Table 1.
Patient demographic and baseline disease characteristics in the efficacy-evaluable population
| Parameter | Efficacy-evaluable population (n = 147) |
|---|---|
| Sex, n (%) | |
| Male/female | 74 (50)/73 (50) |
| Age (y) | |
| Median (range) | 71 (32-87) |
| ECOG PS, n (%) | |
| 0 | 45 (31) |
| 1 | 76 (52) |
| 2 | 23 (16) |
| AML type, n (%) | |
| Primary de novo | 97 (66) |
| Secondary: | 50 (34) |
| MDS | 39 (78) |
| Therapy related | 4 (8) |
| Other∗ | 7 (14) |
| AML cytogenetic risk category (per either NCCN or ELN guidelines),†n (%) | |
| Favorable | 6 (4) |
| Intermediate | 107 (73) |
| Poor | 25 (17) |
| Unknown | 9 (6) |
| Prior AML therapy outcome, n (%) | |
| Refractory | 51 (35) |
| Relapsed | 96 (65) |
| Remission duration ≤12 months | 67 (70) |
| Remission duration >12 months | 29 (30) |
| Number of priorregimens | |
| Median (range) | 2 (1-7) |
| 1 regimen, n (%) | 48 (33) |
| 2 regimens, n (%) | 45 (31) |
| ≥3 regimens, n (%) | 54 (37) |
| Prior treatments received,‡n (%) | |
| Induction therapy§ | 143 (97) |
| Cytarabine | 105 (71) |
| Idarubicin | 64 (44) |
| Daunorubicin | 31 (21) |
| Fludarabine | 25 (17) |
| Hypomethylating agent | 58 (39) |
| As a single agent | 21 (14) |
| In combination with venetoclax | 8 (5) |
| Gemtuzumab based combinations | 11 (7) |
| Venetoclax | 12 (8) |
| Allogeneic HSCT | 17 (12) |
| Hematologic laboratory parameters, median (range) | |
| Percentage of bone marrow blasts | 42 (4-98) |
| Percentage of peripheral blood blasts | 22.5 (1-96) |
| White blood cells ×109/L | 2 (0.1-75) |
| Absolute neutrophil count ×109/L | 0.35 (0-5.3) |
| Renal function (creatinine clearance), n (%) | |
| Normal (≥90 mL/min) | 60 (41) |
| Mildly impaired (60-89 mL/min) | 66 (45) |
| Moderately impaired (30-59 mL/min) | 21 (14) |
| Severely impaired (15-29 mL/min) | 0 |
| IDH1mutation type (as determined by investigator), n (%) | |
| R132C | 90 (61) |
| R132H | 34 (23) |
| R132G | 10 (7) |
| R132S | 10 (7) |
| R132L | 3 (2) |
| Number of co-occurring mutations, n (%)‖ | |
| 1 to 3 | 91 (62) |
| 4 to 7 | 19 (13) |
| None | 4 (3) |
| Not done/unknown | 33 (22) |
| Co-occurring mutations in >5% patients, n (%)‖ | |
| Epigenetic | 80 (54) |
| DNMT3A | 67 (46) |
| TET2 | 22 (15) |
| Differentiation | 62 (42) |
| NPM1 | 31 (21) |
| RUNX1 | 20 (14) |
| PHF6 | 8 (5) |
| RTK pathways | 58 (40) |
| FLT3 | 15 (10) |
| NRAS | 14 (10) |
| JAK2 | 12 (8) |
| Chromatin | 50 (34) |
| ASXL1 | 23 (16) |
| STAG2 | 12 (8) |
| BCOR | 12 (8) |
| Splicing | 43 (29) |
| SRSF2 | 25 (17) |
| U2AF1 | 10 (7) |
| Other | 15 (10) |
| TP53 | 9 (6) |
ECOG, Eastern Cooperative Oncology Group; ELN, European Leukemia Net; NCCN, National Comprehensive Cancer Network; PS, performance status; RTK, receptor tyrosine kinase.
Other secondary AML types were essential thrombocytopenia and myelofibrosis (each 2 patients; 1%) and chronic myelomonocytic leukemia myeloproliferative neoplasm and polycythemia vera (each 1 patient; 1%).
Data on the type of classification system of cytogenetic risk was used (either NCCN or ELN guidelines) were not collected.
Other prior antineoplastic agents received were lomustine (11 patients; 7%); mitoxantrone (9 patients; 6%); etoposide (5 patients; 3%); amsacrine (3 patients; 2%); cladribine, doxorubicin, durvalumab, melphalan, and midostaurin (each 2 patients; 1%); and alemtuzumab, cyclophosphamide, dinaciclib, enasidenib, glasdegib, hydroxycarbamide, idasanutlin, mercaptopurine, and methotrexate (each 1 patient; 1%).
Data on the specific induction regimen used were not collected.
Mutations apart from IDH1.