Table 3.
Primary and secondary efficacy endpoints and treatment duration.
| Outcome | Locally advanced basal cell carcinoma |
|---|---|
| Downstaging procedure (ITT) (%) 95% CI | 44/55 (80%) [67 to 90] |
| Downstaging procedure after ≥4 months of vismodegib | 35/42 (85,7%) [71 to 95] |
| Overall Response Rate according to RECIST 1.1 criteria | 39/55 (70,9%) [59 to 83] |
| Complete Response | 14/55 (25,5%) [14 to 37] |
| Partial Response | 25/55 (45,5%) [32 to 59] |
| Stability | 16/55 (29,1%) [17 to 41] |
| Progression | 0/55 (0%) [0 to 5] |
| Improvement of Skindex score at each cycle | 2.07/visit |
| p-value | <0.0001 |
| Duration of treatment (months) | 6.0 (± 2.3) |
| Median | 6.0 |
| 3-Year follow-up of target lesion, for success group patients (N=44) | |
| Recurrence | 16/44 (36,4%) [22 to 51] |
| Response ongoing | 10/44 (22,7%) [10 to 35] |
| Lost to follow-up without any known recurrence | 12/44 (27,3%) [14 to 40] |
| Died without any known recurrence | 6/44 (13,6%) [3 to 24] |
CI, confidence interval; ITT, intent to treat.