Table 2. Outcome Measures and Reasons for Treatment Discontinuation.
| Outcome measure | Patients, No. (%) (N = 33) |
|---|---|
| Best overall response to immunotherapya | |
| Major response | 3 (9) |
| Partial response | 9 (27) |
| Stable disease | 16 (48) |
| Progression of diseaseb | 4 (12) |
| Unevaluablec | 1 (3) |
| Reason for treatment discontinuation | |
| Completed therapy | 29 (88) |
| Toxic effect from drug | 2 (6) |
| Progression of disease | 2 (6) |
| Physician discretion | 0 |
| Withdrawal of consent | 0 |
| Death | 0 |
| Follow-up, median (range), mo | 21.1 (5.4-43.6+) |
| CFS, median (95% CI), mo | NR (24.3-NR) |
| No. of eventsd | 9 (27) |
| 1-y CFS, % (95% CI) | 76.6 (56.8-88.2) |
| 2-y CFS, % (95% CI) | 72.8 (52.6-85.5) |
| 3-y CFS, % (95% CI) | 66.2 (43.3-81.5) |
| OS, median (95% CI), mo | NR |
| No. of events | 0 |
| 2-y OS, % | 100 |
Abbreviations: CFS, cancer-free survival; NR, not reached; OS, overall survival; +, censored at last follow-up as of data cutoff.
a
Determined by the change in bidirectional measurements and degree of pathologic dysplasia composite scoring of target leukoplakia lesion(s).
b
Determined via composite score (at least a 10% increase in the total composite score from baseline) or development of either oral squamous cell carcinoma or carcinoma in situ while receiving study treatment.
c
One patient experienced toxic effects and withdrew consent from treatment prior to rebiopsy.
d
CFS events include development of oral squamous cell carcinoma documented via biopsy confirmation or death, whichever occurred first. None of the 9 events were due to death alone.