Baseline patient characteristics
| Characteristic . | Phase 1/2 Population (n = 22) . | RP2D Population (n = 14) . |
|---|---|---|
| Age (y), median (range) | 65 (45-80) | 66.5 (60-80) |
| Sex | ||
| Male | 14 (64) | 9 (64) |
| Race | ||
| White | 16 (73) | 10 (71) |
| ECOG performance status | ||
| 0 | 7 (32) | 5 (36) |
| 1 | 12 (54) | 6 (43) |
| 2 | 3 (14) | 3 (21) |
| Median time from diagnosis, d | 21 | 17 |
| Monoclonal light chain and IgH isotype | ||
| Λ | 19 (86) | 12 (86) |
| Λ light chain only | 8 (36) | 6 (43) |
| IgG λ | 5 (23) | 4 (29) |
| IgA λ | 3 (14) | 1 (7) |
| IgM λ | 2 (9) | 0 (0) |
| IgD λ | 1 (4.5) | 1 (7) |
| κ | 3 (14) | 2 (14) |
| κ light chain only | 2 (9) | 1 (7) |
| IgG κ | 1 (4.5) | 1 (7) |
| dFLC (mg/L), median (range) | 27.8 (5.7-114.8) | 25.5 (5.7-114.8) |
| Organ involvement at screening, median (range) | 2 (1-3) | 2 (1-2) |
| Multiorgan involvement (≥2 organs) | 17 (77) | 9 (64) |
| Involved organ* | ||
| Cardiac | 18 (82) | 10 (71) |
| Renal | 8 (36) | 4 (29) |
| GI (nonhepatic) | 4 (18) | 4 (29) |
| GI (hepatic) | 1 (5) | 0 (0) |
| Mayo 2004/EU cardiac stage12,13 | ||
| I | 6 (27) | 5 (36) |
| II | 9 (41) | 5 (36) |
| IIIa | 6 (27) | 4 (29) |
| Missing data | 1 (6) | 0 (0) |
| Evaluable for cardiac response† | 15 (68) | 9 (64) |
| NYHA class | ||
| I | 12 (55) | 9 (64) |
| II | 9 (41) | 5 (36) |
| Missing data | 1 (5) | 0 (0) |
| NT-proBNP (pg/mL), median (range) | 1521 (56-7621) | 1625 (56-7621) |
| Renal stage11 | ||
| I | 12 (55) | 7 (50) |
| II | 9 (41) | 7 (50) |
| III | 1 (5) | 0 (0) |
| Evaluable for renal response‡ | 12 (55) | 6 (43) |
| Median baseline eGFR | 17 | 9 |
| >50 mL/min per 1.73 m2 | 13 (76) | 6 (67) |
| ≤50 mL/min per 1.73 m2 | 4 (24) | 3 (33) |
| 24-h urine protein (mg/24h), median (range) | 1209 (0-15884) | 433 (0-15884) |
| Characteristic . | Phase 1/2 Population (n = 22) . | RP2D Population (n = 14) . |
|---|---|---|
| Age (y), median (range) | 65 (45-80) | 66.5 (60-80) |
| Sex | ||
| Male | 14 (64) | 9 (64) |
| Race | ||
| White | 16 (73) | 10 (71) |
| ECOG performance status | ||
| 0 | 7 (32) | 5 (36) |
| 1 | 12 (54) | 6 (43) |
| 2 | 3 (14) | 3 (21) |
| Median time from diagnosis, d | 21 | 17 |
| Monoclonal light chain and IgH isotype | ||
| Λ | 19 (86) | 12 (86) |
| Λ light chain only | 8 (36) | 6 (43) |
| IgG λ | 5 (23) | 4 (29) |
| IgA λ | 3 (14) | 1 (7) |
| IgM λ | 2 (9) | 0 (0) |
| IgD λ | 1 (4.5) | 1 (7) |
| κ | 3 (14) | 2 (14) |
| κ light chain only | 2 (9) | 1 (7) |
| IgG κ | 1 (4.5) | 1 (7) |
| dFLC (mg/L), median (range) | 27.8 (5.7-114.8) | 25.5 (5.7-114.8) |
| Organ involvement at screening, median (range) | 2 (1-3) | 2 (1-2) |
| Multiorgan involvement (≥2 organs) | 17 (77) | 9 (64) |
| Involved organ* | ||
| Cardiac | 18 (82) | 10 (71) |
| Renal | 8 (36) | 4 (29) |
| GI (nonhepatic) | 4 (18) | 4 (29) |
| GI (hepatic) | 1 (5) | 0 (0) |
| Mayo 2004/EU cardiac stage12,13 | ||
| I | 6 (27) | 5 (36) |
| II | 9 (41) | 5 (36) |
| IIIa | 6 (27) | 4 (29) |
| Missing data | 1 (6) | 0 (0) |
| Evaluable for cardiac response† | 15 (68) | 9 (64) |
| NYHA class | ||
| I | 12 (55) | 9 (64) |
| II | 9 (41) | 5 (36) |
| Missing data | 1 (5) | 0 (0) |
| NT-proBNP (pg/mL), median (range) | 1521 (56-7621) | 1625 (56-7621) |
| Renal stage11 | ||
| I | 12 (55) | 7 (50) |
| II | 9 (41) | 7 (50) |
| III | 1 (5) | 0 (0) |
| Evaluable for renal response‡ | 12 (55) | 6 (43) |
| Median baseline eGFR | 17 | 9 |
| >50 mL/min per 1.73 m2 | 13 (76) | 6 (67) |
| ≤50 mL/min per 1.73 m2 | 4 (24) | 3 (33) |
| 24-h urine protein (mg/24h), median (range) | 1209 (0-15884) | 433 (0-15884) |
dFLC, difference in the involved and uninvolved free light chains; ECOG, Eastern Cooperative Oncology Group; eGFR, estimated glomerular filtration rate; GI, gastrointestinal; NYHA, New York Heart Association.
Data are n (%) unless noted.
n (%) includes all patients with ≥1 organ involved.
Based on the Palladini 2012 response criteria (ie, baseline NT-proBNP ≥650 ng/L required for cardiac response determination).10
Includes patients who met the International Society of Amyloidosis (ISA) 2005 criteria for renal involvement (ie, proteinuria >0.5 g per 24 hours, predominantly albumin).14 Renal response based on the Palladini 2014 renal response criteria.11