Demographics and baseline disease characteristics
| . | All patients (n = 260) . | Patients with Mayo stage IV AL amyloidosis (n = 77) . | ||
|---|---|---|---|---|
| Birtamimab + SOC (n = 130) . | Placebo + SOC (n = 130) . | Birtamimab + SOC (n = 38) . | Placebo + SOC (n = 39) . | |
| Age, y; median (Q1, Q3) | 64.2 (57.6, 70.9) | 62.6 (57.0, 69.3) | 63.6 (55.7, 69.8) | 63.7 (57.0, 68.4) |
| Gender, n (%) | ||||
| Male | 82 (63) | 90 (69) | 25 (66) | 28 (72) |
| Female | 48 (37) | 40 (31) | 13 (34) | 11 (28) |
| Ethnicity, n (%) | ||||
| Hispanic or Latino | 2 (2) | 2 (2) | 0 | 0 |
| Not Hispanic or Latino | 116 (89) | 122 (94) | 34 (90) | 36 (92) |
| Not provided or unknown | 12 (9) | 6 (5) | 4 (11) | 3 (8) |
| Race, n (%) | ||||
| White | 118 (91) | 120 (92) | 36 (95) | 36 (92) |
| Black or African American | 9 (7) | 3 (2) | 2 (5) | 2 (5) |
| Asian | 2 (2) | 2 (2) | 0 | 0 |
| Other | 1 (1) | 5 (4) | 0 | 1 (3) |
| Age at AL amyloidosis diagnosis, y; median (Q1, Q3) | 64.1 (57.5, 70.9) | 62.4 (56.8, 69.3) | 63.5 (55.6, 69.7) | 63.8 (56.8, 68.5) |
| Duration since AL amyloidosis diagnosis, mo; median (Q1, Q3) | 1.31 (0.92, 1.87) | 1.48 (0.95, 2.17) | 1.15 (0.69, 1.58) | 1.45 (0.89, 1.81) |
| Number of derived involved organs at baseline; median (Q1, Q3) | 2.0 (1.0, 2.0) | 2.0 (1.0, 2.0) | 1.5 (1.0, 2.0) | 2.0 (1.0, 2.0) |
| Baseline NT-proBNP ≥1800 pg/mL, n (%) | 95 (73) | 100 (77) | 38 (100) | 39 (100) |
| Baseline NT-proBNP, pg/mL; median (Q1, Q3) | 3146.2 (1650.0, 5173.0) | 3183.7 (1910.0, 5551.0) | 5141.3 (3228.0, 5939.4) | 5415.0 (4054.0, 8073.0) |
| Baseline troponin T, ng/mL∗; median (Q1, Q3) | 0.03 (0.02, 0.06) | 0.02 (0.02, 0.08) | 0.05 (0.04, 0.09) | 0.09 (0.06, 0.13) |
| Baseline FLC ratio, median (Q1, Q3) | 0.10 (0.03, 0.32) | 0.11 (0.04, 0.51) | 0.05 (0.02, 0.08) | 0.05 (0.03, 11.14) |
| Baseline dFLC,† mg/dL; median (Q1, Q3) | 26.31 (13.83, 53.05) | 38.18 (18.00, 63.06) | 44.44 (25.13, 56.17) | 57.42 (35.52, 106.28) |
| Mayo stage, n (%) | ||||
| I | 11 (8) | 10 (8) | NA | NA |
| II | 34 (26) | 28 (22) | NA | NA |
| III | 47 (36) | 53 (41) | NA | NA |
| IV | 38 (29) | 39 (30) | 38 (100) | 39 (100) |
| Renal stage, n (%) | ||||
| I | 91 (70) | 96 (74) | 28 (74) | 29 (74) |
| II | 33 (25) | 26 (20) | 9 (24) | 9 (23) |
| III | 6 (5) | 8 (6) | 1 (3) | 1 (3) |
| Baseline 6MWT distance, n (%) | ||||
| <300 m | 44 (34) | 43 (33) | 13 (34) | 16 (41) |
| ≥300 m | 86 (66) | 87 (67) | 25 (66) | 23 (59) |
| . | All patients (n = 260) . | Patients with Mayo stage IV AL amyloidosis (n = 77) . | ||
|---|---|---|---|---|
| Birtamimab + SOC (n = 130) . | Placebo + SOC (n = 130) . | Birtamimab + SOC (n = 38) . | Placebo + SOC (n = 39) . | |
| Age, y; median (Q1, Q3) | 64.2 (57.6, 70.9) | 62.6 (57.0, 69.3) | 63.6 (55.7, 69.8) | 63.7 (57.0, 68.4) |
| Gender, n (%) | ||||
| Male | 82 (63) | 90 (69) | 25 (66) | 28 (72) |
| Female | 48 (37) | 40 (31) | 13 (34) | 11 (28) |
| Ethnicity, n (%) | ||||
| Hispanic or Latino | 2 (2) | 2 (2) | 0 | 0 |
| Not Hispanic or Latino | 116 (89) | 122 (94) | 34 (90) | 36 (92) |
| Not provided or unknown | 12 (9) | 6 (5) | 4 (11) | 3 (8) |
| Race, n (%) | ||||
| White | 118 (91) | 120 (92) | 36 (95) | 36 (92) |
| Black or African American | 9 (7) | 3 (2) | 2 (5) | 2 (5) |
| Asian | 2 (2) | 2 (2) | 0 | 0 |
| Other | 1 (1) | 5 (4) | 0 | 1 (3) |
| Age at AL amyloidosis diagnosis, y; median (Q1, Q3) | 64.1 (57.5, 70.9) | 62.4 (56.8, 69.3) | 63.5 (55.6, 69.7) | 63.8 (56.8, 68.5) |
| Duration since AL amyloidosis diagnosis, mo; median (Q1, Q3) | 1.31 (0.92, 1.87) | 1.48 (0.95, 2.17) | 1.15 (0.69, 1.58) | 1.45 (0.89, 1.81) |
| Number of derived involved organs at baseline; median (Q1, Q3) | 2.0 (1.0, 2.0) | 2.0 (1.0, 2.0) | 1.5 (1.0, 2.0) | 2.0 (1.0, 2.0) |
| Baseline NT-proBNP ≥1800 pg/mL, n (%) | 95 (73) | 100 (77) | 38 (100) | 39 (100) |
| Baseline NT-proBNP, pg/mL; median (Q1, Q3) | 3146.2 (1650.0, 5173.0) | 3183.7 (1910.0, 5551.0) | 5141.3 (3228.0, 5939.4) | 5415.0 (4054.0, 8073.0) |
| Baseline troponin T, ng/mL∗; median (Q1, Q3) | 0.03 (0.02, 0.06) | 0.02 (0.02, 0.08) | 0.05 (0.04, 0.09) | 0.09 (0.06, 0.13) |
| Baseline FLC ratio, median (Q1, Q3) | 0.10 (0.03, 0.32) | 0.11 (0.04, 0.51) | 0.05 (0.02, 0.08) | 0.05 (0.03, 11.14) |
| Baseline dFLC,† mg/dL; median (Q1, Q3) | 26.31 (13.83, 53.05) | 38.18 (18.00, 63.06) | 44.44 (25.13, 56.17) | 57.42 (35.52, 106.28) |
| Mayo stage, n (%) | ||||
| I | 11 (8) | 10 (8) | NA | NA |
| II | 34 (26) | 28 (22) | NA | NA |
| III | 47 (36) | 53 (41) | NA | NA |
| IV | 38 (29) | 39 (30) | 38 (100) | 39 (100) |
| Renal stage, n (%) | ||||
| I | 91 (70) | 96 (74) | 28 (74) | 29 (74) |
| II | 33 (25) | 26 (20) | 9 (24) | 9 (23) |
| III | 6 (5) | 8 (6) | 1 (3) | 1 (3) |
| Baseline 6MWT distance, n (%) | ||||
| <300 m | 44 (34) | 43 (33) | 13 (34) | 16 (41) |
| ≥300 m | 86 (66) | 87 (67) | 25 (66) | 23 (59) |
NA, not applicable.
Mayo stage criteria for troponin T levels were modified from a value of 0.025 ng/mL, cited in the article by Kumar et al,12 to 0.03 ng/mL, the lowest validated determination for the commercially available test.
Baseline dFLC is calculated only for patients with an abnormal baseline FLC ratio (κ:λ <0.26 or >1.65).