Patient characteristics, responses, and results of correlative studies
Dose, mg . | Patient no./FLT3 mutation . | Age, y (sex) . | FAB . | AML status . | ECOG performance status . | Salvage regimen . | In vitro cytotoxic response . | In vivo inhibition FLT3 <15% . | Best plasma CEP-701 at trough, μM . | Peripheral blood blast or marrow*blast responses . | Peripheral blood responses, other . |
|---|---|---|---|---|---|---|---|---|---|---|---|
| 40 | 101/ITD | 67 (F) | M1 | Relapse | 0 | 1 | - | + | 10 | — | — |
| 40 | 102/D835 | 23 (F) | M2 | Relapse | 1 | 2 | + | - | NA | — | — |
| 40 | 103/ITD | 66 (M) | M1 | Refractory | 1 | 2 | + | - | 2.4 | — | — |
| 60 | 104/ITD | 71 (M) | M5 | Refractory | 1 | 1 | + | + | 4.9 | *25% → < 5% | ANC > 1000 transfusion independent |
| 60† | 105/ITD | 74 (F) | M5 | Refractory | 0 | 4 | + | + | 3.4 | 32% → < 5% | ANC > 1000 ↓ transfusions |
| 60 | 106/ITD | 60 (F) | M2 | Relapse | 0 | 4 | - | + | NA | — | — |
| 60† | 107/ITD | 69 (F) | M1 | Refractory | 1 | 1 | + | + | 12.8 | 94% → < 1% | — |
| 60 | 108/ITD | 62 (M) | M5 | Refractory | 1 | 2 | + | - | 6.5 | — | — |
| 60 | 201/ITD | 46 (M) | M1 | Refractory | 1 | 1 | NA | - | 1.2 | — | — |
| 60† | 202/ITD | 61 (M) | M5 | Refractory | 1 | 3 | NA | + | 7.7 | 74% → 1% | ANC > 1000 ↓ transfusions |
| 60 | 203/ITD | 18 (F) | M6 | Relapse | 0 | 3 | NA | - | 2.1 | — | — |
| 60 | 204/ITD | 59 (M) | M4 | Refractory | 1 | 1 | NA | + | 3.3 | 27% → 1% | — |
| 60 | 205/ITD | 62 (F) | M2 | Relapse | 1 | 1 | NA | + | 3.0 | — | — |
| 60 | 206/ITD | 71 (M) | Refractory | 2 | 1 | NA | - | 2.8 | — | — | |
| 60 | 207/ITD | 47 (M) | M1 | Relapse | 1 | 1 | NA | NA | NA | — | — |
| 60 | 208/ITD | 70 (F) | M2 | Refractory | 1 | 0 | NA | NA | NA | — | — |
| 60 | 209/ITD | 30 (M) | M1 | Relapse | 1 | 2 | NA | NA | NA | — | — |
Dose, mg . | Patient no./FLT3 mutation . | Age, y (sex) . | FAB . | AML status . | ECOG performance status . | Salvage regimen . | In vitro cytotoxic response . | In vivo inhibition FLT3 <15% . | Best plasma CEP-701 at trough, μM . | Peripheral blood blast or marrow*blast responses . | Peripheral blood responses, other . |
|---|---|---|---|---|---|---|---|---|---|---|---|
| 40 | 101/ITD | 67 (F) | M1 | Relapse | 0 | 1 | - | + | 10 | — | — |
| 40 | 102/D835 | 23 (F) | M2 | Relapse | 1 | 2 | + | - | NA | — | — |
| 40 | 103/ITD | 66 (M) | M1 | Refractory | 1 | 2 | + | - | 2.4 | — | — |
| 60 | 104/ITD | 71 (M) | M5 | Refractory | 1 | 1 | + | + | 4.9 | *25% → < 5% | ANC > 1000 transfusion independent |
| 60† | 105/ITD | 74 (F) | M5 | Refractory | 0 | 4 | + | + | 3.4 | 32% → < 5% | ANC > 1000 ↓ transfusions |
| 60 | 106/ITD | 60 (F) | M2 | Relapse | 0 | 4 | - | + | NA | — | — |
| 60† | 107/ITD | 69 (F) | M1 | Refractory | 1 | 1 | + | + | 12.8 | 94% → < 1% | — |
| 60 | 108/ITD | 62 (M) | M5 | Refractory | 1 | 2 | + | - | 6.5 | — | — |
| 60 | 201/ITD | 46 (M) | M1 | Refractory | 1 | 1 | NA | - | 1.2 | — | — |
| 60† | 202/ITD | 61 (M) | M5 | Refractory | 1 | 3 | NA | + | 7.7 | 74% → 1% | ANC > 1000 ↓ transfusions |
| 60 | 203/ITD | 18 (F) | M6 | Relapse | 0 | 3 | NA | - | 2.1 | — | — |
| 60 | 204/ITD | 59 (M) | M4 | Refractory | 1 | 1 | NA | + | 3.3 | 27% → 1% | — |
| 60 | 205/ITD | 62 (F) | M2 | Relapse | 1 | 1 | NA | + | 3.0 | — | — |
| 60 | 206/ITD | 71 (M) | Refractory | 2 | 1 | NA | - | 2.8 | — | — | |
| 60 | 207/ITD | 47 (M) | M1 | Relapse | 1 | 1 | NA | NA | NA | — | — |
| 60 | 208/ITD | 70 (F) | M2 | Refractory | 1 | 0 | NA | NA | NA | — | — |
| 60 | 209/ITD | 30 (M) | M1 | Relapse | 1 | 2 | NA | NA | NA | — | — |