Patient characteristics
| . | All patients (N = 655) . | Relapsed patients (n = 365) . | Refractory patients (n = 290) . | P . | |||
|---|---|---|---|---|---|---|---|
| Median or n . | Range or % . | Median or n . | Range or % . | Median or n . | Range or % . | ||
| Age, y (n = 636) | 65 | 16-92 | 65 | 16-89 | 64 | 19-92 | .8197 |
| Sex (N = 655) | .5432 | ||||||
| Male | 381 | 58.2 | 208 | 57 | 173 | 59.7 | |
| Female | 274 | 41.8 | 157 | 43 | 117 | 40.3 | |
| AML type (n = 650) | .0065 | ||||||
| De novo | 458 | 70.5 | 272 | 74.9 | 186 | 64.8 | |
| Secondary | 192 | 29.5 | 91 | 25.1 | 101 | 35.2 | |
| WHO type at diagnosis (n = 651) | |||||||
| AML with recurrent genetic abnormalities | 38 | 5.8 | 31 | 8.5 | 7 | 2.4 | .0020 |
| AML with myelodysplasia-related features | 175 | 26.9 | 86 | 23.6 | 89 | 31.1 | .0385 |
| Therapy-related AML | 52 | 8 | 31 | 8.5 | 21 | 7.3 | .6952 |
| AML, not otherwise specified | 386 | 59.3 | 217 | 59.5 | 169 | 59.1 | .9899 |
| CBC prior to initiation of HMA | |||||||
| WBC (n = 455) | 3.2 | 0.1-110.5 | 3.2 | 0.1-110.5 | 3.3 | 0.1-79.8 | .2843 |
| ANC (n = 415) | 0.9 | 0-72 | 1 | 0-72 | 0.8 | 0-54 | .7160 |
| Platelets (n = 454) | 40 | 0.6-810 | 44 | 2-293 | 35 | 0.6-810 | .4455 |
| PB blast % (n = 392) | 8 | 0-98 | 8 | 0-96 | 8 | 0-98 | .4862 |
| BM prior to initiation of HMA | |||||||
| BM blast % (n = 305) | 27 | 2-100 | 27 | 2-100 | 26.5 | 2-95 | .5606 |
| BM cellularity % (n = 244) | 40 | 2-100 | 40 | 2-100 | 40 | 2-100 | .2496 |
| MRC cytogenetic risk group prior to initiation of HMA (n = 225) | .7794 | ||||||
| Good | 4 | 1.8 | 3 | 2.5 | 1 | 1 | |
| Intermediate | 131 | 58.2 | 70 | 57.9 | 61 | 58.7 | |
| Poor | 90 | 40 | 48 | 39.7 | 42 | 40.4 | |
| Chromosomal abnormalities | |||||||
| Complex (n = 224) | 54 | 24.1 | 29 | 24 | 25 | 24.3 | 1 |
| Monosomy (n = 234) | 37 | 15.8 | 20 | 15.6 | 17 | 16 | 1 |
| Chromosome 7 abn (n = 224) | 50 | 22.3 | 28 | 23.1 | 22 | 21.4 | .8744 |
| Chromosome 5 abn (n = 224) | 44 | 19.6 | 21 | 17.4 | 23 | 22.3 | .4441 |
| Mutational status (prior to start of HMA or at diagnosis) | |||||||
| TP53 (n = 93) | 7 | 7.5 | 5 | 7.7 | 2 | 7.1 | 1 |
| FLT3 (n = 269) | 46 | 17.1 | 34 | 20.9 | 12 | 11.3 | .0622 |
| NPM1 (n = 228) | 55 | 24.1 | 42 | 30.7 | 13 | 14.3 | .0076 |
| CEBPa (n = 124) | 8 | 6.5 | 7 | 8.3 | 1 | 2.5 | .4349 |
| DNMT3A (n = 39) | 5 | 12.8 | 3 | 11.1 | 2 | 16.7 | .6342 |
| TET2 (n = 37) | 2 | 5.4 | 2 | 7.4 | 0 | 0 | 1 |
| IDH1/2 (n = 39) | 5 | 12.8 | 4 | 13.8 | 1 | 10 | 1 |
| ASXL1 (n = 11) | 0 | 0 | 0 | 0 | 0 | 0 | 1 |
| SF3B1 (n = 7) | 0 | 0 | 0 | 0 | 0 | 0 | 1 |
| Number of therapy lines prior to HMA (n = 648) | 1 | 1-7 | 1 | 1-7 | 1 | 1-7 | .8463 |
| Duration of CR1 prior to initiation of HMA (n = 329) | 1.95 | 0-180 | 8 | 0.5-180 | 0 | 0-0 | <.0001 |
| AlloSCT prior to initiation of HMA (months) (n = 618) | 115 | 18.6 | 91 | 27 | 24 | 8.5 | <.0001 |
| HMA used (n = 634) | .1695 | ||||||
| Azacitidine | 360 | 56.8 | 192 | 54.2 | 168 | 60 | |
| Decitabine | 274 | 43.2 | 162 | 45.8 | 112 | 40 | |
| Number of cycles of HMA (n = 633) | 3 | 1-36 | 3 | 1-36 | 3 | 1-34 | .4922 |
| Azacitidine | 3 | 1-36 | 3 | 1-36 | 4 | 1-34 | .4684 |
| Decitabine | 2 | 1-35 | 2 | 1-35 | 2 | 1-22 | .8129 |
| HMA administration schedule (n = 587) | .3654 | ||||||
| Azacitidine (n = 336) | |||||||
| 7-0 | 257 | 76.5 | 131 | 73.2 | 126 | 80.3 | |
| 5-2-2 | 18 | 2.4 | 10 | 5.6 | 8 | 5.1 | |
| 5-0 | 60 | 17.9 | 38 | 21.2 | 22 | 14.0 | |
| 10-0 | 1 | 0.3 | 0 | 0 | 1 | 0.6 | |
| Decitabine (n = 251) | |||||||
| 7-0 | 3 | 1.2 | 1 | 0.7 | 2 | 1.8 | |
| 5-2-2 | 2 | 0.8 | 0 | 0 | 2 | 1.8 | |
| 5-0 | 181 | 72.1 | 104 | 73.8 | 77 | 70.0 | |
| 10-0 | 50 | 19.9 | 29 | 20.6 | 21 | 19.1 | |
| Others | 15 | 6.0 | 7 | 5.0 | 8 | 7.3 | |
| Agents used concurrently with HMA (n = 532) | .0585 | ||||||
| Gemtuzumab ozogamicin | 64 | 12 | 25 | 9 | 39 | 15.4 | |
| Valproic acid | 7 | 1.3 | 2 | 0.7 | 5 | 2 | |
| Valproic acid plus all-trans retinoic acid | 24 | 4.5 | 11 | 3.9 | 13 | 5.1 | |
| Hydroxyurea | 12 | 2.3 | 5 | 1.8 | 7 | 2.8 | |
| Cytarabine | 2 | 0.4 | 1 | 0.4 | 1 | 0.4 | |
| Anthracycline | 4 | 0.8 | 0 | 0 | 4 | 1.6 | |
| Sorafenib | 13 | 2.4 | 10 | 3.6 | 3 | 1.2 | |
| Hedgehog inhibitor | 4 | 0.8 | 1 | 0.4 | 3 | 1.2 | |
| IDH inhibitor | 1 | 0.2 | 0 | 0 | 1 | 0.4 | |
| Erythrocyte-stimulating agents | 7 | 1.3 | 5 | 1.8 | 2 | 0.8 | |
| Granulocyte colony-stimulating factor | 8 | 1.5 | 4 | 1.4 | 4 | 1.6 | |
| JAK | 1 | 0.2 | 1 | 0.4 | 0 | 0 | |
| Dead at end of study (n = 608) | 529 | 87 | 295 | 86.8 | 234 | 87.3 | .9376 |
| . | All patients (N = 655) . | Relapsed patients (n = 365) . | Refractory patients (n = 290) . | P . | |||
|---|---|---|---|---|---|---|---|
| Median or n . | Range or % . | Median or n . | Range or % . | Median or n . | Range or % . | ||
| Age, y (n = 636) | 65 | 16-92 | 65 | 16-89 | 64 | 19-92 | .8197 |
| Sex (N = 655) | .5432 | ||||||
| Male | 381 | 58.2 | 208 | 57 | 173 | 59.7 | |
| Female | 274 | 41.8 | 157 | 43 | 117 | 40.3 | |
| AML type (n = 650) | .0065 | ||||||
| De novo | 458 | 70.5 | 272 | 74.9 | 186 | 64.8 | |
| Secondary | 192 | 29.5 | 91 | 25.1 | 101 | 35.2 | |
| WHO type at diagnosis (n = 651) | |||||||
| AML with recurrent genetic abnormalities | 38 | 5.8 | 31 | 8.5 | 7 | 2.4 | .0020 |
| AML with myelodysplasia-related features | 175 | 26.9 | 86 | 23.6 | 89 | 31.1 | .0385 |
| Therapy-related AML | 52 | 8 | 31 | 8.5 | 21 | 7.3 | .6952 |
| AML, not otherwise specified | 386 | 59.3 | 217 | 59.5 | 169 | 59.1 | .9899 |
| CBC prior to initiation of HMA | |||||||
| WBC (n = 455) | 3.2 | 0.1-110.5 | 3.2 | 0.1-110.5 | 3.3 | 0.1-79.8 | .2843 |
| ANC (n = 415) | 0.9 | 0-72 | 1 | 0-72 | 0.8 | 0-54 | .7160 |
| Platelets (n = 454) | 40 | 0.6-810 | 44 | 2-293 | 35 | 0.6-810 | .4455 |
| PB blast % (n = 392) | 8 | 0-98 | 8 | 0-96 | 8 | 0-98 | .4862 |
| BM prior to initiation of HMA | |||||||
| BM blast % (n = 305) | 27 | 2-100 | 27 | 2-100 | 26.5 | 2-95 | .5606 |
| BM cellularity % (n = 244) | 40 | 2-100 | 40 | 2-100 | 40 | 2-100 | .2496 |
| MRC cytogenetic risk group prior to initiation of HMA (n = 225) | .7794 | ||||||
| Good | 4 | 1.8 | 3 | 2.5 | 1 | 1 | |
| Intermediate | 131 | 58.2 | 70 | 57.9 | 61 | 58.7 | |
| Poor | 90 | 40 | 48 | 39.7 | 42 | 40.4 | |
| Chromosomal abnormalities | |||||||
| Complex (n = 224) | 54 | 24.1 | 29 | 24 | 25 | 24.3 | 1 |
| Monosomy (n = 234) | 37 | 15.8 | 20 | 15.6 | 17 | 16 | 1 |
| Chromosome 7 abn (n = 224) | 50 | 22.3 | 28 | 23.1 | 22 | 21.4 | .8744 |
| Chromosome 5 abn (n = 224) | 44 | 19.6 | 21 | 17.4 | 23 | 22.3 | .4441 |
| Mutational status (prior to start of HMA or at diagnosis) | |||||||
| TP53 (n = 93) | 7 | 7.5 | 5 | 7.7 | 2 | 7.1 | 1 |
| FLT3 (n = 269) | 46 | 17.1 | 34 | 20.9 | 12 | 11.3 | .0622 |
| NPM1 (n = 228) | 55 | 24.1 | 42 | 30.7 | 13 | 14.3 | .0076 |
| CEBPa (n = 124) | 8 | 6.5 | 7 | 8.3 | 1 | 2.5 | .4349 |
| DNMT3A (n = 39) | 5 | 12.8 | 3 | 11.1 | 2 | 16.7 | .6342 |
| TET2 (n = 37) | 2 | 5.4 | 2 | 7.4 | 0 | 0 | 1 |
| IDH1/2 (n = 39) | 5 | 12.8 | 4 | 13.8 | 1 | 10 | 1 |
| ASXL1 (n = 11) | 0 | 0 | 0 | 0 | 0 | 0 | 1 |
| SF3B1 (n = 7) | 0 | 0 | 0 | 0 | 0 | 0 | 1 |
| Number of therapy lines prior to HMA (n = 648) | 1 | 1-7 | 1 | 1-7 | 1 | 1-7 | .8463 |
| Duration of CR1 prior to initiation of HMA (n = 329) | 1.95 | 0-180 | 8 | 0.5-180 | 0 | 0-0 | <.0001 |
| AlloSCT prior to initiation of HMA (months) (n = 618) | 115 | 18.6 | 91 | 27 | 24 | 8.5 | <.0001 |
| HMA used (n = 634) | .1695 | ||||||
| Azacitidine | 360 | 56.8 | 192 | 54.2 | 168 | 60 | |
| Decitabine | 274 | 43.2 | 162 | 45.8 | 112 | 40 | |
| Number of cycles of HMA (n = 633) | 3 | 1-36 | 3 | 1-36 | 3 | 1-34 | .4922 |
| Azacitidine | 3 | 1-36 | 3 | 1-36 | 4 | 1-34 | .4684 |
| Decitabine | 2 | 1-35 | 2 | 1-35 | 2 | 1-22 | .8129 |
| HMA administration schedule (n = 587) | .3654 | ||||||
| Azacitidine (n = 336) | |||||||
| 7-0 | 257 | 76.5 | 131 | 73.2 | 126 | 80.3 | |
| 5-2-2 | 18 | 2.4 | 10 | 5.6 | 8 | 5.1 | |
| 5-0 | 60 | 17.9 | 38 | 21.2 | 22 | 14.0 | |
| 10-0 | 1 | 0.3 | 0 | 0 | 1 | 0.6 | |
| Decitabine (n = 251) | |||||||
| 7-0 | 3 | 1.2 | 1 | 0.7 | 2 | 1.8 | |
| 5-2-2 | 2 | 0.8 | 0 | 0 | 2 | 1.8 | |
| 5-0 | 181 | 72.1 | 104 | 73.8 | 77 | 70.0 | |
| 10-0 | 50 | 19.9 | 29 | 20.6 | 21 | 19.1 | |
| Others | 15 | 6.0 | 7 | 5.0 | 8 | 7.3 | |
| Agents used concurrently with HMA (n = 532) | .0585 | ||||||
| Gemtuzumab ozogamicin | 64 | 12 | 25 | 9 | 39 | 15.4 | |
| Valproic acid | 7 | 1.3 | 2 | 0.7 | 5 | 2 | |
| Valproic acid plus all-trans retinoic acid | 24 | 4.5 | 11 | 3.9 | 13 | 5.1 | |
| Hydroxyurea | 12 | 2.3 | 5 | 1.8 | 7 | 2.8 | |
| Cytarabine | 2 | 0.4 | 1 | 0.4 | 1 | 0.4 | |
| Anthracycline | 4 | 0.8 | 0 | 0 | 4 | 1.6 | |
| Sorafenib | 13 | 2.4 | 10 | 3.6 | 3 | 1.2 | |
| Hedgehog inhibitor | 4 | 0.8 | 1 | 0.4 | 3 | 1.2 | |
| IDH inhibitor | 1 | 0.2 | 0 | 0 | 1 | 0.4 | |
| Erythrocyte-stimulating agents | 7 | 1.3 | 5 | 1.8 | 2 | 0.8 | |
| Granulocyte colony-stimulating factor | 8 | 1.5 | 4 | 1.4 | 4 | 1.6 | |
| JAK | 1 | 0.2 | 1 | 0.4 | 0 | 0 | |
| Dead at end of study (n = 608) | 529 | 87 | 295 | 86.8 | 234 | 87.3 | .9376 |
abn, abnormality; ANC, absolute neutrophil count; MRC, Modified British Medical Research Council.