Patient characteristics
| Characteristic . | Phase 1 part . | Phase 2 part (n = 35) . | All treated (n = 72) . | |||
|---|---|---|---|---|---|---|
| Dose escalation . | ||||||
| 1.2 mg/m2 (n = 3) . | 1.6 mg/m2 (n = 12) . | 1.8 mg/m2 (n = 9) . | Dose expansion 1.8 mg/m2 (n = 13) . | |||
| Age, median, y (range) | 64.0 (62-65) | 43 (23-69) | 42 (23-67) | 57 (24-75) | 34 (20-79) | 45 (20-79) |
| <65, n (%) | 2 (67) | 10 (83) | 8 (89) | 10 (77) | 31 (89) | 61 (85) |
| Male, n (%) | 3 (100) | 11 (92) | 3 (33) | 7 (54) | 27 (77) | 51 (71) |
| Race, n (%) | ||||||
| White | 3 (100) | 9 (75) | 8 (89) | 10 (77) | 25 (71) | 55 (76) |
| Asian | 0 | 0 | 1 (11) | 1 (8) | 4 (11) | 6 (8) |
| Black | 0 | 1 (8) | 0 | 1 (8) | 0 | 2 (3) |
| Other/unspecified | 0 | 2 (17) | 0 | 1 (8) | 6 (17) | 9 (13) |
| ECOG performance status, n (%) | ||||||
| 0 | 0 | 4 (33) | 1 (11) | 4 (31) | 3 (9) | 12 (17) |
| 1 | 2 (67) | 5 (42) | 5 (56) | 7 (54) | 22 (63) | 41 (57) |
| 2 | 1 (33) | 3 (25) | 3 (33) | 1 (8) | 8 (23) | 16 (22) |
| 3 | 0 | 0 | 0 | 1 (8) | 2 (6) | 3 (4) |
| Salvage status, n (%) | ||||||
| 1 | 0 | 4 (33) | 3 (33) | 7 (54) | 2 (6)* | 16 (22) |
| 2 | 1 (33) | 5 (42) | 3 (33) | 4 (31) | 16 (46) | 29 (40) |
| 3 | 2 (67) | 2 (17) | 2 (22) | 1 (8) | 11 (31) | 18 (25) |
| 4 | 0 | 0 | 0 | 0 | 2 (6) | 2 (3) |
| 5 | 0 | 1 (8) | 1 (11) | 1 (8) | 1 (3) | 4 (6) |
| >5 | 0 | 0 | 0 | 0 | 3 (9) | 3 (4) |
| Median (range) duration since primary diagnosis, y | 1.5 (0.5-4.9) | 2.1 (0.2-6.6) | 1.1 (0.4-20.4) | 1.6 (0.2-4.9) | 2.5 (0.4-16.0) | 1.7 (0.2-20.4) |
| Response to last induction therapy, n (%) | ||||||
| Relapsed | 1 (33) | 5 (42) | 5 (56) | 7 (54) | 12 (34) | 30 (42) |
| Refractory | 2 (67) | 5 (42) | 4 (44) | 5 (38) | 19 (54) | 35 (49) |
| Unknown | 0 | 2 (17) | 0 | 1 (8) | 4 (11) | 7 (10) |
| Prior SCT, n (%) | 1 (33) | 1 (8) | 3 (33) | 3 (23) | 15 (43) | 23 (32) |
| Cytogenetics, n (%) | ||||||
| Ph+ | 2 (67) | 1 (8) | 1 (11) | 3 (23) | 9 (26) | 16 (22) |
| Complex† | 0 | 1 (8) | 2 (22) | 1 (8) | 11 (31) | 15 (21) |
| Normal‡ | 0 | 6 (50) | 4 (44) | 1 (8) | 4 (11) | 15 (21) |
| Unknown§ | 0 | 1 (8) | 0 | 0 | 3 (9) | 4 (6) |
| Other | 1 (33) | 2 (17) | 2 (22) | 7 (54) | 8 (23) | 20 (28) |
| t(4;11) | 0 | 0 | 0 | 2 (15) | 0 | 2 (15) |
| Median (range) CD22+,‖ % blasts | 99.6 (96-100) | 98.7 (31-100) | 98.4 (97-100) | 95.1 (52-100) | 99.2 (78-100) | 99.0 (31-100) |
| BM blasts, n (%)¶ | ||||||
| <50% | 0 | 4 (33) | 1 (11) | 1 (8) | 5 (14) | 11 (15) |
| ≥50% | 3 (100) | 8 (67) | 8 (89) | 11 (85) | 28 (80) | 58 (81) |
| Peripheral blasts, cells/mm3, n (%) | ||||||
| 0 | 0 | 7 (58) | 5 (56) | 2 (15) | 13 (37) | 27 (38) |
| >0-1000 | 3 (100) | 4 (33) | 0 | 1 (8) | 6 (17) | 14 (19) |
| >1000-5000 | 0 | 0 | 2 (22) | 5 (38) | 7 (20) | 14 (19) |
| >5000-10 000 | 0 | 1 (8) | 1 (11) | 1 (8) | 3 (9) | 6 (8) |
| >10 000 | 0 | 0 | 1 (11) | 4 (31) | 6 (17) | 11 (15) |
| Median (range) WBC, × 109/L | 1.2 (0.6-1.4) | 2.6 (0.5-27.8) | 5.4 (2.5-29.1) | 17.9 (0.5-67.2) | 3.3 (0.5-57.1) | 4.4 (0.5-67.2) |
| Characteristic . | Phase 1 part . | Phase 2 part (n = 35) . | All treated (n = 72) . | |||
|---|---|---|---|---|---|---|
| Dose escalation . | ||||||
| 1.2 mg/m2 (n = 3) . | 1.6 mg/m2 (n = 12) . | 1.8 mg/m2 (n = 9) . | Dose expansion 1.8 mg/m2 (n = 13) . | |||
| Age, median, y (range) | 64.0 (62-65) | 43 (23-69) | 42 (23-67) | 57 (24-75) | 34 (20-79) | 45 (20-79) |
| <65, n (%) | 2 (67) | 10 (83) | 8 (89) | 10 (77) | 31 (89) | 61 (85) |
| Male, n (%) | 3 (100) | 11 (92) | 3 (33) | 7 (54) | 27 (77) | 51 (71) |
| Race, n (%) | ||||||
| White | 3 (100) | 9 (75) | 8 (89) | 10 (77) | 25 (71) | 55 (76) |
| Asian | 0 | 0 | 1 (11) | 1 (8) | 4 (11) | 6 (8) |
| Black | 0 | 1 (8) | 0 | 1 (8) | 0 | 2 (3) |
| Other/unspecified | 0 | 2 (17) | 0 | 1 (8) | 6 (17) | 9 (13) |
| ECOG performance status, n (%) | ||||||
| 0 | 0 | 4 (33) | 1 (11) | 4 (31) | 3 (9) | 12 (17) |
| 1 | 2 (67) | 5 (42) | 5 (56) | 7 (54) | 22 (63) | 41 (57) |
| 2 | 1 (33) | 3 (25) | 3 (33) | 1 (8) | 8 (23) | 16 (22) |
| 3 | 0 | 0 | 0 | 1 (8) | 2 (6) | 3 (4) |
| Salvage status, n (%) | ||||||
| 1 | 0 | 4 (33) | 3 (33) | 7 (54) | 2 (6)* | 16 (22) |
| 2 | 1 (33) | 5 (42) | 3 (33) | 4 (31) | 16 (46) | 29 (40) |
| 3 | 2 (67) | 2 (17) | 2 (22) | 1 (8) | 11 (31) | 18 (25) |
| 4 | 0 | 0 | 0 | 0 | 2 (6) | 2 (3) |
| 5 | 0 | 1 (8) | 1 (11) | 1 (8) | 1 (3) | 4 (6) |
| >5 | 0 | 0 | 0 | 0 | 3 (9) | 3 (4) |
| Median (range) duration since primary diagnosis, y | 1.5 (0.5-4.9) | 2.1 (0.2-6.6) | 1.1 (0.4-20.4) | 1.6 (0.2-4.9) | 2.5 (0.4-16.0) | 1.7 (0.2-20.4) |
| Response to last induction therapy, n (%) | ||||||
| Relapsed | 1 (33) | 5 (42) | 5 (56) | 7 (54) | 12 (34) | 30 (42) |
| Refractory | 2 (67) | 5 (42) | 4 (44) | 5 (38) | 19 (54) | 35 (49) |
| Unknown | 0 | 2 (17) | 0 | 1 (8) | 4 (11) | 7 (10) |
| Prior SCT, n (%) | 1 (33) | 1 (8) | 3 (33) | 3 (23) | 15 (43) | 23 (32) |
| Cytogenetics, n (%) | ||||||
| Ph+ | 2 (67) | 1 (8) | 1 (11) | 3 (23) | 9 (26) | 16 (22) |
| Complex† | 0 | 1 (8) | 2 (22) | 1 (8) | 11 (31) | 15 (21) |
| Normal‡ | 0 | 6 (50) | 4 (44) | 1 (8) | 4 (11) | 15 (21) |
| Unknown§ | 0 | 1 (8) | 0 | 0 | 3 (9) | 4 (6) |
| Other | 1 (33) | 2 (17) | 2 (22) | 7 (54) | 8 (23) | 20 (28) |
| t(4;11) | 0 | 0 | 0 | 2 (15) | 0 | 2 (15) |
| Median (range) CD22+,‖ % blasts | 99.6 (96-100) | 98.7 (31-100) | 98.4 (97-100) | 95.1 (52-100) | 99.2 (78-100) | 99.0 (31-100) |
| BM blasts, n (%)¶ | ||||||
| <50% | 0 | 4 (33) | 1 (11) | 1 (8) | 5 (14) | 11 (15) |
| ≥50% | 3 (100) | 8 (67) | 8 (89) | 11 (85) | 28 (80) | 58 (81) |
| Peripheral blasts, cells/mm3, n (%) | ||||||
| 0 | 0 | 7 (58) | 5 (56) | 2 (15) | 13 (37) | 27 (38) |
| >0-1000 | 3 (100) | 4 (33) | 0 | 1 (8) | 6 (17) | 14 (19) |
| >1000-5000 | 0 | 0 | 2 (22) | 5 (38) | 7 (20) | 14 (19) |
| >5000-10 000 | 0 | 1 (8) | 1 (11) | 1 (8) | 3 (9) | 6 (8) |
| >10 000 | 0 | 0 | 1 (11) | 4 (31) | 6 (17) | 11 (15) |
| Median (range) WBC, × 109/L | 1.2 (0.6-1.4) | 2.6 (0.5-27.8) | 5.4 (2.5-29.1) | 17.9 (0.5-67.2) | 3.3 (0.5-57.1) | 4.4 (0.5-67.2) |
WBC, white blood cells.
Two patients enrolled in the phase 2 part were undergoing first salvage (protocol deviations).
Patients had ≥5 chromosomal aberrations.
Based on a minimum of 20 metaphases.
Two patients had missing data (phase 1 [1.6 mg/m2], n = 1; phase 1 dose expansion, n = 1).
Based on central laboratory analysis; the results for 2 patients were based on peripheral blood analysis (aspirate not collected).
Three patients had missing data (phase 1 dose expansion, n = 1; phase 2, n = 2).