Baseline demographic and disease characteristics for patients with relapsed or refractory AML and all patients in the dose-escalation and expansion study phases
| Characteristic . | Relapsed or refractory AML . | All patients (N = 239) . | ||||
|---|---|---|---|---|---|---|
| Enasidenib 100 mg per day (n = 109) . | All doses (n = 176) . | |||||
| No. . | % . | No. . | % . | No. . | % . | |
| Age, median (range), y | 67 (19-100) | 67 (19-100) | 70 (19-100) | |||
| Sex | ||||||
| Male | 46 | 42 | 90 | 51 | 137 | 57 |
| Female | 63 | 58 | 86 | 49 | 102 | 43 |
| AML classification* | 213 | 89 | ||||
| Myelodysplasia-related changes | 27 | 25 | 45 | 26 | 57 | 27 |
| Recurrent genetic abnormalities | 7 | 6 | 15 | 9 | 17 | 8 |
| Therapy-related myeloid neoplasms | 1 | <1 | 2 | 1 | 4 | 2 |
| Not otherwise specified | 62 | 57 | 91 | 52 | 111 | 52 |
| Missing† | 12 | 11 | 23 | 13 | 24 | 11 |
| Outcomes of prior AML therapyठ| ||||||
| Refractory to initial induction or re-induction treatment | 35 | 32 | 57 | 32 | — | |
| Relapsed/refractory to ≥2 cycles of first-line lower-intensity therapy§ | 25 | 23 | 43 | 24 | — | |
| Relapsed within 1 y of initial treatment | 27 | 25 | 41 | 23 | — | |
| Relapsed posttransplant | 12 | 11 | 24 | 14 | — | |
| In second or later relapse | 13 | 12 | 22 | 13 | — | |
| Relapsed >1 y after initial treatment | 8 | 7 | 15 | 9 | — | |
| Cytogenetic risk status | 80 | 73 | 128 | 73 | 175 | 73 |
| Intermediate | 51 | 64 | 85 | 66 | 117 | 67 |
| Poor | 29 | 36 | 43 | 34 | 58 | 33 |
| Co-occurring mutations | 47 | 43 | 100 | 57 | 134 | 56 |
| NPM1 | 8 | 17 | 16 | 16 | 17 | 13 |
| CEBPA | 1 | 2 | 6 | 6 | 10 | 8 |
| FLT3-ITD | 1 | 2 | 4 | 4 | 4 | 3 |
| Antecedent history of myelodysplastic syndromes | 17 | 16 | 30 | 17 | 30 | 13 |
| Prior stem cell transplantation | 12 | 11 | 24 | 14 | 28 | 12 |
| ECOG performance status|| | ||||||
| 0 | 25 | 23 | 39 | 22 | 55 | 23 |
| 1 | 68 | 62 | 106 | 60 | 139 | 58 |
| 2 | 16 | 15 | 31 | 18 | 45 | 19 |
| IDH2 mutation location | ||||||
| R140 | 83 | 76 | 130 | 74 | 179 | 75 |
| R172 | 25 | 23 | 45 | 26 | 57 | 24 |
| Other¶ | 1 | <1 | 1 | <1 | 3 | 1 |
| Bone marrow blasts, median (range), %# | 49 (0-96) | 49 (0-98) | 41 (0**-98) | |||
| Hematology, median (range) | ||||||
| WBC, ×109/L | 3.0 (0.2-88) | 2.7 (0.2-88) | 2.6 (0.2-88) | |||
| Hemoglobin, g/dL | 9.3 (6.9-13.8) | 9.2 (6.9-13.8) | 9.1 (6.9-15.2) | |||
| Platelets, ×109/L | 39 (1-372) | 44 (1-507) | 45 (1-644) | |||
| Characteristic . | Relapsed or refractory AML . | All patients (N = 239) . | ||||
|---|---|---|---|---|---|---|
| Enasidenib 100 mg per day (n = 109) . | All doses (n = 176) . | |||||
| No. . | % . | No. . | % . | No. . | % . | |
| Age, median (range), y | 67 (19-100) | 67 (19-100) | 70 (19-100) | |||
| Sex | ||||||
| Male | 46 | 42 | 90 | 51 | 137 | 57 |
| Female | 63 | 58 | 86 | 49 | 102 | 43 |
| AML classification* | 213 | 89 | ||||
| Myelodysplasia-related changes | 27 | 25 | 45 | 26 | 57 | 27 |
| Recurrent genetic abnormalities | 7 | 6 | 15 | 9 | 17 | 8 |
| Therapy-related myeloid neoplasms | 1 | <1 | 2 | 1 | 4 | 2 |
| Not otherwise specified | 62 | 57 | 91 | 52 | 111 | 52 |
| Missing† | 12 | 11 | 23 | 13 | 24 | 11 |
| Outcomes of prior AML therapyठ| ||||||
| Refractory to initial induction or re-induction treatment | 35 | 32 | 57 | 32 | — | |
| Relapsed/refractory to ≥2 cycles of first-line lower-intensity therapy§ | 25 | 23 | 43 | 24 | — | |
| Relapsed within 1 y of initial treatment | 27 | 25 | 41 | 23 | — | |
| Relapsed posttransplant | 12 | 11 | 24 | 14 | — | |
| In second or later relapse | 13 | 12 | 22 | 13 | — | |
| Relapsed >1 y after initial treatment | 8 | 7 | 15 | 9 | — | |
| Cytogenetic risk status | 80 | 73 | 128 | 73 | 175 | 73 |
| Intermediate | 51 | 64 | 85 | 66 | 117 | 67 |
| Poor | 29 | 36 | 43 | 34 | 58 | 33 |
| Co-occurring mutations | 47 | 43 | 100 | 57 | 134 | 56 |
| NPM1 | 8 | 17 | 16 | 16 | 17 | 13 |
| CEBPA | 1 | 2 | 6 | 6 | 10 | 8 |
| FLT3-ITD | 1 | 2 | 4 | 4 | 4 | 3 |
| Antecedent history of myelodysplastic syndromes | 17 | 16 | 30 | 17 | 30 | 13 |
| Prior stem cell transplantation | 12 | 11 | 24 | 14 | 28 | 12 |
| ECOG performance status|| | ||||||
| 0 | 25 | 23 | 39 | 22 | 55 | 23 |
| 1 | 68 | 62 | 106 | 60 | 139 | 58 |
| 2 | 16 | 15 | 31 | 18 | 45 | 19 |
| IDH2 mutation location | ||||||
| R140 | 83 | 76 | 130 | 74 | 179 | 75 |
| R172 | 25 | 23 | 45 | 26 | 57 | 24 |
| Other¶ | 1 | <1 | 1 | <1 | 3 | 1 |
| Bone marrow blasts, median (range), %# | 49 (0-96) | 49 (0-98) | 41 (0**-98) | |||
| Hematology, median (range) | ||||||
| WBC, ×109/L | 3.0 (0.2-88) | 2.7 (0.2-88) | 2.6 (0.2-88) | |||
| Hemoglobin, g/dL | 9.3 (6.9-13.8) | 9.2 (6.9-13.8) | 9.1 (6.9-15.2) | |||
| Platelets, ×109/L | 39 (1-372) | 44 (1-507) | 45 (1-644) | |||
ECOG, Eastern Cooperative Oncology Group; WBC, white blood cell count.
Per World Health Organization (WHO) 2008 AML classifications of myeloid neoplasms.21
During the study, the protocol was amended to determine AML subtypes according to the WHO classification scheme (AML classification was previously determined by French-American-British criteria).
Prior (initial) AML treatment of relapsed/refractory patients.
Individual patients may be counted in more than 1 category.
ECOG performance status scores of 0, 1, or 2 (on a scale from 0 to 5, with 0 indicating that the patient is fully active and higher numbers indicating greater disability).
One patient had an IDH2 G145fs mutation, and mutation site was not reported for 2 patients.
Local assessment.
Nine patients had extramedullary disease only at relapse.