Patient characteristics and outcomes according to CEBPA, FLT3, and NPM1 mutational status
| Characteristics . | mutCEBPA (n = 7), n (%) . | wtCEBPA (n = 105), n (%) . | P value . | mutFLT3 (n = 9), n (%) . | wtFLT3 (n = 117), n (%) . | P value . | mutNPM1 (n = 11), n (%) . | wtNPM1 (n = 115), n (%) . | P value . |
|---|---|---|---|---|---|---|---|---|---|
| Age, y, median (range) | 64 (50-76) | 71 (28-95) | .08 | 63 (55-75) | 71 (28-95) | .04 | 60 (47-86) | 71 (28-95) | .1 |
| Sex (men/women) | 7/0 (100/0) | 79/26 (75.2/24.8) | NS | 7/2 (77.8/22.2) | 88/29 (75.2/24.8) | NS | 8/3 (72.7/27.3) | 87/28 (75.7/24.3) | NS |
| Leukocytes, ×109/L, median (range) | 8.9 (4-78) | 8.3 (2-94) | NS | 22 (6-56) | 7.7 (2-94) | .004 | 20.4 (10-31) | 7.6 (2-94) | .002 |
| Neutrophils, median (range) | 2.6 (0.4-41) | 4 (0.4-40.3) | NS | 7.2 (2-20) | 3.5 (0.4-41) | .1 | 5.2 (2.2-20) | 3.5 (0.4-41) | .05 |
| Monocytes, median (range) | 1.4 (1.1-29) | 1.8 (0.5-33.7) | NS | 3 (1.5-24) | 1.6 (0.5-34) | .01 | 5 (1.5-12.2) | 1.6 (0.5-33.7) | .002 |
| % monocytes, median (range) | 22.5 (15-36) | 21.5 (10-62) | NS | 25.3 (10-42) | 21.9 (10-62) | NS | 28 (10-62) | 21 (10-61) | NS |
| Platelets, ×109/L, median (range) | 107.5 (39-207) | 111.5 (6-982) | NS | 70 (6-149) | 127 (7-982) | .09 | 86 (13-167) | 125 (6-982) | NS |
| Hemoglobin, g/dL, median (range) | 10.6 (6-13) | 11 (5-8.5) | NS | 8.6 (5-12) | 11.3 (6-8.5) | .002 | 8.3 (5-11) | 11.4 (6-8.5) | <.001 |
| Blasts BM, % median (range) | 12 (6-19) | 4 (0-19) | .002 | 15 (2-19) | 4 (0-19) | .003 | 14 (1-19) | 4 (0-19) | <.001 |
| Cytogenetic alterations | 2 (28.6) | 20 (21.1) | NS | 2 (22.2) | 21 (19.6) | NS | 1 (10) | 22 (20.8) | NS |
| Cytogenetic risk | NS | NS | NS | ||||||
| Low | 5 (83.3) | 77 (81) | 7 (77.8) | 88 (83) | 9 (90) | 86 (81.9) | |||
| Intermediate | 1 (16.7) | 8 (8.5) | 2 (22.2) | 7 (6.6) | 1 (10) | 8 (7.6) | |||
| High | 0 | 10 (10.5) | 0 | 11 (10.4) | 0 | 11 (10.5) | |||
| Therapy-related | 0 | 4 (4) | NS | 0 | 5 (4.6) | NS | 0 | 5 (4.7) | NS |
| ICC/WHO 2022 classification | NS | .006 | .008 | ||||||
| MD-CMML | 4 (57.1) | 69 (65.7) | 2 (22.2) | 82 (70.1) | 3 (27.3) | 80 (69.6) | |||
| MP-CMML | 3 (42.9) | 36 (34.3) | 7 (77.8) | 35 (29.9) | 8 (72.7) | 35 (30.4) | |||
| ICC/WHO 2022 classifications | .001 | .002 | <.001 | ||||||
| CMML-1 | 1 (14.3) | 81 (77.9) | 2 (22.2) | 89 (76.7) | 1 (9.1) | 89 (78.1) | |||
| CMML-2 | 6 (85.7) | 23 (22.1) | 7 (77.8) | 27 (23.3) | 10 (90.9) | 25 (21.9) | |||
| CPSS | NS | .03 | <.001 | ||||||
| Low | 1 (14.3) | 33 (34.7) | 0 | 41 (38.3) | 0 | 41 (38.7) | |||
| Intermediate-1 | 1 (14.3) | 28 (29.5) | 2 (22.2) | 32 (29.9) | 1 (10) | 32 (30.2) | |||
| Intermediate-2 | 4 (57.1) | 28 (29.5) | 6 (66.7) | 27 (25.3) | 9 (90) | 25 (23.6) | |||
| High | 1 (14.3) | 6 (6.3) | 1 (11.1) | 7 (6.5) | 0 | 8 (7.5) | |||
| Dichotomized CPSS | .1 | .009 | <.001 | ||||||
| Low + intermediate-1 | 2 (28.6) | 61 (64.2) | 2 (22.2) | 73 (68.2) | 1 (10) | 73 (68.9) | |||
| Intermediate-2 + high | 5 (71.4) | 34 (35.8) | 7 (77.8) | 34 (31.8) | 9 (90) | 33 (31.1) | |||
| CPSS-Mol | .06 | .01 | .003 | ||||||
| Low | 0 | 20 (23.3) | 0 | 23 (23.5) | 0 | 23 (23.7) | |||
| Intermediate-1 | 1 (16.7) | 23 (26.7) | 0 | 27 (27.6) | 1 (12.5) | 26 (26.8) | |||
| Intermediate-2 | 1 (16.7) | 26 (30.2) | 6 (85.7) | 26 (26.5) | 7 (87.5) | 25 (25.8) | |||
| High | 4 (66.6) | 17 (19.8) | 1 (14.3) | 22 (22.4) | 0 | 23 (23.7) | |||
| Dichotomized CPSS-Mol | NS | .013 | .06 | ||||||
| Low + intermediate-1 | 1 (16.7) | 43 (50) | 0 | 50 (51) | 1 (12.5) | 49 (50.5) | |||
| Intermediate-2 + high | 5 (83.3) | 43 (50) | 7 (100) | 48 (49) | 7 (87.5) | 48 (49.5) | |||
| Transfusion dependence | 3 (42.9) | 29 (27.6) | NS | 8 (88.9) | 28 (4.3) | <.001 | 9 (81.8) | 27 (23.9) | <.001 |
| Patients receiving modifying treatment | 6 (85.7) | 44 (41.9) | .04 | 7 (77.8) | 46 (39.3) | .04 | 9 (81.8) | 43 (37.4) | .008 |
| Patients receiving HMA | 5 (71.4) | 36 (34.3) | .098 | 5 (55.6) | 39 (33.3) | .3 | 5 (45.5) | 38 (33) | .5 |
| Patients receiving chemotherapy | 4 (57.1) | 14 (13.3) | .01 | 6 (66.7) | 12 (10.3) | <.001 | 7 (63.6) | 11 (9.6) | <.001 |
| HMA response | NS | .3 | NS | ||||||
| Complete response | 3 (60) | 10 (30.3) | 0 | 13 (38.2) | 2 (40) | 11 (33.3) | |||
| Marrow response | 0 | 1 (3) | 0 | 1 (2.9) | 0 | 1 (3) | |||
| Partial remission | 1 (20) | 3 (9.1) | 0 | 4 (11.8) | 0 | 4 (12.1) | |||
| Clinical benefit | 0 | 3 (9.1) | 1 (25) | 2 (5.9) | 0 | 3 (9.2) | |||
| No response | 1 (20) | 16 (48.5) | 3 (75) | 14 (41.2) | 3 (60) | 14 (42.4) | |||
| Chemotherapy response | NS | NS | .068 | ||||||
| Complete response | 3 (75) | 9 (64.3) | 5 (83.3) | 7 (58.3) | 6 (85.7) | 6 (54.5) | |||
| Partial response | 0 | 1 (7.1) | 0 | 1 (8.4) | 1 (14.3) | 0 | |||
| No response | 1 (25) | 4 (28.6) | 1 (16.7) | 4 (33.3) | 0 | 5 (45.5) | |||
| Time to treatment, median (range) | 1.3 (0.3-4.3) | 3 (0.07-111.7) | .1 | 0.9 (0.3-31) | 2.8 (0.07-111.7) | .1 | 0.8 (0.07-5.8) | 3 (0.1-111.7) | .01 |
| Allogeneic transplant | 5 (71.4) | 17 (16.2) | .003 | 6 (66.7) | 18 (15.4) | .002 | 6 (54.5) | 17 (14.8) | .005 |
| OS, median (95% CI)∗ | 10 (3.6-16.4) | 29.8 (18.4-41.3) | .002 | 7.6 (5.3-9.9) | 31 (18.7-43.3) | <.001 | 9 (6.2-11.7) | 35.2 (22.4-48) | <.001 |
| OS, median (95% CI) | 22.2 (3.2-41) | 36.6 (15.3-58) | .056 | 27.8 (13.7-41.8) | 38.2 (18.7-57.7) | .2 | 25.1 (4.8-45.5) | 36.6 (17.3-55.9) | .1 |
| CIP to AML at 2 y, % (95% CI) | 28.6 (2.5-65.4) | 18.1 (11.2-26.3) | .6 | 50 (12.5-79.4) | 14.5 (8.7-21.8) | <.001 | 36.4 (10.1-64) | 15 (8.9-22.4) | .01 |
| Characteristics . | mutCEBPA (n = 7), n (%) . | wtCEBPA (n = 105), n (%) . | P value . | mutFLT3 (n = 9), n (%) . | wtFLT3 (n = 117), n (%) . | P value . | mutNPM1 (n = 11), n (%) . | wtNPM1 (n = 115), n (%) . | P value . |
|---|---|---|---|---|---|---|---|---|---|
| Age, y, median (range) | 64 (50-76) | 71 (28-95) | .08 | 63 (55-75) | 71 (28-95) | .04 | 60 (47-86) | 71 (28-95) | .1 |
| Sex (men/women) | 7/0 (100/0) | 79/26 (75.2/24.8) | NS | 7/2 (77.8/22.2) | 88/29 (75.2/24.8) | NS | 8/3 (72.7/27.3) | 87/28 (75.7/24.3) | NS |
| Leukocytes, ×109/L, median (range) | 8.9 (4-78) | 8.3 (2-94) | NS | 22 (6-56) | 7.7 (2-94) | .004 | 20.4 (10-31) | 7.6 (2-94) | .002 |
| Neutrophils, median (range) | 2.6 (0.4-41) | 4 (0.4-40.3) | NS | 7.2 (2-20) | 3.5 (0.4-41) | .1 | 5.2 (2.2-20) | 3.5 (0.4-41) | .05 |
| Monocytes, median (range) | 1.4 (1.1-29) | 1.8 (0.5-33.7) | NS | 3 (1.5-24) | 1.6 (0.5-34) | .01 | 5 (1.5-12.2) | 1.6 (0.5-33.7) | .002 |
| % monocytes, median (range) | 22.5 (15-36) | 21.5 (10-62) | NS | 25.3 (10-42) | 21.9 (10-62) | NS | 28 (10-62) | 21 (10-61) | NS |
| Platelets, ×109/L, median (range) | 107.5 (39-207) | 111.5 (6-982) | NS | 70 (6-149) | 127 (7-982) | .09 | 86 (13-167) | 125 (6-982) | NS |
| Hemoglobin, g/dL, median (range) | 10.6 (6-13) | 11 (5-8.5) | NS | 8.6 (5-12) | 11.3 (6-8.5) | .002 | 8.3 (5-11) | 11.4 (6-8.5) | <.001 |
| Blasts BM, % median (range) | 12 (6-19) | 4 (0-19) | .002 | 15 (2-19) | 4 (0-19) | .003 | 14 (1-19) | 4 (0-19) | <.001 |
| Cytogenetic alterations | 2 (28.6) | 20 (21.1) | NS | 2 (22.2) | 21 (19.6) | NS | 1 (10) | 22 (20.8) | NS |
| Cytogenetic risk | NS | NS | NS | ||||||
| Low | 5 (83.3) | 77 (81) | 7 (77.8) | 88 (83) | 9 (90) | 86 (81.9) | |||
| Intermediate | 1 (16.7) | 8 (8.5) | 2 (22.2) | 7 (6.6) | 1 (10) | 8 (7.6) | |||
| High | 0 | 10 (10.5) | 0 | 11 (10.4) | 0 | 11 (10.5) | |||
| Therapy-related | 0 | 4 (4) | NS | 0 | 5 (4.6) | NS | 0 | 5 (4.7) | NS |
| ICC/WHO 2022 classification | NS | .006 | .008 | ||||||
| MD-CMML | 4 (57.1) | 69 (65.7) | 2 (22.2) | 82 (70.1) | 3 (27.3) | 80 (69.6) | |||
| MP-CMML | 3 (42.9) | 36 (34.3) | 7 (77.8) | 35 (29.9) | 8 (72.7) | 35 (30.4) | |||
| ICC/WHO 2022 classifications | .001 | .002 | <.001 | ||||||
| CMML-1 | 1 (14.3) | 81 (77.9) | 2 (22.2) | 89 (76.7) | 1 (9.1) | 89 (78.1) | |||
| CMML-2 | 6 (85.7) | 23 (22.1) | 7 (77.8) | 27 (23.3) | 10 (90.9) | 25 (21.9) | |||
| CPSS | NS | .03 | <.001 | ||||||
| Low | 1 (14.3) | 33 (34.7) | 0 | 41 (38.3) | 0 | 41 (38.7) | |||
| Intermediate-1 | 1 (14.3) | 28 (29.5) | 2 (22.2) | 32 (29.9) | 1 (10) | 32 (30.2) | |||
| Intermediate-2 | 4 (57.1) | 28 (29.5) | 6 (66.7) | 27 (25.3) | 9 (90) | 25 (23.6) | |||
| High | 1 (14.3) | 6 (6.3) | 1 (11.1) | 7 (6.5) | 0 | 8 (7.5) | |||
| Dichotomized CPSS | .1 | .009 | <.001 | ||||||
| Low + intermediate-1 | 2 (28.6) | 61 (64.2) | 2 (22.2) | 73 (68.2) | 1 (10) | 73 (68.9) | |||
| Intermediate-2 + high | 5 (71.4) | 34 (35.8) | 7 (77.8) | 34 (31.8) | 9 (90) | 33 (31.1) | |||
| CPSS-Mol | .06 | .01 | .003 | ||||||
| Low | 0 | 20 (23.3) | 0 | 23 (23.5) | 0 | 23 (23.7) | |||
| Intermediate-1 | 1 (16.7) | 23 (26.7) | 0 | 27 (27.6) | 1 (12.5) | 26 (26.8) | |||
| Intermediate-2 | 1 (16.7) | 26 (30.2) | 6 (85.7) | 26 (26.5) | 7 (87.5) | 25 (25.8) | |||
| High | 4 (66.6) | 17 (19.8) | 1 (14.3) | 22 (22.4) | 0 | 23 (23.7) | |||
| Dichotomized CPSS-Mol | NS | .013 | .06 | ||||||
| Low + intermediate-1 | 1 (16.7) | 43 (50) | 0 | 50 (51) | 1 (12.5) | 49 (50.5) | |||
| Intermediate-2 + high | 5 (83.3) | 43 (50) | 7 (100) | 48 (49) | 7 (87.5) | 48 (49.5) | |||
| Transfusion dependence | 3 (42.9) | 29 (27.6) | NS | 8 (88.9) | 28 (4.3) | <.001 | 9 (81.8) | 27 (23.9) | <.001 |
| Patients receiving modifying treatment | 6 (85.7) | 44 (41.9) | .04 | 7 (77.8) | 46 (39.3) | .04 | 9 (81.8) | 43 (37.4) | .008 |
| Patients receiving HMA | 5 (71.4) | 36 (34.3) | .098 | 5 (55.6) | 39 (33.3) | .3 | 5 (45.5) | 38 (33) | .5 |
| Patients receiving chemotherapy | 4 (57.1) | 14 (13.3) | .01 | 6 (66.7) | 12 (10.3) | <.001 | 7 (63.6) | 11 (9.6) | <.001 |
| HMA response | NS | .3 | NS | ||||||
| Complete response | 3 (60) | 10 (30.3) | 0 | 13 (38.2) | 2 (40) | 11 (33.3) | |||
| Marrow response | 0 | 1 (3) | 0 | 1 (2.9) | 0 | 1 (3) | |||
| Partial remission | 1 (20) | 3 (9.1) | 0 | 4 (11.8) | 0 | 4 (12.1) | |||
| Clinical benefit | 0 | 3 (9.1) | 1 (25) | 2 (5.9) | 0 | 3 (9.2) | |||
| No response | 1 (20) | 16 (48.5) | 3 (75) | 14 (41.2) | 3 (60) | 14 (42.4) | |||
| Chemotherapy response | NS | NS | .068 | ||||||
| Complete response | 3 (75) | 9 (64.3) | 5 (83.3) | 7 (58.3) | 6 (85.7) | 6 (54.5) | |||
| Partial response | 0 | 1 (7.1) | 0 | 1 (8.4) | 1 (14.3) | 0 | |||
| No response | 1 (25) | 4 (28.6) | 1 (16.7) | 4 (33.3) | 0 | 5 (45.5) | |||
| Time to treatment, median (range) | 1.3 (0.3-4.3) | 3 (0.07-111.7) | .1 | 0.9 (0.3-31) | 2.8 (0.07-111.7) | .1 | 0.8 (0.07-5.8) | 3 (0.1-111.7) | .01 |
| Allogeneic transplant | 5 (71.4) | 17 (16.2) | .003 | 6 (66.7) | 18 (15.4) | .002 | 6 (54.5) | 17 (14.8) | .005 |
| OS, median (95% CI)∗ | 10 (3.6-16.4) | 29.8 (18.4-41.3) | .002 | 7.6 (5.3-9.9) | 31 (18.7-43.3) | <.001 | 9 (6.2-11.7) | 35.2 (22.4-48) | <.001 |
| OS, median (95% CI) | 22.2 (3.2-41) | 36.6 (15.3-58) | .056 | 27.8 (13.7-41.8) | 38.2 (18.7-57.7) | .2 | 25.1 (4.8-45.5) | 36.6 (17.3-55.9) | .1 |
| CIP to AML at 2 y, % (95% CI) | 28.6 (2.5-65.4) | 18.1 (11.2-26.3) | .6 | 50 (12.5-79.4) | 14.5 (8.7-21.8) | <.001 | 36.4 (10.1-64) | 15 (8.9-22.4) | .01 |
HMA, hypomethylating agents; ICC, International Consensus Classification; MD, myelodysplastic; BM, bone marrow; mut, mutated; NS, Not Significant; WHO, World Health Organization; wt, wild-type.
Patients who received alloSCT were censored at the time of alloHSCT.