Demographic and baseline clinical characteristics of the patients
| Variables . | Enrolled patients n = 67 . | Treated patients n = 48 . |
|---|---|---|
| Age, median (range), y | 33 (18-59) | 32 (18-58) |
| Sex | ||
| Female | 33 (49.3%) | 22 (45.8%) |
| Male | 34 (50.7%) | 26 (54.2%) |
| ECOG grade, n (%) | ||
| 0 | 29 (43.3%) | 20 (41.7%) |
| 1 | 38 (56.7%) | 28 (58.3%) |
| Disease status at screening, n (%) | ||
| Relapse (first relapse with the first remission lasting <12 mo) | 12 (17.9%) | 9 (18.8%) |
| Refractory | 55 (82.1%) | 39 (81.2%) |
| Refractory subcategories | ||
| Primary refractory | 13 (19.4%) | 12 (25.0%) |
| Relapse after ≥2 CRs | 11 (16.4%) | 8 (16.7%) |
| First relapse, no remission after at least 1 salvage therapy | 20 (29.9%) | 11 (22.9%) |
| Relapsed or refractory after HSCT | 11 (16.4%) | 8 (16.7%) |
| Type of HSCT | ||
| Autologous | 4 (6.0%) | 3 (6.2%) |
| Allogeneic | 7 (10.4%) | 5 (10.4%) |
| Maximum prior lines of therapy, n (%) | ||
| Median (range) | 2.0 (1-7) | 2 (1–4) |
| 1 line | 15 (22.4%) | 13 (27.1%) |
| 2 lines | 35 (52.2%) | 25 (52.1%) |
| 3 lines | 10 (14.9%) | 7 (14.6%) |
| 4 lines | 5 (7.5%) | 3 (6.2%) |
| 5 lines | 1 (1.5%) | 0 |
| 7 lines | 1 (1.5%) | 0 |
| CD19+, n (%) | ||
| Yes | 67 (100%) | 48 (100%) |
| No | 0 | 0 |
| Blast in bone marrow at screening, % | ||
| Mean (SD) | 57.1 (29.3) | 53.8 (29.5) |
| Median | 65.0 | 62.5 |
| Min, Max | 5.0, 97.0 | 5.0, 96.5 |
| Blast in bone marrow at screening, n (%) | ||
| ≥5% and ≤25% | 13 (19.4%) | 11 (22.9%) |
| >25% and ≤50% | 11 (16.4%) | 8 (16.7%) |
| >50% and ≤75% | 18 (26.9%) | 13 (27.1%) |
| >75% and ≤100% | 25 (37.3%) | 16 (33.3%) |
| Extramedullary disease at screening, n (%) | ||
| Yes | 2 (3.0%) | 2 (4.2%) |
| No | 65 (97.0%) | 46 (95.8%) |
| Cytogenetic alterations, n (%) | ||
| Alterations of IKZF1 | 15 (22.4%) | 11 (22.9%) |
| Ph+ | 14 (20.9%) | 10 (20.8%) |
| MLL rearrangements | 10 (14.9%) | 7 (14.6%) |
| TP53 gene deletion/mutation | 10 (14.9%) | 7 (14.6%) |
| E2A::PBX1 fusion gene | 2 (3.0%) | 2 (4.2%) |
| Ph-like | 4 (6.0%) | 2 (4.2%) |
| Variables . | Enrolled patients n = 67 . | Treated patients n = 48 . |
|---|---|---|
| Age, median (range), y | 33 (18-59) | 32 (18-58) |
| Sex | ||
| Female | 33 (49.3%) | 22 (45.8%) |
| Male | 34 (50.7%) | 26 (54.2%) |
| ECOG grade, n (%) | ||
| 0 | 29 (43.3%) | 20 (41.7%) |
| 1 | 38 (56.7%) | 28 (58.3%) |
| Disease status at screening, n (%) | ||
| Relapse (first relapse with the first remission lasting <12 mo) | 12 (17.9%) | 9 (18.8%) |
| Refractory | 55 (82.1%) | 39 (81.2%) |
| Refractory subcategories | ||
| Primary refractory | 13 (19.4%) | 12 (25.0%) |
| Relapse after ≥2 CRs | 11 (16.4%) | 8 (16.7%) |
| First relapse, no remission after at least 1 salvage therapy | 20 (29.9%) | 11 (22.9%) |
| Relapsed or refractory after HSCT | 11 (16.4%) | 8 (16.7%) |
| Type of HSCT | ||
| Autologous | 4 (6.0%) | 3 (6.2%) |
| Allogeneic | 7 (10.4%) | 5 (10.4%) |
| Maximum prior lines of therapy, n (%) | ||
| Median (range) | 2.0 (1-7) | 2 (1–4) |
| 1 line | 15 (22.4%) | 13 (27.1%) |
| 2 lines | 35 (52.2%) | 25 (52.1%) |
| 3 lines | 10 (14.9%) | 7 (14.6%) |
| 4 lines | 5 (7.5%) | 3 (6.2%) |
| 5 lines | 1 (1.5%) | 0 |
| 7 lines | 1 (1.5%) | 0 |
| CD19+, n (%) | ||
| Yes | 67 (100%) | 48 (100%) |
| No | 0 | 0 |
| Blast in bone marrow at screening, % | ||
| Mean (SD) | 57.1 (29.3) | 53.8 (29.5) |
| Median | 65.0 | 62.5 |
| Min, Max | 5.0, 97.0 | 5.0, 96.5 |
| Blast in bone marrow at screening, n (%) | ||
| ≥5% and ≤25% | 13 (19.4%) | 11 (22.9%) |
| >25% and ≤50% | 11 (16.4%) | 8 (16.7%) |
| >50% and ≤75% | 18 (26.9%) | 13 (27.1%) |
| >75% and ≤100% | 25 (37.3%) | 16 (33.3%) |
| Extramedullary disease at screening, n (%) | ||
| Yes | 2 (3.0%) | 2 (4.2%) |
| No | 65 (97.0%) | 46 (95.8%) |
| Cytogenetic alterations, n (%) | ||
| Alterations of IKZF1 | 15 (22.4%) | 11 (22.9%) |
| Ph+ | 14 (20.9%) | 10 (20.8%) |
| MLL rearrangements | 10 (14.9%) | 7 (14.6%) |
| TP53 gene deletion/mutation | 10 (14.9%) | 7 (14.6%) |
| E2A::PBX1 fusion gene | 2 (3.0%) | 2 (4.2%) |
| Ph-like | 4 (6.0%) | 2 (4.2%) |
ECOG, Eastern Cooperative Oncology Group; Max, maximum; Min, minimum; MLL, mixed-lineage leukemia; Ph, Philadelphia chromosome; SD, standard deviation.