Table 1

Patient and disease characteristics at baseline

CharacteristicPhase 1Phase 2Phase 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 y1 (33)08 (53)5 (83)26 (29)34 (33)
 65 to <75 y1 (33)2 (67)6 (40)1 (17)35 (39)41 (39)
 ≥75 y1 (33)1 (33)1 (7)028 (31)29 (28)
Sex, n (%)
 Male2 (67)1 (33)11 (73)1 (17)49 (55)60 (58)
 Female1 (33)2 (67)4 (27)5 (83)40 (45)44 (42)
ECOG PS, n (%)
 01 (33)2 (67)6 (40)3 (50)39 (44)45 (43)
 12 (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 (%)
 I1 (33)3 (100)9 (60)5 (83)33 (37)42 (40)
 II1 (33)04 (27)1 (17)41 (46)45 (43)
 III1 (33)02 (13)013 (15)15 (14)
 Missing00002 (2)2 (2)
Cytogenetics by FISH,* n (%)
 High riskN/AN/AN/AN/A18 (20)18 (17)
 Standard riskN/AN/AN/AN/A51 (57)51 (49)
 UnknownN/AN/AN/AN/A20 (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)05 (33)1 (17)50 (56)55 (53)
 LEN-refractory, n (%)2 (67)03 (20)1 (17)31 (35)34 (33)||
Refractory to BTZ and LEN, n (%)1 (33)03 (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).