Clinical and molecular characteristics of PTPN11-mut and PTPN11-wt patients
| Parameter . | PTPN11-wt . | PTPN11-mut . | P . |
|---|---|---|---|
| No of patients (n) | 1423 | 106 | |
| Age (y), median (IQR) | 55 (44-64.5) | 54 (42-63) | 0.377 |
| Sex, n (%) | .857 | ||
| Female | 678 (47.6) | 52 (49.1) | |
| Male | 745 (52.4) | 54 (50.9) | |
| Disease status, n (%) | .313 | ||
| De novo | 1198 (85.1) | 89 (85.6) | |
| tAML | 159 (11.3) | 14 (13.5) | |
| sAML | 50 (3.6) | 1 (1) | |
| ELN risk 2017, n (%) | <.001 | ||
| Favorable | 511 (39.1) | 59 (57.8) | |
| Intermediate | 543 (41.5) | 23 (22.5) | |
| Adverse | 253 (19.4) | 20 (19.6) | |
| Complex aberrant karyotype, n (%) | .154 | ||
| No | 1139 (87.5) | 92 (92.9) | |
| Yes | 162 (12.5) | 7 (7.1) | |
| FLT3-ITD, n (%) | .200 | ||
| No | 1089 (77.1) | 88 (83) | |
| Yes | 323 (22.9) | 18 (17) | |
| NPM1 mutation, n (%) | <.001 | ||
| No | 984 (70.3) | 38 (36.5) | |
| Yes | 416 (29.7) | 66 (63.5) | |
| ECOG score, n (%) | .064 | ||
| 0-1 | 842 (71.7) | 53 (61.6) | |
| 2-4 | 333 (28.3) | 33 (38.4) | |
| Laboratory, median (IQR) | |||
| WBC (Gpt/L) | 18.8 (4.4-54.37) | 26.76 (13.5-68.17) | .007 |
| Hemoglobin (mmol/L) | 5.9 (5.03-7.01) | 5.77 (5.03-6.77) | .408 |
| Platelets (Gpt/L) | 50 (27-94) | 54 (32-83) | .585 |
| Peripheral blasts (%) | 42 (12-75) | 38 (13.5-64) | .477 |
| LDH (U/L) | 448 (280-786) | 473.5 (374.25-853.75) | .025 |
| BM blasts (%) | 63 (44.38-79) | 62.75 (45.5-78) | .708 |
| Clinical outcome | |||
| CR rate, n (%) | 1038 (72.9) | 71 (67) | .008* |
| ED30, n (%) | 80 (5.6) | 12 (11.3) | .001* |
| EFS (mo), median (95% CI) | 7.42 (6.7-8.3) | 7.53 (4.41-10.91) | .022* |
| RFS (mo), median (95% CI) | 18.41 (15.87-23.34) | 14.66 (8.02-28.37) | .013* |
| OS (mo), median (95% CI) | 19.23 (17.16-21.76) | 13.44 (10.13-20.25) | <.001* |
| Parameter . | PTPN11-wt . | PTPN11-mut . | P . |
|---|---|---|---|
| No of patients (n) | 1423 | 106 | |
| Age (y), median (IQR) | 55 (44-64.5) | 54 (42-63) | 0.377 |
| Sex, n (%) | .857 | ||
| Female | 678 (47.6) | 52 (49.1) | |
| Male | 745 (52.4) | 54 (50.9) | |
| Disease status, n (%) | .313 | ||
| De novo | 1198 (85.1) | 89 (85.6) | |
| tAML | 159 (11.3) | 14 (13.5) | |
| sAML | 50 (3.6) | 1 (1) | |
| ELN risk 2017, n (%) | <.001 | ||
| Favorable | 511 (39.1) | 59 (57.8) | |
| Intermediate | 543 (41.5) | 23 (22.5) | |
| Adverse | 253 (19.4) | 20 (19.6) | |
| Complex aberrant karyotype, n (%) | .154 | ||
| No | 1139 (87.5) | 92 (92.9) | |
| Yes | 162 (12.5) | 7 (7.1) | |
| FLT3-ITD, n (%) | .200 | ||
| No | 1089 (77.1) | 88 (83) | |
| Yes | 323 (22.9) | 18 (17) | |
| NPM1 mutation, n (%) | <.001 | ||
| No | 984 (70.3) | 38 (36.5) | |
| Yes | 416 (29.7) | 66 (63.5) | |
| ECOG score, n (%) | .064 | ||
| 0-1 | 842 (71.7) | 53 (61.6) | |
| 2-4 | 333 (28.3) | 33 (38.4) | |
| Laboratory, median (IQR) | |||
| WBC (Gpt/L) | 18.8 (4.4-54.37) | 26.76 (13.5-68.17) | .007 |
| Hemoglobin (mmol/L) | 5.9 (5.03-7.01) | 5.77 (5.03-6.77) | .408 |
| Platelets (Gpt/L) | 50 (27-94) | 54 (32-83) | .585 |
| Peripheral blasts (%) | 42 (12-75) | 38 (13.5-64) | .477 |
| LDH (U/L) | 448 (280-786) | 473.5 (374.25-853.75) | .025 |
| BM blasts (%) | 63 (44.38-79) | 62.75 (45.5-78) | .708 |
| Clinical outcome | |||
| CR rate, n (%) | 1038 (72.9) | 71 (67) | .008* |
| ED30, n (%) | 80 (5.6) | 12 (11.3) | .001* |
| EFS (mo), median (95% CI) | 7.42 (6.7-8.3) | 7.53 (4.41-10.91) | .022* |
| RFS (mo), median (95% CI) | 18.41 (15.87-23.34) | 14.66 (8.02-28.37) | .013* |
| OS (mo), median (95% CI) | 19.23 (17.16-21.76) | 13.44 (10.13-20.25) | <.001* |
BM, bone marrow; CI, confidence interval; CR, complete remission; ED30, early death within 30 days; EFS, event-free survival; ELN, European LeukemiaNET; IQR, interquartile range; LDH, lactate dehydrogenase; OS, overall survival; PB, peripheral blood; RFS, relapse-free survival; sAML, secondary acute myeloid leukemia; tAML, therapy-related acute myeloid leukemia; WBC, white blood cells.
Results of the multivariable regression model.