Patient characteristics and outcomes
| . | CEBPA+ subset 1 (n = 26) . | CEBPA+ subset 2 (n = 10) . | CEBPA+ subset 3 (n = 17) . | All CEBPA+ (n = 53) . | All CEBPA wt (n = 585) . | P* . |
|---|---|---|---|---|---|---|
| Patient characteristic | ||||||
| Median age, y (range) | 44 (16-62) | 55 (24-64) | 51 (23-64) | 48 (16-64) | 47 (16-70) | .73 |
| Male/female, n | 17/9 | 6/4 | 12/5 | 35/18 | 305/280 | .06 |
| Median WBC, ×109/L (range) | 21 (0.5-250) | 56 (2-170) | 9 (2-96) | 20 (0.5-250) | 13 (0.6-400) | .17 |
| Secondary AML, n (%) | 0 | 0 | 0 | 0 (0%) | 12 (2%) | .61 |
| Cytogenetics, n (%) | ||||||
| Favorable | 0 | 0 | 2 | 2 (4%)† | 82 (14%) | .05 |
| Intermediate | 26 | 10 | 10 | 46 (87%)‡ | 425 (73%) | |
| Unfavorable | 0 | 0 | 5 | 5 (9%)§ | 78 (13%) | |
| CN-AML, n (%) | ||||||
| Yes | 26 | 10 | 0 | 36 (68%) | 285 (49%) | .01 |
| No | 0 | 0 | 17 | 17 (32%) | 300 (51%) | |
| FLT3-ITD, n (%) | ||||||
| Yes | 0 | 10 | 1 | 11 (21%) | 94 (16%) | .44 |
| No | 26 | 0 | 16 | 42 (79%) | 491 (84%) | |
| NPM1 mutation, n (%) | ||||||
| Yes | 3 | 1 | 0 | 4 (7%) | 139 (24%) | .008 |
| No | 19 | 4 | 16 | 39 (74%) | 376 (64%) | |
| Not available | 4 | 5 | 1 | 10 (19%) | 70 (12%) | |
| ALFA trial, n (%) | ||||||
| 9000 | 6 | 5 | 2 | 13 (24%) | 109 (19%) | .53 |
| 9801 | 6 | 4 | 9 | 19 (36%) | 212 (36%) | |
| 9802 | 14 | 1 | 6 | 21 (40%) | 264 (45%) | |
| Patient outcome | ||||||
| Patients, n‖ | 26 | 10 | 15 | 51 | 491 | |
| CR1 induction, n (%) | ||||||
| CR | 23 (88%)¶ | 7 (70%)# | 11 (73%)** | 41 (80%) | 404 (82%) | .54 |
| Refractory AML | 2 | 1 | 2 | 5 (10%) | 58 (12%) | |
| Early death | 1 | 2 | 2 | 5 (10%) | 29 (6%) | |
| Events in CR1, n (%) | ||||||
| Allo-SCT in CR1 | 3 | 0 | 1 | 4 (9%) | 71 (16%) | .27 |
| Relapse | 8 | 4 | 6 | 18 (44%) | 254 (63%) | .03 |
| Death in CR | 0 | 0 | 2 | 2 (5%) | 31 (8%) | .76 |
| 5-year RFS (95% CI) | 62% (38-79)¶ | 43% (10-73)# | 34% (8-63)** | 52% (35-67) | 27% (22-32) | .04 |
| 5-year DFS (95% CI) | 62% (38-79)¶ | 43% (10-73)# | 27% (7-54)** | 50% (33-64) | 24% (19-29) | .03 |
| 5-year OS (95% CI) | 64% (36-82)¶ | 30% (7-58)# | 32% (11-56)** | 47% (31-61) | 31% (27-36) | .11 |
| . | CEBPA+ subset 1 (n = 26) . | CEBPA+ subset 2 (n = 10) . | CEBPA+ subset 3 (n = 17) . | All CEBPA+ (n = 53) . | All CEBPA wt (n = 585) . | P* . |
|---|---|---|---|---|---|---|
| Patient characteristic | ||||||
| Median age, y (range) | 44 (16-62) | 55 (24-64) | 51 (23-64) | 48 (16-64) | 47 (16-70) | .73 |
| Male/female, n | 17/9 | 6/4 | 12/5 | 35/18 | 305/280 | .06 |
| Median WBC, ×109/L (range) | 21 (0.5-250) | 56 (2-170) | 9 (2-96) | 20 (0.5-250) | 13 (0.6-400) | .17 |
| Secondary AML, n (%) | 0 | 0 | 0 | 0 (0%) | 12 (2%) | .61 |
| Cytogenetics, n (%) | ||||||
| Favorable | 0 | 0 | 2 | 2 (4%)† | 82 (14%) | .05 |
| Intermediate | 26 | 10 | 10 | 46 (87%)‡ | 425 (73%) | |
| Unfavorable | 0 | 0 | 5 | 5 (9%)§ | 78 (13%) | |
| CN-AML, n (%) | ||||||
| Yes | 26 | 10 | 0 | 36 (68%) | 285 (49%) | .01 |
| No | 0 | 0 | 17 | 17 (32%) | 300 (51%) | |
| FLT3-ITD, n (%) | ||||||
| Yes | 0 | 10 | 1 | 11 (21%) | 94 (16%) | .44 |
| No | 26 | 0 | 16 | 42 (79%) | 491 (84%) | |
| NPM1 mutation, n (%) | ||||||
| Yes | 3 | 1 | 0 | 4 (7%) | 139 (24%) | .008 |
| No | 19 | 4 | 16 | 39 (74%) | 376 (64%) | |
| Not available | 4 | 5 | 1 | 10 (19%) | 70 (12%) | |
| ALFA trial, n (%) | ||||||
| 9000 | 6 | 5 | 2 | 13 (24%) | 109 (19%) | .53 |
| 9801 | 6 | 4 | 9 | 19 (36%) | 212 (36%) | |
| 9802 | 14 | 1 | 6 | 21 (40%) | 264 (45%) | |
| Patient outcome | ||||||
| Patients, n‖ | 26 | 10 | 15 | 51 | 491 | |
| CR1 induction, n (%) | ||||||
| CR | 23 (88%)¶ | 7 (70%)# | 11 (73%)** | 41 (80%) | 404 (82%) | .54 |
| Refractory AML | 2 | 1 | 2 | 5 (10%) | 58 (12%) | |
| Early death | 1 | 2 | 2 | 5 (10%) | 29 (6%) | |
| Events in CR1, n (%) | ||||||
| Allo-SCT in CR1 | 3 | 0 | 1 | 4 (9%) | 71 (16%) | .27 |
| Relapse | 8 | 4 | 6 | 18 (44%) | 254 (63%) | .03 |
| Death in CR | 0 | 0 | 2 | 2 (5%) | 31 (8%) | .76 |
| 5-year RFS (95% CI) | 62% (38-79)¶ | 43% (10-73)# | 34% (8-63)** | 52% (35-67) | 27% (22-32) | .04 |
| 5-year DFS (95% CI) | 62% (38-79)¶ | 43% (10-73)# | 27% (7-54)** | 50% (33-64) | 24% (19-29) | .03 |
| 5-year OS (95% CI) | 64% (36-82)¶ | 30% (7-58)# | 32% (11-56)** | 47% (31-61) | 31% (27-36) | .11 |
For the CEBPA+ versus CEBPA-wt comparison.
Both had inv(16) AML.
Including 36 normal karyotype, 3 del(9q), 1 trisomy 13, 1 trisomy 8, 1 trisomy 21, 1 abnormality 11q23, and 3 other various single abnormalities.
Including 2 complex karyotypes, 2 monosomy 7, and 1 abnormality 3q.
Patients with favorable cytogenetics as those with secondary AML were not included in outcome estimations and comparisons.
P values: .99, .030, .013, and .035 for CR rate, RFS, DFS, and OS, respectively, compared with the corresponding subset of 221 CEBPA-wt patients with de novo CN-AML and no FLT3-ITD, in whom the CR rate was 87%, and 5-year RFS, DFS, and OS, 29% (22%-37%), 26% (19%-34%), and 37% (30%-45%), respectively.
P = .69, .51, .33, and .85 for CR rate, RFS, DFS, and OS, respectively, compared with the corresponding subset of 59 CEBPA-wt patients with de novo CN-AML and FLT3-ITD, in whom the CR rate was 78%, and 5-year RFS, DFS, and OS, 27% (14%-43%), 23% (11%-37%), and 28% (17%-41%), respectively.
P = .75, .91, .55, and .80 for CR rate, RFS, DFS, and OS, respectively, compared with the corresponding subset of 211 CEBPA-wt patients with de novo AML and non-CBF abnormal karyotype, in whom the CR rate was 78%, and 5-year RFS, DFS, and OS, 23% (15%-31%), 20% (14%-28%), and 26% (20%-33%), respectively.