Table 1.
Clinical characteristics of 60 patients with European LeukemiaNet-defined primary refractory acute myeloid leukemia (AML), stratified by the achievement of response after documented salvage therapy.
| Variables | All patients n = 60 (100%) |
Patients receiving documented salvage (n = 48) |
P-value A vs B |
||
|---|---|---|---|---|---|
| Patients with response (CR/CRi/MLFS) (Group A) n = 13 (27%) |
Response rate (%) | Patients without response (Group B) n = 35 (73%) |
|||
| Overall response category | |||||
| CR, n (%) | 7 (54) | 15% | |||
| CRi, n (%) | 2 (15) | 4% | |||
| MLFS, n (%) | 4 (31) | 8% | |||
| Age in years, median (range) | 63 (23–78) | 58 (27–72) | 63 (23–78) | 0.48 | |
| Age ≥ 60 years, n (%) | 38 (63) | 6 (46) | 21% | 22 (63) | 0.30 |
| Males, n (%) | 40 (67) | 9 (69) | 29% | 22 (67) | 0.87 |
| AML type | |||||
| De novo (primary), n (%) | 28 (48) | 9 (69) | 38% | 15 (43) | 0.10 |
| Secondary, n (%) | 31 (52) | 4 (31) | 17% | 20 (57) | |
| FLT3/NPM1 distributions: n = evaluable | n = 35 | n = 10 | n = 19 | 0.26 | |
| FLT3-ITD and NPM1 negative, n (%) | 23 (66) | 6 (60) | 33% | 12 (63) | |
| FLT3-ITD negative NPM1 positive, n (%) | 1 (3) | 0 (0) | 43% | 0 (0) | |
| FLT3-ITD and NPM1 positive, n (%) | 3 (9) | 0 (0) | 3 (16) | ||
| FLT3-ITD positive and NPM1 negative, n (%) | 7 (20) | 3 (30) | 4 (21) | ||
| FLT3-TKD positive and NPM1 negative, n (%) | 1 (3) | 1 (10) | 0 (0) | ||
| Positive molecular mutations on NGS: n = evaluable | n = 37 | n = 6 | n = 31 | ||
| ASXL1, n (%) | 1 (3) | 1 (17) | – | ||
| BCOR, n (%) | 1 (3) | 1 (3) | |||
| CEBPA, n (%)—silent | 1 (3) | 1 (3) | |||
| CSF3R, n (%) | 1 (3) | 1 (3) | |||
| DNMT3A, n (%) | 2 (6) | 2 (6) | |||
| GATA2, n (%) | 1 (3) | 1 (3) | |||
| KRAS, n (%) | 1 (3) | 1 (3) | |||
| NRAS, n (%) | 1 (3) | 1 (3) | |||
| RUNX1, n (%) | 1 (3) | – | |||
| SETBP1, n (%) | 1 (3) | 1 (3) | |||
| SF3B1, n (%) | 2 (6) | 1 (3) | |||
| TET2, n (%) | 1 (3) | 1 (3) | |||
| TP53, n (%) | 4 (11) | 4 (13) | |||
| U2AF1, n (%) | 1 (3) | – | |||
| WT1, n (%) | 1 (3) | – | |||
| IDH2, n (%) | 1 (3) | – | 1 (3) | ||
| ELN karyotype at diagnosis: n = evaluable | n = 59 | n = 13 | n = 34 | ||
| Intermediate, n (%) | 24 (51) | 8 (62) | 44% | 10 (29) | 0.04 |
| Unfavorable, n (%) | 35 (59) | 5 (38) | 17% | 24 (71) | |
| % BM blasts before salvage therapy: n = evaluable | n = 60 | n = 13 | n = 35 | ||
| Median (range) | 23 (0–99) | 25 (5–99) | N/A | 21 (0–90) | 0.66 |
| Documented number of salvage regimens to response or death: n = evaluable | n = 48 | n = 13 | 27% | n = 35 | 0.16 |
| One, n (%) | 26 (54) | 7 (54) | 35% | 19 (54) | |
| Two, n (%) | 17 (35) | 6 (46) | 11 (31) | ||
| Three or more, n (%) | 5 (10) | 0 (0) | 5 (14) | ||
| Salvage intensity: | 0.47 | ||||
| Intensive, n (%) | 30 (63) | 9 (69) | 30% | 21 (60) | |
| Less Intensive, n (%) | 13 (27) | 2 (15) | 15% | 11 (31) | |
| Targeted, n (%) | 5 (10) | 2 (15) | 40% | 3 (9) | |
| AHSCT, n (%) | 12 (20) | 8 (62) | N/A | 4 (12) | 0.001 |
| Relapse after AHSCT, n (%) |
n = 8 2 (25) |
N/A |
n = 4 3 (75) |
0.09 | |
ELN European LeukemiaNet, AML acute myelogenous leukemia, BM bone marrow, CR/CRi complete remission with (CR) or without (CRi) blood count recovery, MLFS morphologic leukemia-free state, AHSCT allogeneic hematopoietic stem cell transplant, Secondary prior chemotherapy or radiation therapy-related AML, AML arising from prior myeloproliferative neoplasm, myelodysplastic syndrome, or chronic myelomonocytic leukemia, FLT3 FMS-like tyrosine kinase 3, ITD internal tandem duplication, TKD tyrosine kinase domain, NPM1 Nucleophosmin 1 = included 1 patient with favorable cytogenetics, N/A not applicable.
Bold values indicate statistically significant values.