia-free state | 10 | 9.2 | 14 | 8.0 | ||||||
| Stable disease‡ | 58 | 53.2 | 85 | 48.3 | ||||||
| Progressive disease§ | 5 | 4.6 | 9 | 5.1 | ||||||
| Not evaluable | 2 | 1.8 | 3 | 1. 7 | ||||||
| Time to first response, mo | 1.9 | 0.5-9.4 | 1.9 | 0.5-9.4 | ||||||
| Duration of response, mo | 3.8-9.7 | 5.6 | 3.9-7.4 | 5.8 | ||||||
| Time to CR, mo | 3.7 | 0.7-11.2 | 3.8 | 0.5-11.2 | ||||||
| Duration of response in patients who attained CR, mo || | 5.3-NR | 8.8 | 6.4-NR | 8.8 | ||||||
| Response . | Relapsed or refractory AML . | |||||||||
|---|---|---|---|---|---|---|---|---|---|---|
| Enasidenib 100 mg per day (n = 109) . | All doses (N = 176) . | |||||||||
| No. . | % . | 95% CI . | Median . | Range . | No. . | % . | 95% CI . | Median . | Range . | |
| ORR*† | 42 | 38.5 | 29.4-48.3 | 71 | 40.3 | 33.0-48.0 | ||||
| Best response | ||||||||||
| CR | 22 | 20.2 | 13.1-28.9 | 34 | 19.3 | 13.8-25.9 | ||||
| CR with incomplete hematologic recovery/CR with incomplete platelet recovery | 7 | 6.4 | 12 | 6.8 | ||||||
| Partial remission | 3 | 2.8 | 11 | 6.3 | ||||||
| Morphologic leukemia-free state | 10 | 9.2 | 14 | 8.0 | ||||||
| Stable disease‡ | 58 | 53.2 | 85 | 48.3 | ||||||
| Progressive disease§ | 5 | 4.6 | 9 | 5.1 | ||||||
| Not evaluable | 2 | 1.8 | 3 | 1. 7 | ||||||
| Time to first response, mo | 1.9 | 0.5-9.4 | 1.9 | 0.5-9.4 | ||||||
| Duration of response, mo | 3.8-9.7 | 5.6 | 3.9-7.4 | 5.8 | ||||||
| Time to CR, mo | 3.7 | 0.7-11.2 | 3.8 | 0.5-11.2 | ||||||
| Duration of response in patients who attained CR, mo || | 5.3-NR | 8.8 | 6.4-NR | 8.8 | ||||||
Responses were evaluated by study investigators and classified according to the 2003 revised International Working Group criteria for AML22
CR, complete remission; NR, not reached.
ORR includes patients with CR, CR with incomplete hematologic recovery, CR with incomplete platelet recovery, partial remission, or morphologic leukemia-free state.
Of 9 patients with extramedullary disease at study entry, 1 patient achieved CR with incomplete platelet recovery, 5 patients maintained stable disease, 1 patient experienced disease progression, and 2 patients were not evaluable for response.
Stable disease was defined as failure to achieve a response but not meeting criteria for disease progression for a period of more than 8 weeks.
Patients must have had previous partial remission or stable disease. For patients with 5% to 66% bone marrow blasts at nadir, progressive disease was defined as a >50% increase in bone marrow blast count percentage from the nadir and a blast percentage of ≥20%; for patients with ≥67% bone marrow blasts at nadir, progressive disease was defined as a doubling of the nadir absolute peripheral blood blast count with a final absolute peripheral blood blast count of >10 × 109/L.
Date of first documented response to date of relapse, disease progression, or death.