Table 1 Baseline characteristics of NPM1-mut adult AML patients included in the HARMONY database, comparing the training cohort to the internal validation cohort.

Female sex

543 (54.2%)

419 (55%)

0.7938

Median age in years (range)

52.9 (18–81)

57 (18 - 86)

<0.0001

 Age ≥ 60 years

269 (26.8%)

320 (42%)

<0.0001

AML type

 De novo AML

963 (96.2%)

573 (90.5%)

<0.0001

 Secondary AML

38 (3.8%)

60 (9.5%)

  Prior HM

15 (1.5%)

48 (7.6%)

<0.0001

  Therapy-related AML

23 (2.3%)

12 (1.6%)

0.2811

Hemoglobin (g/dL)

8.9 [Q1 = 7.6, Q3 = 10.3]

9.2 [Q1 = 8.2, Q3 = 10.5]

0.0037

WBC (x10^9/L)

23.8 [Q1 = 6.9, Q3 = 62.8]

36.8 [Q1 = 13.4, Q3 = 85.6]

<0.0001

 WBC > 100 ×10^9/L

136 (13.6%)

144 (20.4%)

Platelets (x10^9/L)

66.5 [Q1 = 38, Q3 = 116]

66.5[Q1 = 38.2, Q3 = 111]

0.8798

Bone marrow % of blasts

75 [Q1 = 45, Q3 = 89]

72.75 [Q1 = 52, Q3 = 87]

0.9417

ELN 2022

 Favorable

601 (60%)

405 (53.2%)

0.0185

 Intermediate

391 (39.1%)

346 (45.4%)

 Adverse

9 (0.9%)

11 (1.4%)

FLT3-ITD

393 (39.3%)

349 (45.8%)

0.0068

Treatment response

 CRc

874 (87.3%)

606 (79.5%)

<0.0001

 Refractory

89 (8.9%)

95 (12.5%)

 Not evaluable (early death)

38 (3.8%)

61 (8%)

Early death

 30-day mortality

38 (3.8%)

61 (8%)

0.0002

 60-day mortality

62 (6.2%)

88 (11.5%)

<0.0001

Allogeneic HSCT

341 (34.1%)

215 (29.3%)

0.0419

 In CR1

242 (24.2%)

130 (17.1%)

0.0104

 In other situations

99 (9.9%)

85 (11.1%)

Median survival in years (95% CI)

8.25 (5.14–9.77)

2.84 (2.06–4.09)

<0.0001