Patient characteristics and outcomes according to mutational status of CEBPA, FLT3, and/or NPM1 (mutCFN vs wtCFN)
| Characteristics . | mutCFN (n = 21), n (%) . | wtCFN (n = 93), n (%) . | P value . |
|---|---|---|---|
| Age, y, median (range) | 63 (47-86) | 71 (28-95) | .03 |
| Sex (men/women) | 17/4 (81/19) | 71/22 (76/24) | NS |
| Leukocytes, ×109/L, median (range) | 19.6 (4-78) | 7.6 (2-94) | .003 |
| Neutrophils, median (range) | 5 (0.4-41) | 3.8 (0.4-40) | .1 |
| Monocytes, median (range) | 2.9 (1.1-29) | 1.7 (0.5-33.7) | .006 |
| % monocytes, median (range) | 28 (10-62) | 19 (10-61) | .2 |
| Platelets, ×109/L, median (range) | 98 (6-207) | 117 (7-982) | .04 |
| Hemoglobin, g/dL, median (range) | 8.6 (5-13) | 11.4 (7-85) | <.001 |
| Blasts BM, % median (range) | 12 (1-19) | 4 (0-18) | <.001 |
| Cytogenetic risk | NS | ||
| Low | 16 (84.2) | 68 (81) | |
| Intermediate | 3 (15.8) | 6 (7.1) | |
| High | 0 | 10 (11.9) | |
| ICC/WHO 2022 classification | .01 | ||
| MD-CMML | 8 (38.1) | 66 (71) | |
| MP-CMML | 13 (61.9) | 27 (29) | |
| ICC/WHO 2022 classifications | <.001 | ||
| CMML-1 | 4 (19) | 19 (85.9) | |
| CMML-2 | 17 (81) | 13 (14.1) | |
| CPSS | .001 | ||
| Low | 1 (5) | 33 (39.2) | |
| Intermediate-1 | 4 (20) | 26 (31) | |
| Intermediate-2 | 13 (65) | 20 (23.8) | |
| High | 2 (10) | 5 (6) | |
| CPSS-Mol | .005 | ||
| Low | 0 | 20 (26.7) | |
| Intermediate-1 | 2 (11.8) | 22 (29.3) | |
| Intermediate-2 | 10 (58.8) | 17 (22.7) | |
| High | 5 (29.4) | 16 (21.3) | |
| Transfusion dependence | 14 (66.7) | 20 (21.5) | <.001 |
| Patients receiving modifying treatment | 17 (81) | 34 (36.6) | <.001 |
| Patients receiving HMA | 13 (61.9) | 29 (31.2) | .01 |
| Patients receiving chemotherapy | 12 (57.1) | 6 (6.5) | <.001 |
| Chemotherapy response | |||
| Complete response | 9 (75) | 3 (50) | NS |
| Partial response | 1 (8.3) | 0 | NA |
| No response | 2 (16.7) | 3 (50) | NS |
| Overall response rate | 10 (83.3) | 3 (50) | NS |
| Time to treatment, median (range) | 0.9 (0.07-30.5) | 4.5 (0.1-111.7) | .002 |
| Allogeneic transplant | 12 (57.1) | 11 (11.8) | <.001 |
| OS, median (95% CI)∗ | 9.1 (6.3-12) | 36 (20-52.4) | <.001 |
| OS, median (95% CI) | 22.2 (8.2-36.1) | 46.8 (26-67.6) | .01 |
| CIP to AML at 2 y, % (95% CI) | 39.8 (18.3-60.7) | 13.7 (7.5-21.8) | <.001 |
| Characteristics . | mutCFN (n = 21), n (%) . | wtCFN (n = 93), n (%) . | P value . |
|---|---|---|---|
| Age, y, median (range) | 63 (47-86) | 71 (28-95) | .03 |
| Sex (men/women) | 17/4 (81/19) | 71/22 (76/24) | NS |
| Leukocytes, ×109/L, median (range) | 19.6 (4-78) | 7.6 (2-94) | .003 |
| Neutrophils, median (range) | 5 (0.4-41) | 3.8 (0.4-40) | .1 |
| Monocytes, median (range) | 2.9 (1.1-29) | 1.7 (0.5-33.7) | .006 |
| % monocytes, median (range) | 28 (10-62) | 19 (10-61) | .2 |
| Platelets, ×109/L, median (range) | 98 (6-207) | 117 (7-982) | .04 |
| Hemoglobin, g/dL, median (range) | 8.6 (5-13) | 11.4 (7-85) | <.001 |
| Blasts BM, % median (range) | 12 (1-19) | 4 (0-18) | <.001 |
| Cytogenetic risk | NS | ||
| Low | 16 (84.2) | 68 (81) | |
| Intermediate | 3 (15.8) | 6 (7.1) | |
| High | 0 | 10 (11.9) | |
| ICC/WHO 2022 classification | .01 | ||
| MD-CMML | 8 (38.1) | 66 (71) | |
| MP-CMML | 13 (61.9) | 27 (29) | |
| ICC/WHO 2022 classifications | <.001 | ||
| CMML-1 | 4 (19) | 19 (85.9) | |
| CMML-2 | 17 (81) | 13 (14.1) | |
| CPSS | .001 | ||
| Low | 1 (5) | 33 (39.2) | |
| Intermediate-1 | 4 (20) | 26 (31) | |
| Intermediate-2 | 13 (65) | 20 (23.8) | |
| High | 2 (10) | 5 (6) | |
| CPSS-Mol | .005 | ||
| Low | 0 | 20 (26.7) | |
| Intermediate-1 | 2 (11.8) | 22 (29.3) | |
| Intermediate-2 | 10 (58.8) | 17 (22.7) | |
| High | 5 (29.4) | 16 (21.3) | |
| Transfusion dependence | 14 (66.7) | 20 (21.5) | <.001 |
| Patients receiving modifying treatment | 17 (81) | 34 (36.6) | <.001 |
| Patients receiving HMA | 13 (61.9) | 29 (31.2) | .01 |
| Patients receiving chemotherapy | 12 (57.1) | 6 (6.5) | <.001 |
| Chemotherapy response | |||
| Complete response | 9 (75) | 3 (50) | NS |
| Partial response | 1 (8.3) | 0 | NA |
| No response | 2 (16.7) | 3 (50) | NS |
| Overall response rate | 10 (83.3) | 3 (50) | NS |
| Time to treatment, median (range) | 0.9 (0.07-30.5) | 4.5 (0.1-111.7) | .002 |
| Allogeneic transplant | 12 (57.1) | 11 (11.8) | <.001 |
| OS, median (95% CI)∗ | 9.1 (6.3-12) | 36 (20-52.4) | <.001 |
| OS, median (95% CI) | 22.2 (8.2-36.1) | 46.8 (26-67.6) | .01 |
| CIP to AML at 2 y, % (95% CI) | 39.8 (18.3-60.7) | 13.7 (7.5-21.8) | <.001 |
HMA, hypomethylating agents; ICC, International Consensus Classification; MD, myelodysplastic; BM, bone marrow; mut, mutated; NA, Not Achieved; NS, Not Significant; WHO, World Health Organization; wt, wild-type.
Patients who received alloHSCT were censored at the time of alloSCT.