Patient demographics and pretreatment clinical and biological characteristics
| . | All doses (n = 436) . | 400 mg monotherapy (n = 347) . |
|---|---|---|
| Age | ||
| Median (range), y | 66 (28-88) | 66 (28-85) |
| ≥70 y, n (%) | 152 (35) | 116 (33) |
| Male, n (%) | 298 (68) | 236 (68) |
| Diagnosis, n (%) | ||
| Chronic lymphocytic leukemia | 421 (97) | 339 (98) |
| Small lymphocytic lymphoma | 15 (3) | 8 (2) |
| ECOG performance status, n (%) | ||
| 0 | 187 (43) | 139 (40) |
| 1 | 225 (52) | 184 (53) |
| 2 | 22 (5) | 22 (6) |
| No. of prior therapies, median (range) | 3 (1-15) | 3 (1-15) |
| No. of prior therapies, n (%) | ||
| 1 | 75 (17) | 61 (18) |
| 2 to 3 | 172 (39) | 133 (38) |
| >3 | 189 (43) | 153 (44) |
| Bulky nodes, n (%) | ||
| <5 cm | 216 (51) | 167 (49) |
| 5 to <10 cm | 156 (36) | 124 (37) |
| ≥10 cm | 56 (13) | 48 (14) |
| Cytogenetic abnormalities by FISH*, n (%) | ||
| 17p deletion | 231 (53) | 208 (60) |
| 11q deletion | 125 (29) | 98 (28) |
| Trisomy 12 | 85 (20) | 62 (18) |
| 13q deletion | 261 (60) | 222 (64) |
| No abnormality | 47 (11) | 37 (10) |
| Other/missing | 23 (5) | 11 (3) |
| TP53 mutation†&/or 17p deletion, n/N (%) | ||
| Either or both | 243 (71) | 216 (76) |
| Neither | 101 (29) | 68 (24) |
| NOTCH1 mutation†, n/N (%) | ||
| Mutated | 37 (15) | 26 (13) |
| Unmutated | 217 (85) | 167 (87) |
| SF3B1 mutation†, n/N (%) | ||
| Mutated | 58 (23) | 45 (23) |
| Unmutated | 196 (77) | 148 (77) |
| IGHV mutational status†, n/N (%) | ||
| Mutated | 57 (24) | 43 (24) |
| Unmutated | 176 (76) | 138 (76) |
| Prior BCRi therapy, n (%) | ||
| Yes | 149 (34) | 146 (42) |
| Refractory | 115 (26) | 112 (32) |
| Nonrefractory | 34 (8) | 34 (10) |
| No | 287 (66) | 201 (58) |
| Fludarabine refractory, n (%) | 134 (31) | 107 (31) |
| . | All doses (n = 436) . | 400 mg monotherapy (n = 347) . |
|---|---|---|
| Age | ||
| Median (range), y | 66 (28-88) | 66 (28-85) |
| ≥70 y, n (%) | 152 (35) | 116 (33) |
| Male, n (%) | 298 (68) | 236 (68) |
| Diagnosis, n (%) | ||
| Chronic lymphocytic leukemia | 421 (97) | 339 (98) |
| Small lymphocytic lymphoma | 15 (3) | 8 (2) |
| ECOG performance status, n (%) | ||
| 0 | 187 (43) | 139 (40) |
| 1 | 225 (52) | 184 (53) |
| 2 | 22 (5) | 22 (6) |
| No. of prior therapies, median (range) | 3 (1-15) | 3 (1-15) |
| No. of prior therapies, n (%) | ||
| 1 | 75 (17) | 61 (18) |
| 2 to 3 | 172 (39) | 133 (38) |
| >3 | 189 (43) | 153 (44) |
| Bulky nodes, n (%) | ||
| <5 cm | 216 (51) | 167 (49) |
| 5 to <10 cm | 156 (36) | 124 (37) |
| ≥10 cm | 56 (13) | 48 (14) |
| Cytogenetic abnormalities by FISH*, n (%) | ||
| 17p deletion | 231 (53) | 208 (60) |
| 11q deletion | 125 (29) | 98 (28) |
| Trisomy 12 | 85 (20) | 62 (18) |
| 13q deletion | 261 (60) | 222 (64) |
| No abnormality | 47 (11) | 37 (10) |
| Other/missing | 23 (5) | 11 (3) |
| TP53 mutation†&/or 17p deletion, n/N (%) | ||
| Either or both | 243 (71) | 216 (76) |
| Neither | 101 (29) | 68 (24) |
| NOTCH1 mutation†, n/N (%) | ||
| Mutated | 37 (15) | 26 (13) |
| Unmutated | 217 (85) | 167 (87) |
| SF3B1 mutation†, n/N (%) | ||
| Mutated | 58 (23) | 45 (23) |
| Unmutated | 196 (77) | 148 (77) |
| IGHV mutational status†, n/N (%) | ||
| Mutated | 57 (24) | 43 (24) |
| Unmutated | 176 (76) | 138 (76) |
| Prior BCRi therapy, n (%) | ||
| Yes | 149 (34) | 146 (42) |
| Refractory | 115 (26) | 112 (32) |
| Nonrefractory | 34 (8) | 34 (10) |
| No | 287 (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.