Table 1:
Baseline characteristics
| Characteristic | All (CPX-351) | 50 units/m2 | 75 units/m2 | 100 units/m2 | P |
|---|---|---|---|---|---|
|
| |||||
| No. of patients | 56 | 16 | 24 | 16 | |
| Median age, years (range) | 69 (55–84) | 67 (57–81) | 68 (55–84) | 70 (63–80) | 0.41 |
| Female, no. (%) | 19 (34) | 7 (44) | 8 (33) | 4 (25) | 0.52 |
| Male, no (%) | 37 (66) | 9 (56) | 16 (67) | 12 (75) | |
| WBC, median × 109/L (range) | 2.5 (0.3–61) | 3.5 (1.3–61) | 2.5 (0.3–27) | 1.8 (0.3–56) | 0.15 |
| Hg, median g/L (range) | 8.8 (6.9–10.5) | 9.0 (7.4–9.8) | 9.0 (6.9–10.5) | 8.3 (7.0–10.1) | 0.09 |
| Platelets, median × 109/L (range) | 27 (2–129) | 18 (9–99) | 35 (2–129) | 21 (5–96) | 0.10 |
| Creatinine > 1.3 g/dL, no. (%) | 2 (4) | 1 (1) | 1 (4) | 0 (0) | 0.46 |
| ECOG performance status score, no. (%) | 0.005 | ||||
| 0–1 | 41 (73) | 9 (56) | 22 (92) | 10 (62) | |
| 2–3 | 15 (27) | 7 (44) | 2 (8) | 6 (38) | |
| s-AML/t-AML, no. (%) | 51 (91) | 16 (100) | 21 (88) | 14 (88) | 0.56 |
| AML MRC, no. (%) | 5 (9) | 0 | 3 (12) | 2 (12) | |
| Prior HMA exposure* | 46 (82) | 15 (94) | 18 (75) | 13 (81) | 0.38 |
| Cytogenetics, no. (%) | 0.45 | ||||
| Diploid | 6 (11) | 2 (13) | 3 (13) | 1 (7) | |
| Adverse^ | 30 (54) | 10 (62) | 12 (50) | 8 (50) | |
| Other | 14 (25) | 4 (25) | 6 (25) | 4 (25) | |
| IM or not done | 6 (10) | 0 | 3 (12) | 3 (18) | |
| Median no. of induction mortality factors (range)# | 2 (1–5) | 3 (1–5) | 2 (1–4) | 2 (1–4) | 0.04 |
| Mutations, no. (%) | |||||
| ASXL1 | 16 (29) | 5 (31) | 6 (25) | 5 (33) | 0.87 |
| TP53 | 14 (25) | 3 (19) | 6 (25) | 5 (33) | 0.69 |
| KRAS/NRAS | 13 (23) | 6 (38) | 4 (16) | 3 (19) | 0.32 |
| TET2 | 10 (18) | 5 (31) | 1 (4) | 4 (25) | 0.05 |
| IDH1/2 | 6 (11) | 1 (6) | 4 (16) | 1 (7) | 0.64 |
| RUNX1 | 6 (11) | 3 (19) | 2 (8) | 1 (1) | 0.55 |
| DNMT3A | 5 (9) | 2 (12) | 3 (12) | 0 (0) | 0.50 |
| EZH2 | 4 (7) | 2 (12) | 0 (0) | 2 (13) | 0.20 |
| JAK2 | 2 (4) | 1 (6) | 0 (0) | 1 (7) | 0.32 |
Abbreviations: WBC, white blood cell; Hg, hemoglobin; ECOG, Eastern Cooperative Oncology Group; s-AML/t AML, secondary or therapy-related acute myeloid leukemia; MDS RC, myelodysplastic syndrome-related changes; HMA, hypomethylating agent; IM, insufficient metaphases.
^
Adverse risk cytogenetics according to the European LeukemiaNet (ELN) classification.
*
All received HMA for prior MDS, CMML or MPN and all but one had no benefit or disease progression following therapy with a hypomethylating agent.
#
Factors used as eligibility criteria indicating a high risk of induction mortality.