Patients’ demographics and baseline disease characteristics
| Characteristic . | Dose-escalation cohorts (n = 26) . | Expansion cohorts (n = 40)* . | Total (n = 60) . |
|---|---|---|---|
| Median age, years (range) | 65 (50-83) | 65 (50-86) | 65 (50-86) |
| Male, n (%) | 16 (62) | 19 (48) | 32 (53) |
| Race, n (%) | |||
| White | 22 (85) | 38 (95) | 54 (90) |
| African American | 4 (15) | 1 (3) | 5 (8) |
| Other | 0 | 1 (3) | 1 (2) |
| ECOG performance status, n (%)† | |||
| 0 | 6 (23) | 16 (40) | 19 (32) |
| 1 | 17 (65) | 20 (50) | 35 (58) |
| 2 | 3 (12) | 3 (8) | 5 (8) |
| ISS disease stage, n (%)‡ | |||
| I | 8 (31) | 13 (33) | 20 (33) |
| II | 11 (42) | 13 (33) | 20 (33) |
| III | 7 (27) | 13 (33) | 19 (32) |
| Median creatinine clearance, mL/min (range) | 76.9 (23.5-163.0) | 67.7 (25.7-125.3) | 73.6 (23.5-163.0) |
| Creatinine clearance <50 mL/min, n (%)§ | 7 (27) | 13 (33) | 18 (30) |
| Median time since MM diagnosis, years (range) | 4.7 (1.1-24.3) | 4.9 (1.0-12.6) | 4.8 (1.0-24.3) |
| MM subtype, n (%) | |||
| IgG | 14 (54) | 27 (68) | 38 (63) |
| IgA | 5 (19) | 8 (20) | 11 (18) |
| Λ light chain | 4 (15) | 2 (5) | 6 (10) |
| κ light chain | 3 (12) | 3 (8) | 5 (8) |
| Median number of prior lines of therapy, n (range) | 4 (2-28) | 4 (1-12) | 4 (1-28) |
| Prior therapy included:, n (%) | |||
| Bortezomib | 26 (100) | 33 (83) | 53 (88) |
| Lenalidomide | 23 (88) | 35 (88) | 53 (88) |
| Thalidomide | 16 (62) | 26 (65) | 37 (62) |
| Carfilzomib/marizomib | 1 (4) | 2 (5) | 3 (5) |
| Stem cell transplantation | 16 (62) | 23 (58) | 36 (60) |
| Patients refractory to their last prior therapy, n (%)¶ | 14 (58) | 25 (64) | 34 (60) |
| Bortezomib-refractory, n (%) | 7 (27) | 12 (30) | 16 (27) |
| Cytogenetic abnormalities, n (%) | |||
| del 17 | 4 (16) | 3 (10) | 5 (10) |
| t(4;14) | 1 (4) | 2 (7) | 2 (4) |
| Hyperdiploidy | 3 (12) | 7 (23) | 8 (16) |
| Characteristic . | Dose-escalation cohorts (n = 26) . | Expansion cohorts (n = 40)* . | Total (n = 60) . |
|---|---|---|---|
| Median age, years (range) | 65 (50-83) | 65 (50-86) | 65 (50-86) |
| Male, n (%) | 16 (62) | 19 (48) | 32 (53) |
| Race, n (%) | |||
| White | 22 (85) | 38 (95) | 54 (90) |
| African American | 4 (15) | 1 (3) | 5 (8) |
| Other | 0 | 1 (3) | 1 (2) |
| ECOG performance status, n (%)† | |||
| 0 | 6 (23) | 16 (40) | 19 (32) |
| 1 | 17 (65) | 20 (50) | 35 (58) |
| 2 | 3 (12) | 3 (8) | 5 (8) |
| ISS disease stage, n (%)‡ | |||
| I | 8 (31) | 13 (33) | 20 (33) |
| II | 11 (42) | 13 (33) | 20 (33) |
| III | 7 (27) | 13 (33) | 19 (32) |
| Median creatinine clearance, mL/min (range) | 76.9 (23.5-163.0) | 67.7 (25.7-125.3) | 73.6 (23.5-163.0) |
| Creatinine clearance <50 mL/min, n (%)§ | 7 (27) | 13 (33) | 18 (30) |
| Median time since MM diagnosis, years (range) | 4.7 (1.1-24.3) | 4.9 (1.0-12.6) | 4.8 (1.0-24.3) |
| MM subtype, n (%) | |||
| IgG | 14 (54) | 27 (68) | 38 (63) |
| IgA | 5 (19) | 8 (20) | 11 (18) |
| Λ light chain | 4 (15) | 2 (5) | 6 (10) |
| κ light chain | 3 (12) | 3 (8) | 5 (8) |
| Median number of prior lines of therapy, n (range) | 4 (2-28) | 4 (1-12) | 4 (1-28) |
| Prior therapy included:, n (%) | |||
| Bortezomib | 26 (100) | 33 (83) | 53 (88) |
| Lenalidomide | 23 (88) | 35 (88) | 53 (88) |
| Thalidomide | 16 (62) | 26 (65) | 37 (62) |
| Carfilzomib/marizomib | 1 (4) | 2 (5) | 3 (5) |
| Stem cell transplantation | 16 (62) | 23 (58) | 36 (60) |
| Patients refractory to their last prior therapy, n (%)¶ | 14 (58) | 25 (64) | 34 (60) |
| Bortezomib-refractory, n (%) | 7 (27) | 12 (30) | 16 (27) |
| Cytogenetic abnormalities, n (%) | |||
| del 17 | 4 (16) | 3 (10) | 5 (10) |
| t(4;14) | 1 (4) | 2 (7) | 2 (4) |
| Hyperdiploidy | 3 (12) | 7 (23) | 8 (16) |
ECOG, Eastern Cooperative Oncology Group; ISS, International Staging System.
Includes 6 patients treated at 2.0 mg/m2 in the dose-escalation cohorts.
Not done for 1 patient in the expansion cohorts.
Unknown in 1 patient in the expansion cohorts.
Three patients, 2 in the dose-escalation cohorts and 2 in the expansion cohorts (including 1 treated at 2.0 mg/m2 in the dose-escalation cohort) had creatinine clearance of 21 to 30 mL/minute.
Missing for 2 patients in the dose-escalation cohorts, 1 patient in the expansion cohorts, and 3 patients overall.