Table 1.
Phase II studies with omacetaxine
| Study | Drug | Population | N | CHR | MCyR | CCR | Grade 3/4 hematologic toxicity | DoR |
|---|---|---|---|---|---|---|---|---|
| O’Brien [11] | HHT | CML, late CP | 71 | 42/58 (72%) | 11 (15%) | 5 (7%) | TCP (30%), neut (39%) | N/A |
| O’Brien [12] | HHT + IFN | CML, early CP | 90 | 83 (92%) | 27% | 4% | TCP (13%), neut (27%) | N/A |
| O’Brien [13] | HHT +IFN | CML, early and late CP | 47 | 36/43 (84%) | (49%) | 10 (21%) | TCP (77%); neut (77%) anemia (36%) | N/A |
| Kantarjian [14] | HHT + AraC | CML, CP and AP | 105 | 61 (72%) | 12 (15%) | 4 (5%) | TCP (4%), neut (13%) | N/A |
| Stone [15] | HHT + AraC | CML, CP | 44 | 36 (82%) | 4 (17%) | N/A | Neut (66%) | N/A |
| Nicolini [53] | Omacetaxine | T315I mutated | 8 | 5 (63%) | 0 | 3 (37%) | 100% | N/A |
| Cortes [54] | Omacetaxine | T315I mutated | 62 | 47 (77%) | 14 (23%) | 10 (16%) | TCP (76%), neut (44%), anemia (32%) | 9.1 months |
| Cortes [55] | Omacetaxine | Failed ≥ 2 TKIs, CP | 46 | 31 (67%) | 10 (22%) | 2 (4%) | TCP (54%), neut (48%), anemia (33%) | 7.0 months |
| Cortes [56] | Omacetaxine | Failed ≥ 2 TKIs, CP | 81 | 0 | 16 (20%) | 8 (10%) | TCP (67%), neut (47%), anemia (37%) | 12.2 months |
| Nicolini [57] | Omacetaxine | Failed ≥ 2 TKIs, AP | 41 | 11 (27%)a | 6 (15%)b | 0 | TCP (51%), neut (22%), anemia (37%) | 9.0 months |
| Cortes [58] | Omacetaxine |
Failed ≥ 2 TKIs, CP Failed ≥ 2 TKIs, AP |
76 35 |
N/A 5 (14%)a |
14 (18%) 0 |
6 (8%) 0 |
79% 73% |
12.5 months 4.7 months |
HHT homoharringtonine, IFN interferon-alpha, AraC cytarabine, CHR complete hematologic response, MCyR major cytogenetic response, CCR complete cytogenetic response, DoR duration of response, TCP thrombocytopenia, neut neutropenia, TKI tyrosine kinase inhibitor, CP chronic phase, AP accelerated phase
aPatients achieved a major hematologic response
bPatients achieved a minor cytogenetic response