Patient and disease characteristics at baseline
| Characteristic . | Phase 1 . | Phase 2 . | Phase 1-2 . | |||
|---|---|---|---|---|---|---|
| 45 mg/m2 (n = 3) . | 56 mg/m2 (n = 3) . | 70 mg/m2 (n = 15) . | 88 mg/m2 (n = 6) . | 70 mg/m2 (n = 89) . | 70 mg/m2 (n = 104) . | |
| Median age, y (range) | 71 (53-84) | 73 (72-76) | 62 (43-78) | 59.5 (48-69) | 70 (41-88) | 68.5 (41-88) |
| Age group, n (%) | ||||||
| <65 y | 1 (33) | 0 | 8 (53) | 5 (83) | 26 (29) | 34 (33) |
| 65 to <75 y | 1 (33) | 2 (67) | 6 (40) | 1 (17) | 35 (39) | 41 (39) |
| ≥75 y | 1 (33) | 1 (33) | 1 (7) | 0 | 28 (31) | 29 (28) |
| Sex, n (%) | ||||||
| Male | 2 (67) | 1 (33) | 11 (73) | 1 (17) | 49 (55) | 60 (58) |
| Female | 1 (33) | 2 (67) | 4 (27) | 5 (83) | 40 (45) | 44 (42) |
| ECOG PS, n (%) | ||||||
| 0 | 1 (33) | 2 (67) | 6 (40) | 3 (50) | 39 (44) | 45 (43) |
| 1 | 2 (67) | 1 (33) | 9 (60) | 3 (50) | 50 (56) | 59 (57) |
| Median time since initial diagnosis, y (range) | 3.1 (1.8-3.6) | 3.4 (2.1-4.8) | 1.3 (0.2-19.4) | 3.6 (1.2-5.9) | 2.9 (0.2-13.2) | 2.8 (0.2-19.4) |
| ISS stage, n (%) | ||||||
| I | 1 (33) | 3 (100) | 9 (60) | 5 (83) | 33 (37) | 42 (40) |
| II | 1 (33) | 0 | 4 (27) | 1 (17) | 41 (46) | 45 (43) |
| III | 1 (33) | 0 | 2 (13) | 0 | 13 (15) | 15 (14) |
| Missing | 0 | 0 | 0 | 0 | 2 (2) | 2 (2) |
| Cytogenetics by FISH,* n (%) | ||||||
| High risk | N/A | N/A | N/A | N/A | 18 (20) | 18 (17) |
| Standard risk | N/A | N/A | N/A | N/A | 51 (57) | 51 (49) |
| Unknown | N/A | N/A | N/A | N/A | 20 (22) | 35 (34) |
| Median number of prior regimens,† n (range) | 2 (1-2) | 1 (1-2) | 1 (1-3) | 1.5 (1-2) | 1 (1-3) | 1 (1-3) |
| Received BTZ, n (%) | 2 (67) | 2 (67) | 13 (87) | 6 (100) | 73 (82) | 86 (83) |
| BTZ-refractory,‡ n (%) | 2 (67) | 1 (33) | 10 (67) | 5 (83) | 44 (49) | 54 (52)§ |
| Received LEN, n (%) | 2 (67) | 0 | 5 (33) | 1 (17) | 50 (56) | 55 (53) |
| LEN-refractory,‡ n (%) | 2 (67) | 0 | 3 (20) | 1 (17) | 31 (35) | 34 (33)|| |
| Refractory to BTZ and LEN,‡ n (%) | 1 (33) | 0 | 3 (20) | 1 (17) | 17 (19) | 20 (19) |
| Characteristic . | Phase 1 . | Phase 2 . | Phase 1-2 . | |||
|---|---|---|---|---|---|---|
| 45 mg/m2 (n = 3) . | 56 mg/m2 (n = 3) . | 70 mg/m2 (n = 15) . | 88 mg/m2 (n = 6) . | 70 mg/m2 (n = 89) . | 70 mg/m2 (n = 104) . | |
| Median age, y (range) | 71 (53-84) | 73 (72-76) | 62 (43-78) | 59.5 (48-69) | 70 (41-88) | 68.5 (41-88) |
| Age group, n (%) | ||||||
| <65 y | 1 (33) | 0 | 8 (53) | 5 (83) | 26 (29) | 34 (33) |
| 65 to <75 y | 1 (33) | 2 (67) | 6 (40) | 1 (17) | 35 (39) | 41 (39) |
| ≥75 y | 1 (33) | 1 (33) | 1 (7) | 0 | 28 (31) | 29 (28) |
| Sex, n (%) | ||||||
| Male | 2 (67) | 1 (33) | 11 (73) | 1 (17) | 49 (55) | 60 (58) |
| Female | 1 (33) | 2 (67) | 4 (27) | 5 (83) | 40 (45) | 44 (42) |
| ECOG PS, n (%) | ||||||
| 0 | 1 (33) | 2 (67) | 6 (40) | 3 (50) | 39 (44) | 45 (43) |
| 1 | 2 (67) | 1 (33) | 9 (60) | 3 (50) | 50 (56) | 59 (57) |
| Median time since initial diagnosis, y (range) | 3.1 (1.8-3.6) | 3.4 (2.1-4.8) | 1.3 (0.2-19.4) | 3.6 (1.2-5.9) | 2.9 (0.2-13.2) | 2.8 (0.2-19.4) |
| ISS stage, n (%) | ||||||
| I | 1 (33) | 3 (100) | 9 (60) | 5 (83) | 33 (37) | 42 (40) |
| II | 1 (33) | 0 | 4 (27) | 1 (17) | 41 (46) | 45 (43) |
| III | 1 (33) | 0 | 2 (13) | 0 | 13 (15) | 15 (14) |
| Missing | 0 | 0 | 0 | 0 | 2 (2) | 2 (2) |
| Cytogenetics by FISH,* n (%) | ||||||
| High risk | N/A | N/A | N/A | N/A | 18 (20) | 18 (17) |
| Standard risk | N/A | N/A | N/A | N/A | 51 (57) | 51 (49) |
| Unknown | N/A | N/A | N/A | N/A | 20 (22) | 35 (34) |
| Median number of prior regimens,† n (range) | 2 (1-2) | 1 (1-2) | 1 (1-3) | 1.5 (1-2) | 1 (1-3) | 1 (1-3) |
| Received BTZ, n (%) | 2 (67) | 2 (67) | 13 (87) | 6 (100) | 73 (82) | 86 (83) |
| BTZ-refractory,‡ n (%) | 2 (67) | 1 (33) | 10 (67) | 5 (83) | 44 (49) | 54 (52)§ |
| Received LEN, n (%) | 2 (67) | 0 | 5 (33) | 1 (17) | 50 (56) | 55 (53) |
| LEN-refractory,‡ n (%) | 2 (67) | 0 | 3 (20) | 1 (17) | 31 (35) | 34 (33)|| |
| Refractory to BTZ and LEN,‡ n (%) | 1 (33) | 0 | 3 (20) | 1 (17) | 17 (19) | 20 (19) |
ECOG PS, Eastern Cooperative Oncology Group performance status; FISH, fluorescence in situ hybridization; ISS, International Staging System. N/A, not applicable.
Cytogenetics by FISH was not collected during the phase 1 portion of the study. High-risk cytogenetic markers included t(4;14), t(14;16), or deletion 17p.
A new line of therapy was considered to start when: (1) a planned course of therapy was modified to include other treatment agents (alone or in combination) as a result of disease progression, relapse, or toxicity; (2) there was an increase in dose of therapy with the intention of recapturing response in a patient who had evidence of progression on therapy; or (3) a planned period of observation off therapy was interrupted by a need for additional treatment of the disease.
Refractory was defined as disease not achieving a minimal response or better, progressing during therapy, or within 60 days after completion of therapy. Only 4 of the 62 BTZ-refractory patients and 4 of the 37 LEN-refractory patients had disease not achieving a minimal response or better as the sole reason for being characterized as refractory.
Twenty-three patients did not achieve a minimal response or better, 26 progressed on therapy, and 25 progressed within 60 days after completion of therapy (patients may belong to more than 1 subcategory).
Sixteen patients did not achieve a minimal response or better, 25 progressed on therapy, and 7 progressed within 60 days after completion of therapy (patients may belong to more than 1 subcategory).