Table 3.
Hematologic, Cytogenetic, and Molecular Responses with Asciminib (Combined Once-Daily and Twice-Daily Schedules).*
| Variable | Chronic-Phase CML | Accelerated-Phase CML | ||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|
| No T315I Mutation | T315I Mutation | No T315I Mutation | T315I Mutation | |||||||||
| Overall (N = 113)† | Response Achieved | Response Maintained | Overall (N = 28)† | Response Achieved | Response Maintained | Overall (N = 4)† | Response Achieved | Response Maintained | Overall (N = 5)† | Response Achieved | Response Maintained | |
| Median follow-up (range) — wk | 72 (0.1–167) | 37 (0.7–167) | 46 (15–72) | 16 (6–120) | ||||||||
| Patients remaining in the study — no. (%) | 88 (78) | 19 (68) | 2 (50) | 1 (20) | ||||||||
| Complete hematologic response — no./total no. (%)‡ | 34/37 (92) | 14/16 (88) | 3/3 (100) | 4/5 (80) | ||||||||
| Major cytogenetic response — no./total no. (%)‡§ | 85/110 (77) | 24/40 (60) | 61/70 (87) | 15/25 (60) | 11/20 (55) | 4/5 (80) | 0/4 | 0/2 | 0/2 | 1/5 (20) | 1/4 (25) | 0/1 |
| Complete cytogenetic response — no./total no. (%)‡§ | 77/110 (70) | 31/57 (54) | 46/53 (87) | 11/25 (44) | 9/22 (41) | 2/3 (67) | 0/4 | 0/2 | 0/2 | 1/5 (20) | 1/4 (25) | 0/1 |
| Major molecular response — no./total no. (%)‡¶ | ||||||||||||
| In all patients | ||||||||||||
| By 6 mo | 37/99 (37) | 19/80 (24) | 18/19 (95) | 5/20 (25) | 4/19 (21) | 1/1 (100) | 0/4 | 0/3 | 0/1 | 1/5 (20) | 1/5 (20) | 0 |
| By 12 mo | 44/91 (48) | 26/72 (36) | 18/19 (95) | 5/18 (28) | 4/17 (24) | 1/1 (100) | 0/4 | 0/3 | 0/1 | 1/5 (20) | 1/5 (20) | 0 |
| In patients with ≤2 previous TKIs∥ | ||||||||||||
| By 6 mo | 13/25 (52) | 5/15 (33) | 8/10 (80) | 4/10 (40) | 3/9 (33) | 1/1 (100) | 0/1 | 0/1 | 0 | |||
| By 12 mo | 15/25 (60) | 7/15 (47) | 8/10 (80) | 4/9 (44) | 3/8 (38) | 1/1 (100) | 0/1 | 0/1 | 0 | |||
| In patients with >2 previous TKIs** | ||||||||||||
| By 6 mo | 24/74 (32) | 14/64 (22) | 10/10 (100) | 1/10 (10) | 1/10 (10) | 0 | 0/3 | 0/2 | 0/1 | 1/5 (20) | 1/5 (20) | 0 |
| By 12 mo | 29/66 (44) | 19/56 (34) | 10/10 (100) | 1/9 (11) | 1/9 (11) | 0 | 0/3 | 0/2 | 0/1 | 1/5 (20) | 1/5 (20) | 0 |
| In patients with resistance to or unacceptable side effects from ponatinib†† | ||||||||||||
| By 6 mo | 7/17 (41) | 3/13 (23) | 4/4 (100) | 1/7 (14) | 1/7 (14) | 0/0 | 0/2 | 0/2 | ||||
| By 12 mo | 8/14 (57) | 4/10 (40) | 4/4 (100) | 1/6 (17) | 1/6 (17) | 0/0 | 0/2 | 0/2 | ||||
For definitions of hematologic, cytogenetic, and molecular responses, see the Methods section in the Supplementary Appendix.
Shown is the number of patients who received at least one dose of asciminib.
The total number is the number of patients who could be evaluated.
Data on cytogenetic responses are based on patients who presented with Philadelphia chromosome–positive CML at baseline. Calculation of the number of patients in whom a major cytogenetic response or complete cytogenetic response was achieved is based on patients not in the respective response category at baseline.
Molecular-response assessment is reported only for patients with the b2a2 or b3a2 transcripts; 7 patients had atypical BCR-ABL1 transcripts and were not included in the response assessment.
The numbers of patients who received at least one dose of asciminib were as follows: 34 with chronic-phase CML without a T315I mutation, 12 with chronic-phase CML with a T315I mutation, and 1 with accelerated-phase CML without a T315I mutation.
The numbers of patients who received at least one dose of asciminib were as follows: 79 with chronic-phase CML without a T315I mutation, 16 with chronic-phase CML with a T315I mutation, 3 with accelerated-phase CML without a T315I mutation, and 5 with accelerated-phase CML with a T315I mutation.
The numbers of patients who received at least one dose of asciminib were as follows: 18 with chronic-phase CML without a T315I mutation, 11 with chronic-phase CML with a T315I mutation, and 2 with accelerated-phase CML with a T315I mutation.