Characteristics, mutational status, and cytogenetics of patients treated with venetoclax-azacitidine or placebo-azacitidine in the higher-, intermediate-, and lower-benefit groups
| . | Ven + Aza . | Pbo + Aza . | ||||
|---|---|---|---|---|---|---|
| Higher benefit (n = 145) . | Intermediate benefit (n = 71) . | Lower benefit (n = 63) . | Higher benefit (n = 64) . | Intermediate benefit (n = 28) . | Lower benefit (n = 21) . | |
| Baseline characteristics | ||||||
| Median age, y | 77 | 76 | 77 | 77 | 75 | 75 |
| Age group, n (%) | ||||||
| <75 y | 58 (40) | 27 (38) | 24 (38.1) | 20 (31.2) | 14 (50) | 11 (52.4) |
| ≥75 y | 87 (60) | 44 (62) | 39 (61.9) | 44 (68.8) | 14 (50) | 10 (47.6) |
| AML type, n (%) | ||||||
| Primary | 105 (72.4) | 53 (74.6) | 46 (73) | 45 (70.3) | 24 (85.7) | 15 (71.4) |
| Secondary | 40 (27.6) | 18 (25.4) | 17 (27) | 19 (29.7) | 4 (14.3) | 6 (28.6) |
| ECOG PS, n (%) | ||||||
| <2 | 90 (62.1) | 37 (52.1) | 36 (57.1) | 44 (68.8) | 13 (46.4) | 12 (57.1) |
| ≥2 | 55 (37.9) | 34 (47.9) | 27 (42.9) | 20 (31.2) | 15 (53.6) | 9 (42.9) |
| % Blasts, n (%) | ||||||
| <30% | 37 (25.5) | 15 (21.1) | 28 (44.4) | 18 (28.1) | 4 (14.3) | 10 (47.6) |
| ≥30% to <50% | 38 (26.2) | 10 (14.1) | 13 (20.6) | 15 (23.4) | 6 (21.4) | 5 (23.8) |
| ≥50% | 70 (48.3) | 46 (64.8) | 22 (34.9) | 31 (48.4) | 18 (64.3) | 6 (28.6) |
| Mutations, n (%) | ||||||
| FLT3-ITD | 0 | 39 (54.9) | 4 (6.3) | 0 | 21 (75) | 0 |
| NRAS | 0 | 28 (39.4) | 5 (7.9) | 0 | 7 (25) | 1 (4.8) |
| KRAS | 0 | 11 (15.5) | 2 (3.2) | 0 | 2 (7.1) | 0 |
| TP53 | 0 | 0 | 63 (100) | 0 | 0 | 21 (100) |
| NPM1 | 22 (15.2) | 23 (32.4) | 1 (1.6) | 13 (20.3) | 7 (25) | 0 |
| NPM1/FLT3-ITD negative | 22 (15.2) | 7 (9.9) | 1 (1.6) | 13 (20.3) | 0 | 0 |
| NPM1/FLT3-ITD positive | 0 | 16 (22.5) | 0 | 0 | 7 (25) | 0 |
| IDH1/2 | 53 (36.6) | 21 (29.6) | 3 (4.8) | 19 (29.7) | 4 (14.3) | 0 |
| IDH1 | 22 (15.2) | 9 (12.7) | 1 (1.6) | 7 (10.9) | 2 (7.1) | 0 |
| IDH2 | 32 (22.1) | 13 (18.3) | 2 (3.2) | 13 (20.3) | 2 (7.1) | 0 |
| FLT3-TKD | 12 (8.3) | 3 (4.2) | 1 (1.6) | 9 (14.1) | 2 (7.1) | 1 (4.8) |
| DNMT3A | 40 (27.6) | 25 (35.2) | 7 (11.1) | 15 (23.4) | 13 (46.4) | 4 (19) |
| TET2 | 48 (33.1) | 29 (40.8) | 4 (6.3) | 24 (37.5) | 8 (28.6) | 3 (14.3) |
| ASXL1 | 18 (12.4) | 12 (16.9) | 5 (7.9) | 18 (28.1) | 7 (25) | 2 (9.5) |
| BCOR | 15 (10.3) | 13 (18.3) | 1 (1.6) | 9 (14.1) | 5 (17.9) | 1 (4.8) |
| EZH2 | 8 (5.5) | 6 (8.5) | 2 (3.2) | 6 (9.4) | 0 | 0 |
| RUNX1 | 43 (29.7) | 25 (35.2) | 2 (3.2) | 17 (26.6) | 9 (32.1) | 4 (19) |
| SF3B1 | 16 (11.0) | 6 (8.5) | 1 (1.6) | 5 (7.8) | 3 (10.7) | 1 (4.8) |
| SRSF2 | 42 (29.0) | 16 (22.5) | 4 (6.3) | 22 (34.4) | 8 (28.6) | 4 (19) |
| STAG2 | 21 (14.5) | 11 (15.5) | 2 (3.2) | 13 (20.3) | 7 (25) | 4 (19) |
| U2AF1 | 16 (11.0) | 7 (9.9) | 3 (4.8) | 3 (4.7) | 3 (10.7) | 0 |
| CEBPA | 8 (5.5) | 5 (7.0) | 0 | 7 (10.9) | 4 (14.3) | 1 (4.8) |
| ZRSR2 | 4 (2.8) | 2 (2.8) | 0 | 2 (3.1) | 0 | 0 |
| CEBPA-bZip | 4 (2.8) | 0 | 0 | 2 (3.1) | 1 (3.6) | 0 |
| Cytogenetics, n (%) | ||||||
| Complex karyotype | 19 (13.1) | 3 (4.2) | 50 (79.4) | 6 (9.4) | 1 (3.6) | 18 (85.7) |
| del(5) | 9 (6.2) | 0 | 40 (63.5) | 4 (6.2) | 0 | 15 (71.4) |
| del(7) | 20 (13.9) | 6 (8.5) | 22 (34.9) | 11 (17.2) | 2 (7.1) | 8 (38.1) |
| del(17) | 2 (1.4) | 0 | 13 (20.6) | 0 | 1 (3.6) | 4 (19) |
| t(v;11q23) | 2 (1.4) | 0 | 5 (7.9) | 2 (3.1) | 0 | 1 (4.8) |
| . | Ven + Aza . | Pbo + Aza . | ||||
|---|---|---|---|---|---|---|
| Higher benefit (n = 145) . | Intermediate benefit (n = 71) . | Lower benefit (n = 63) . | Higher benefit (n = 64) . | Intermediate benefit (n = 28) . | Lower benefit (n = 21) . | |
| Baseline characteristics | ||||||
| Median age, y | 77 | 76 | 77 | 77 | 75 | 75 |
| Age group, n (%) | ||||||
| <75 y | 58 (40) | 27 (38) | 24 (38.1) | 20 (31.2) | 14 (50) | 11 (52.4) |
| ≥75 y | 87 (60) | 44 (62) | 39 (61.9) | 44 (68.8) | 14 (50) | 10 (47.6) |
| AML type, n (%) | ||||||
| Primary | 105 (72.4) | 53 (74.6) | 46 (73) | 45 (70.3) | 24 (85.7) | 15 (71.4) |
| Secondary | 40 (27.6) | 18 (25.4) | 17 (27) | 19 (29.7) | 4 (14.3) | 6 (28.6) |
| ECOG PS, n (%) | ||||||
| <2 | 90 (62.1) | 37 (52.1) | 36 (57.1) | 44 (68.8) | 13 (46.4) | 12 (57.1) |
| ≥2 | 55 (37.9) | 34 (47.9) | 27 (42.9) | 20 (31.2) | 15 (53.6) | 9 (42.9) |
| % Blasts, n (%) | ||||||
| <30% | 37 (25.5) | 15 (21.1) | 28 (44.4) | 18 (28.1) | 4 (14.3) | 10 (47.6) |
| ≥30% to <50% | 38 (26.2) | 10 (14.1) | 13 (20.6) | 15 (23.4) | 6 (21.4) | 5 (23.8) |
| ≥50% | 70 (48.3) | 46 (64.8) | 22 (34.9) | 31 (48.4) | 18 (64.3) | 6 (28.6) |
| Mutations, n (%) | ||||||
| FLT3-ITD | 0 | 39 (54.9) | 4 (6.3) | 0 | 21 (75) | 0 |
| NRAS | 0 | 28 (39.4) | 5 (7.9) | 0 | 7 (25) | 1 (4.8) |
| KRAS | 0 | 11 (15.5) | 2 (3.2) | 0 | 2 (7.1) | 0 |
| TP53 | 0 | 0 | 63 (100) | 0 | 0 | 21 (100) |
| NPM1 | 22 (15.2) | 23 (32.4) | 1 (1.6) | 13 (20.3) | 7 (25) | 0 |
| NPM1/FLT3-ITD negative | 22 (15.2) | 7 (9.9) | 1 (1.6) | 13 (20.3) | 0 | 0 |
| NPM1/FLT3-ITD positive | 0 | 16 (22.5) | 0 | 0 | 7 (25) | 0 |
| IDH1/2 | 53 (36.6) | 21 (29.6) | 3 (4.8) | 19 (29.7) | 4 (14.3) | 0 |
| IDH1 | 22 (15.2) | 9 (12.7) | 1 (1.6) | 7 (10.9) | 2 (7.1) | 0 |
| IDH2 | 32 (22.1) | 13 (18.3) | 2 (3.2) | 13 (20.3) | 2 (7.1) | 0 |
| FLT3-TKD | 12 (8.3) | 3 (4.2) | 1 (1.6) | 9 (14.1) | 2 (7.1) | 1 (4.8) |
| DNMT3A | 40 (27.6) | 25 (35.2) | 7 (11.1) | 15 (23.4) | 13 (46.4) | 4 (19) |
| TET2 | 48 (33.1) | 29 (40.8) | 4 (6.3) | 24 (37.5) | 8 (28.6) | 3 (14.3) |
| ASXL1 | 18 (12.4) | 12 (16.9) | 5 (7.9) | 18 (28.1) | 7 (25) | 2 (9.5) |
| BCOR | 15 (10.3) | 13 (18.3) | 1 (1.6) | 9 (14.1) | 5 (17.9) | 1 (4.8) |
| EZH2 | 8 (5.5) | 6 (8.5) | 2 (3.2) | 6 (9.4) | 0 | 0 |
| RUNX1 | 43 (29.7) | 25 (35.2) | 2 (3.2) | 17 (26.6) | 9 (32.1) | 4 (19) |
| SF3B1 | 16 (11.0) | 6 (8.5) | 1 (1.6) | 5 (7.8) | 3 (10.7) | 1 (4.8) |
| SRSF2 | 42 (29.0) | 16 (22.5) | 4 (6.3) | 22 (34.4) | 8 (28.6) | 4 (19) |
| STAG2 | 21 (14.5) | 11 (15.5) | 2 (3.2) | 13 (20.3) | 7 (25) | 4 (19) |
| U2AF1 | 16 (11.0) | 7 (9.9) | 3 (4.8) | 3 (4.7) | 3 (10.7) | 0 |
| CEBPA | 8 (5.5) | 5 (7.0) | 0 | 7 (10.9) | 4 (14.3) | 1 (4.8) |
| ZRSR2 | 4 (2.8) | 2 (2.8) | 0 | 2 (3.1) | 0 | 0 |
| CEBPA-bZip | 4 (2.8) | 0 | 0 | 2 (3.1) | 1 (3.6) | 0 |
| Cytogenetics, n (%) | ||||||
| Complex karyotype | 19 (13.1) | 3 (4.2) | 50 (79.4) | 6 (9.4) | 1 (3.6) | 18 (85.7) |
| del(5) | 9 (6.2) | 0 | 40 (63.5) | 4 (6.2) | 0 | 15 (71.4) |
| del(7) | 20 (13.9) | 6 (8.5) | 22 (34.9) | 11 (17.2) | 2 (7.1) | 8 (38.1) |
| del(17) | 2 (1.4) | 0 | 13 (20.6) | 0 | 1 (3.6) | 4 (19) |
| t(v;11q23) | 2 (1.4) | 0 | 5 (7.9) | 2 (3.1) | 0 | 1 (4.8) |
Aza, azacitidine; ECOG PS, Eastern Cooperative Oncology Group performance status; Pbo, placebo; TKD, tyrosine kinase domain; Ven, venetoclax.