Patient characteristics and clinical outcomes
| . | Total (N = 85) . | A 50 mg (n = 8) . | B 100 mg (n = 8) . | C 200 mg (n = 9) . | D 400 mg (n = 8) . | E 600 mg (n = 18) . | F 50 mg + POSA (n = 9) . | G 100 mg + POSA (n = 9) . | H 600 mg (n = 16) . | CR + CRh + CRi . | Median OS, mo (95% CI) . |
|---|---|---|---|---|---|---|---|---|---|---|---|
| Median age, y (range) | 71 (63-80) | 73 (68-80) | 70 (65-77) | 73 (63-80) | 70 (64-78) | 71 (67-77) | 73 (67-75) | 70 (66-76) | 69 (63-75) | — | — |
| De novo AML, n (%) | 52 (61) | 3 (38) | 5 (62) | 6 (67) | 5 (62) | 9 (50) | 6 (67) | 6 (67) | 12 (75) | 88% | 33.1 (19.3-54.3) |
| Secondary AML, n (%) | 33 (39) | 5 (63) | 3 (38) | 3 (33) | 3 (38) | 9 (50) | 3 (33) | 3 (33) | 4 (25) | 54% | 8.0 (4.6-13.2) |
| t-AML, n (%) | 6 (7) | 1 (12) | — | — | 1 (12) | 1 (5) | — | 1 (11) | 2 (13) | 33% | 17.0 (0.9 to NA) |
| Prior MDS, n (%) | 17 (20) | 4 (50) | 2 (25) | 3 (33) | 1 (12) | 5 (28) | 2 (22) | — | — | 59% | 7.2 (3.7-15.1) |
| Prior CMML, n (%) | 5 (6) | — | 1 (13) | — | — | 2 (11) | 1 (11) | 1 (11) | — | 60% | 6.3 (0.8 to NA) |
| Prior MPN, n (%) | 5 (6) | — | — | — | 1 (12) | 2 (11) | — | — | 2 (13) | 40% | 8.6 (0.3 to NA) |
| Prior HMA, n (%) | 16 (19) | 3 (38) | 3 (38) | 2 (22) | 2 (25) | 6 (33) | — | — | — | 44% | 5.7 (1.4-8.8) |
| ELN 2022, n (%) | |||||||||||
| Favorable | 18 (21) | 1 (12) | 1 (12) | 2 (22) | 1 (12) | 2 (11) | 3 (33) | 4 (44) | 4 (25) | 89% | 54.3 (12.5 to NA) |
| Intermediate | 16 (19) | 1 (12) | 2 (25) | 2 (22) | 3 (38) | 4 (22) | — | 1 (11) | 3 (19) | 81% | 29.4 (3.5 to NA) |
| Adverse | 51 (60) | 6 (75) | 5 (62) | 5 (56) | 4 (50) | 12 (67) | 6 (67) | 4 (44) | 9 (56) | 69% | 13.2 (8.0 to 20.1) |
| Selected mutations, n (%) | |||||||||||
| NPM1 | 20 (24) | 1 (12) | 1 (12) | 3 (33) | 1 (12) | 3 (17) | 3 (33) | 4 (44) | 4 (25) | 85% | 43.9 (5.5 to NA) |
| IDH2 | 18 (21) | — | — | 1 (11) | 2 (25) | 5 (28) | 2 (22) | 3 (33) | 5 (31) | 89% | 58.5 (33.2 to NA) |
| IDH1 | 8 (9) | 1 (12) | 1 (12) | — | 1 (12) | 4 (22) | — | — | 1 (6) | 88% | 34.8 (3.45 to NA) |
| FLT3-ITD | 6 (7) | — | 1 (12) | 2 (22) | 1 (12) | 1 (5) | — | — | 1 (6) | 83% | 17.5 (0.8 to NA) |
| NRAS | 18 (21) | — | 4 (50) | 2 (22) | 3 (38) | 4 (22) | 2 (22) | 1 (11) | 2 (13) | 89% | 15.1 (6.3-24.1) |
| KRAS | 6 (7) | — | 1 (12) | 1 (12) | 1 (12) | 2 (11) | — | 1 (11) | — | 100% | 8.1 (5.3 to NA) |
| TP53 | 15 (18) | 3 (38) | 1 (12) | 3 (33) | 2 (25) | 3 (17) | — | — | 3 (19) | 40% | 4.6 (1.2-8.4) |
| CR + CRh + CRi | 75% | 63% | 76% | 100% | 63% | 67% | 55% | 100% | 81% |
| . | Total (N = 85) . | A 50 mg (n = 8) . | B 100 mg (n = 8) . | C 200 mg (n = 9) . | D 400 mg (n = 8) . | E 600 mg (n = 18) . | F 50 mg + POSA (n = 9) . | G 100 mg + POSA (n = 9) . | H 600 mg (n = 16) . | CR + CRh + CRi . | Median OS, mo (95% CI) . |
|---|---|---|---|---|---|---|---|---|---|---|---|
| Median age, y (range) | 71 (63-80) | 73 (68-80) | 70 (65-77) | 73 (63-80) | 70 (64-78) | 71 (67-77) | 73 (67-75) | 70 (66-76) | 69 (63-75) | — | — |
| De novo AML, n (%) | 52 (61) | 3 (38) | 5 (62) | 6 (67) | 5 (62) | 9 (50) | 6 (67) | 6 (67) | 12 (75) | 88% | 33.1 (19.3-54.3) |
| Secondary AML, n (%) | 33 (39) | 5 (63) | 3 (38) | 3 (33) | 3 (38) | 9 (50) | 3 (33) | 3 (33) | 4 (25) | 54% | 8.0 (4.6-13.2) |
| t-AML, n (%) | 6 (7) | 1 (12) | — | — | 1 (12) | 1 (5) | — | 1 (11) | 2 (13) | 33% | 17.0 (0.9 to NA) |
| Prior MDS, n (%) | 17 (20) | 4 (50) | 2 (25) | 3 (33) | 1 (12) | 5 (28) | 2 (22) | — | — | 59% | 7.2 (3.7-15.1) |
| Prior CMML, n (%) | 5 (6) | — | 1 (13) | — | — | 2 (11) | 1 (11) | 1 (11) | — | 60% | 6.3 (0.8 to NA) |
| Prior MPN, n (%) | 5 (6) | — | — | — | 1 (12) | 2 (11) | — | — | 2 (13) | 40% | 8.6 (0.3 to NA) |
| Prior HMA, n (%) | 16 (19) | 3 (38) | 3 (38) | 2 (22) | 2 (25) | 6 (33) | — | — | — | 44% | 5.7 (1.4-8.8) |
| ELN 2022, n (%) | |||||||||||
| Favorable | 18 (21) | 1 (12) | 1 (12) | 2 (22) | 1 (12) | 2 (11) | 3 (33) | 4 (44) | 4 (25) | 89% | 54.3 (12.5 to NA) |
| Intermediate | 16 (19) | 1 (12) | 2 (25) | 2 (22) | 3 (38) | 4 (22) | — | 1 (11) | 3 (19) | 81% | 29.4 (3.5 to NA) |
| Adverse | 51 (60) | 6 (75) | 5 (62) | 5 (56) | 4 (50) | 12 (67) | 6 (67) | 4 (44) | 9 (56) | 69% | 13.2 (8.0 to 20.1) |
| Selected mutations, n (%) | |||||||||||
| NPM1 | 20 (24) | 1 (12) | 1 (12) | 3 (33) | 1 (12) | 3 (17) | 3 (33) | 4 (44) | 4 (25) | 85% | 43.9 (5.5 to NA) |
| IDH2 | 18 (21) | — | — | 1 (11) | 2 (25) | 5 (28) | 2 (22) | 3 (33) | 5 (31) | 89% | 58.5 (33.2 to NA) |
| IDH1 | 8 (9) | 1 (12) | 1 (12) | — | 1 (12) | 4 (22) | — | — | 1 (6) | 88% | 34.8 (3.45 to NA) |
| FLT3-ITD | 6 (7) | — | 1 (12) | 2 (22) | 1 (12) | 1 (5) | — | — | 1 (6) | 83% | 17.5 (0.8 to NA) |
| NRAS | 18 (21) | — | 4 (50) | 2 (22) | 3 (38) | 4 (22) | 2 (22) | 1 (11) | 2 (13) | 89% | 15.1 (6.3-24.1) |
| KRAS | 6 (7) | — | 1 (12) | 1 (12) | 1 (12) | 2 (11) | — | 1 (11) | — | 100% | 8.1 (5.3 to NA) |
| TP53 | 15 (18) | 3 (38) | 1 (12) | 3 (33) | 2 (25) | 3 (17) | — | — | 3 (19) | 40% | 4.6 (1.2-8.4) |
| CR + CRh + CRi | 75% | 63% | 76% | 100% | 63% | 67% | 55% | 100% | 81% |
CI, confidence interval; CMML, chronic myelomonocytic leukemia; CRh, CR with partial hematologic recovery; CRi, CR with incomplete hematologic recovery; HMA, hypomethylating agent; MDS, myelodysplastic syndrome; MPN, myeloproliferative neoplasm; NA, not assessable; POSA, posaconazole; t-AML, therapy-related AML.