Table 1.

Patient demographics and pretreatment clinical and biological characteristics

All doses (n = 436)400 mg monotherapy (n = 347)
Age
 Median (range), y66 (28-88)66 (28-85)
 ≥70 y, n (%)152 (35)116 (33)
Male, n (%)298 (68)236 (68)
Diagnosis, n (%)
 Chronic lymphocytic leukemia421 (97)339 (98)
 Small lymphocytic lymphoma15 (3)8 (2)
ECOG performance status, n (%)
 0187 (43)139 (40)
 1225 (52)184 (53)
 222 (5)22 (6)
No. of prior therapies, median (range)3 (1-15)3 (1-15)
No. of prior therapies, n (%)
 175 (17)61 (18)
 2 to 3172 (39)133 (38)
 >3189 (43)153 (44)
Bulky nodes, n (%)
 <5 cm216 (51)167 (49)
 5 to <10 cm156 (36)124 (37)
 ≥10 cm56 (13)48 (14)
Cytogenetic abnormalities by FISH*, n (%)
 17p deletion231 (53)208 (60)
 11q deletion125 (29)98 (28)
 Trisomy 1285 (20)62 (18)
 13q deletion261 (60)222 (64)
 No abnormality47 (11)37 (10)
 Other/missing23 (5)11 (3)
TP53 mutation &/or 17p deletion, n/N (%)
 Either or both243 (71)216 (76)
 Neither101 (29)68 (24)
NOTCH1 mutation, n/N (%)
 Mutated37 (15)26 (13)
 Unmutated217 (85)167 (87)
SF3B1 mutation, n/N (%)
 Mutated58 (23)45 (23)
 Unmutated196 (77)148 (77)
IGHV mutational status, n/N (%)
 Mutated57 (24)43 (24)
 Unmutated176 (76)138 (76)
Prior BCRi therapy, n (%)
 Yes149 (34)146 (42)
  Refractory115 (26)112 (32)
  Nonrefractory34 (8)34 (10)
 No287 (66)201 (58)
Fludarabine refractory, n (%)134 (31)107 (31)
  • Inclusion of patients with SLL were from M12-175 trial only. Data are missing for some patients for ECOG status (n = 2), node size (n = 8). For each of these variables, positive results are expressed as a percentage of whole population.

  • BCRi, B-cell receptor inhibitor; ECOG, Eastern Cooperative Oncology Group; IGHV, immunoglobulin heavy chain variable region.

  • * FISH data are reported categorically, with each locus considered independently

  • Informative data were available in N = 254 for TP53, NOTCH1, and SF3B1 mutations and N = 233 for IGHV mutational status among all patients; and N = 193 for TP53, NOTCH1, and SF3B1 mutations and N = 181 for IGHV mutation status in the 400-mg monotherapy subgroup. TP53 and/ 17pDEL FISH results were available for N = 344 among all patients and N = 284 in the 400-mg monotherapy subgroup. For these variables, positive results are expressed as a percentage of the population with available results.