Patient demographics and baseline disease characteristics
| . | Dose-escalation cohort (n = 11) . | Dose-expansion cohort (n = 16) . | RP2/3D cohort (N = 22) . | Total (N = 27) . |
|---|---|---|---|---|
| Median age, y (range) | 66.0 (54-77) | 64.5 (57-78) | 64.5 (54-78) | 65.0 (54-78) |
| Male, n (%) | 5 (45) | 9 (56) | 13 (59) | 14 (52) |
| White, n (%) | 10 (91) | 13 (81) | 18 (82) | 23 (85) |
| NYHA classification,n (%)* | ||||
| Class I | 4 (36) | 3 (19) | 6 (27) | 7 (26) |
| Class II | 4 (36) | 9 (56) | 10 (45) | 13 (48) |
| Mayo cardiac biomarker stage, n (%) | ||||
| Stage I | 5 (45) | 8 (50) | 11 (50) | 13 (48) |
| Stage II | 5 (45) | 8 (50) | 10 (45) | 13 (48) |
| Stage III† | 1 (9) | 0 | 1 (5) | 1 (4) |
| Site of AL organ involvement at study entry, n (%) | ||||
| Kidney and heart | 4 (36) | 2 (13) | 5 (23) | 6 (22) |
| Heart (not kidney) | 3 (27) | 8 (50) | 9 (41) | 11 (41) |
| Kidney (not heart) | 4 (36) | 6 (38) | 8 (36) | 10 (37) |
| Median number of organ systems involved, n (range) | 2.0 (1-6) | 1.5 (1-4) | 2.0 (1-6) | 2.0 (1-6) |
| Median dFLC at screening, mg/L (range) | 104 (40-502) | 126 (46-1064) | 121 (40-1064) | 121 (40-1064) |
| Median NTproBNP at screening, pg/mL (range) | 1490.5 (105.1-4033) | 471 (51-5691) | 669.5 (51-5691) | 897.4 (51-5691) |
| Median 24-h urine protein, g/24 h (range) | 4.8 (0.1-22) | 0.5 (0.004-16.5) | 0.6 (0.004-22) | 0.5 (0.004-22) |
| Median time from diagnosis, months (range) | 33.0 (11-86) | 72.5 (10-148) | 37.5 (10-148) | 51.0 (10-148) |
| Median number of prior therapies, n (range) | 3.0 (1-7) | 3.0 (1-8) | 3.0 (1-8) | 3.0 (1-8) |
| Prior therapy type, n (%)‡ | ||||
| Melphalan-containing regimen | 10 (91) | 16 (100) | 21 (95) | 26 (96) |
| Dexamethasone-containing regimen | 9 (82) | 11 (69) | 16 (73) | 20 (74) |
| Bortezomib-containing regimen§ | 8 (73) | 11 (69) | 16 (73) | 19 (70) |
| Autologous stem-cell transplant | 7 (64) | 11 (69) | 16 (73) | 18 (67) |
| Immunomodulatory drug-containing regimen | 5 (45) | 9 (56) | 11 (50) | 14 (52) |
| Cyclophosphamide-containing regimen | 5 (45) | 7 (44) | 10 (45) | 12 (44) |
| . | Dose-escalation cohort (n = 11) . | Dose-expansion cohort (n = 16) . | RP2/3D cohort (N = 22) . | Total (N = 27) . |
|---|---|---|---|---|
| Median age, y (range) | 66.0 (54-77) | 64.5 (57-78) | 64.5 (54-78) | 65.0 (54-78) |
| Male, n (%) | 5 (45) | 9 (56) | 13 (59) | 14 (52) |
| White, n (%) | 10 (91) | 13 (81) | 18 (82) | 23 (85) |
| NYHA classification,n (%)* | ||||
| Class I | 4 (36) | 3 (19) | 6 (27) | 7 (26) |
| Class II | 4 (36) | 9 (56) | 10 (45) | 13 (48) |
| Mayo cardiac biomarker stage, n (%) | ||||
| Stage I | 5 (45) | 8 (50) | 11 (50) | 13 (48) |
| Stage II | 5 (45) | 8 (50) | 10 (45) | 13 (48) |
| Stage III† | 1 (9) | 0 | 1 (5) | 1 (4) |
| Site of AL organ involvement at study entry, n (%) | ||||
| Kidney and heart | 4 (36) | 2 (13) | 5 (23) | 6 (22) |
| Heart (not kidney) | 3 (27) | 8 (50) | 9 (41) | 11 (41) |
| Kidney (not heart) | 4 (36) | 6 (38) | 8 (36) | 10 (37) |
| Median number of organ systems involved, n (range) | 2.0 (1-6) | 1.5 (1-4) | 2.0 (1-6) | 2.0 (1-6) |
| Median dFLC at screening, mg/L (range) | 104 (40-502) | 126 (46-1064) | 121 (40-1064) | 121 (40-1064) |
| Median NTproBNP at screening, pg/mL (range) | 1490.5 (105.1-4033) | 471 (51-5691) | 669.5 (51-5691) | 897.4 (51-5691) |
| Median 24-h urine protein, g/24 h (range) | 4.8 (0.1-22) | 0.5 (0.004-16.5) | 0.6 (0.004-22) | 0.5 (0.004-22) |
| Median time from diagnosis, months (range) | 33.0 (11-86) | 72.5 (10-148) | 37.5 (10-148) | 51.0 (10-148) |
| Median number of prior therapies, n (range) | 3.0 (1-7) | 3.0 (1-8) | 3.0 (1-8) | 3.0 (1-8) |
| Prior therapy type, n (%)‡ | ||||
| Melphalan-containing regimen | 10 (91) | 16 (100) | 21 (95) | 26 (96) |
| Dexamethasone-containing regimen | 9 (82) | 11 (69) | 16 (73) | 20 (74) |
| Bortezomib-containing regimen§ | 8 (73) | 11 (69) | 16 (73) | 19 (70) |
| Autologous stem-cell transplant | 7 (64) | 11 (69) | 16 (73) | 18 (67) |
| Immunomodulatory drug-containing regimen | 5 (45) | 9 (56) | 11 (50) | 14 (52) |
| Cyclophosphamide-containing regimen | 5 (45) | 7 (44) | 10 (45) | 12 (44) |