TABLE A2.
Variable | Splice Donor (n = 33) | Splice Acceptor (n = 20) | SD and SA (BS and I, n = 2) | Base Substitution (n = 29) | Indel (n = 24) |
---|---|---|---|---|---|
Objective response rate, % | 24 (72.7) | 16 (80.0) | 2 (100.0) | 20 (69.0) | 20 (83.3) |
95% CI | 54.5 to 86.7 | 56.3 to 94.3 | 15.8 to 100.0 | 49.2 to 84.7 | 62.6 to 95.3 |
Disease control rate, % | 31 (93.9) | 20 (100.0) | 2 (100.0) | 27 (93.1) | 24 (100.0) |
95% CI | 79.8 to 99.3 | 83.2 to 100.0 | 15.8 to 100.0 | 77.2 to 99.2 | 85.8 to 100.0 |
Duration of response, months, median (95% CI) |
16.7 (13.5 to NE) | 9.2 (3.9 to 17.1) | 10.6 (9.2 to NE) | 17.3 (15.3 to NE) | 9.2 (3.9 to 13.5) |
6-month PFS rate (95% CI) | 80.8 (59.8 to 91.5) | 58.8 (32.5 to 77.8) | 100.0 (100.0 to 100.0) | 86.4 (63.4 to 95.4) | 57.1 (33.8 to 74.9) |
12-month PFS rate (95% CI) | 72.5 (50.7 to 85.8) | 29.4 (10.7 to 51.1) | 0.0 (NE to NE) | 76.5 (52.3 to 89.5) | 33.3 (14.9 to 53.1) |
PFS, months, median (95% CI) | 17.5 (6.3 to 20.3) | 8.2 (4.7 to 17.9) | 15.1 (10.0 to NE) | 17.5 (6.3 to 20.3) | 8.2 (4.7 to 14.5) |
OS, months, median (95% CI) | 22.2 (16.2 to NE) | 20.3 (13.7 to NE) | NR (17.9 to NE) | NR (16.2 to NE) | 19.0 (13.7 to NE) |
Abbreviations: BS, base substitution; I, indel; NE, not estimated; NR, not reached; OS, overall survival; PFS, progression-free survival; SA, splice acceptor; SD, splice donor.