Demographic and baseline disease characteristics (safety analysis cohort)
| . | Part 1 . | Patients treated at RP2D from part 1 and part 2 . | Overall (N = 25) . | |
|---|---|---|---|---|
| Defibrotide 2.5 mg/kg (n = 4) . | Defibrotide 6.25 mg/kg (n = 6) . | Defibrotide 6.25 mg/kg (n = 21) . | ||
| Age, y | ||||
| Median | 63.5 | 72.0 | 69.0 | 68.0 |
| Min, max | 31, 73 | 52, 76 | 43, 78 | 31, 78 |
| Age group, y, n (%) | ||||
| 18-64 | 2 (50) | 2 (33) | 7 (33) | 9 (36) |
| ≥65 | 2 (50) | 4 (67) | 14 (67) | 16 (64) |
| Sex, n (%) | ||||
| Female | 1 (25) | 2 (33) | 7 (33) | 8 (32) |
| Male | 3 (75) | 4 (67) | 14 (67) | 17 (68) |
| White, non-Hispanic, n (%) | 4 (100) | 6 (100) | 21 (100) | 25 (100) |
| ECOG PS at baseline, n (%) | ||||
| 0 | 2 (50) | 0 | 5 (24) | 7 (28) |
| 1 | 2 (50) | 6 (100) | 16 (76) | 18 (72) |
| Number of previous treatment regimens | ||||
| Median | 2.0 | 3.5 | 3.0 | 2.0 |
| Min, max | 2, 2 | 2, 6 | 1, 6 | 1, 6 |
| Primary refractory disease, n (%) | ||||
| Yes | 3 (75) | 2 (33) | 11 (52) | 14 (56) |
| No | 1 (25) | 4 (67) | 10 (48) | 11 (44) |
| Refractory to previous treatment, n (%) | ||||
| Yes | 4 (100) | 6 (100) | 17 (81) | 21 (84) |
| No | 0 | 0 | 4 (19) | 4 (16) |
| Previous autologous stem cell transplantation, n (%) | ||||
| Yes | 0 | 2 (33) | 5 (24) | 5 (20) |
| No | 4 (100) | 4 (67) | 16 (76) | 20 (80) |
| Number of recurrences | ||||
| Median | 1.0 | 2.5 | 2.0 | 1.0 |
| Min, max | 1, 1 | 1, 5 | 0, 5 | 0, 5 |
| Defibrotide exposure, d | ||||
| Median | 11.0 | 11.5 | 11.0 | – |
| Min, max | 1, 12 | 11, 12 | 3, 12 | – |
| Total doses received | ||||
| Median | 34.0 | 35.0 | 35.0 | – |
| Min, max | 1, 35 | 35, 35 | 3, 35 | – |
| Daily dose, mg/kg per day | ||||
| Median | 7.320 | 19.190 | 19.760 | – |
| Min, max | 2.46, 7.89 | 18.26, 20.07 | 6.25, 20.37 | – |
| . | Part 1 . | Patients treated at RP2D from part 1 and part 2 . | Overall (N = 25) . | |
|---|---|---|---|---|
| Defibrotide 2.5 mg/kg (n = 4) . | Defibrotide 6.25 mg/kg (n = 6) . | Defibrotide 6.25 mg/kg (n = 21) . | ||
| Age, y | ||||
| Median | 63.5 | 72.0 | 69.0 | 68.0 |
| Min, max | 31, 73 | 52, 76 | 43, 78 | 31, 78 |
| Age group, y, n (%) | ||||
| 18-64 | 2 (50) | 2 (33) | 7 (33) | 9 (36) |
| ≥65 | 2 (50) | 4 (67) | 14 (67) | 16 (64) |
| Sex, n (%) | ||||
| Female | 1 (25) | 2 (33) | 7 (33) | 8 (32) |
| Male | 3 (75) | 4 (67) | 14 (67) | 17 (68) |
| White, non-Hispanic, n (%) | 4 (100) | 6 (100) | 21 (100) | 25 (100) |
| ECOG PS at baseline, n (%) | ||||
| 0 | 2 (50) | 0 | 5 (24) | 7 (28) |
| 1 | 2 (50) | 6 (100) | 16 (76) | 18 (72) |
| Number of previous treatment regimens | ||||
| Median | 2.0 | 3.5 | 3.0 | 2.0 |
| Min, max | 2, 2 | 2, 6 | 1, 6 | 1, 6 |
| Primary refractory disease, n (%) | ||||
| Yes | 3 (75) | 2 (33) | 11 (52) | 14 (56) |
| No | 1 (25) | 4 (67) | 10 (48) | 11 (44) |
| Refractory to previous treatment, n (%) | ||||
| Yes | 4 (100) | 6 (100) | 17 (81) | 21 (84) |
| No | 0 | 0 | 4 (19) | 4 (16) |
| Previous autologous stem cell transplantation, n (%) | ||||
| Yes | 0 | 2 (33) | 5 (24) | 5 (20) |
| No | 4 (100) | 4 (67) | 16 (76) | 20 (80) |
| Number of recurrences | ||||
| Median | 1.0 | 2.5 | 2.0 | 1.0 |
| Min, max | 1, 1 | 1, 5 | 0, 5 | 0, 5 |
| Defibrotide exposure, d | ||||
| Median | 11.0 | 11.5 | 11.0 | – |
| Min, max | 1, 12 | 11, 12 | 3, 12 | – |
| Total doses received | ||||
| Median | 34.0 | 35.0 | 35.0 | – |
| Min, max | 1, 35 | 35, 35 | 3, 35 | – |
| Daily dose, mg/kg per day | ||||
| Median | 7.320 | 19.190 | 19.760 | – |
| Min, max | 2.46, 7.89 | 18.26, 20.07 | 6.25, 20.37 | – |
ECOG PS, Eastern Cooperative Oncology Group performance status; Max, maximum; Min, minimum.