Table 3.
Efficacy analyses
| Efficacy measure | ITT population (N = 50) | PP population (N = 42) |
|---|---|---|
| Local disease controla,b | ||
| Patients with local disease control at 16 weeks, n (%) | 41 (82.0) | 38 (90.5) |
| Local disease control rate at 16 weeks (95% CI) | 82.0 (68.6-91.4) | 90.5 (77.4-97.3) |
| P valuec | 0.0001 | <0.0001 |
| Best tumour response by RECIST v1.1, n (%)a,b | ||
| Complete response (CR)d | 0 | 0 |
| Partial response (PR)d | 14 (29.8) | 13 (31.0) |
| Stable diseased | 31 (66.0) | 29 (69.0) |
| Progressive disease (PD)d | 2 (4.3) | 0 |
| Not evaluated | 3 | 0 |
| Objective response rate (CR + PR)e | 14 (28.0) | 13 (31.0) |
| Disease control rate (CR + PR + stable disease)e | 45 (90.0) | 42 (100.0) |
| Tumour volume response (CT scan)a,b | ||
| Median (range) maximal decrease from baseline, % | −51.6 (+72.2 to −89.9)g | −51.9 (+11.1 to −89.9) |
| Mean (SD) maximal decrease from baseline, % | −44.0 (34.8)g | −49.1 (26.4) |
| P valuef | <0.0001 | <0.0001 |
| Tumour response by 18F-FDG-PET at 12 weeksa,b | ||
| Patients with evaluable images, n | 42 | 39 |
| TLG: median (range) change from baseline, % | −60.5 (+319.2 to −100.0) | −60.5 (+319.2 to −100.0) |
| mean (SD) change from baseline, % | −37.1 (89.9) | −34.8 (92.8) |
| P valuef | 0.0001 | 0.0003 |
| SUVMax: median (range) change from baseline, % | −40.3 (+76.4 to −100.0) | −40.4 (+76.4 to −100.0) |
| mean (SD) change from baseline, % | −36.3 (43.1) | −35.8 (42.9) |
| P valuef | <0.0001 | <0.0001 |
| SULMax: median (range) change from baseline, % | −43.1 (+75.3 to −100.0) | −43.7 (+75.3 to −100.0) |
| mean (SD) change from baseline, % | −36.2 (46.3) | −35.9 (46.3) |
| P valuef | 0.0188 | 0.0232 |
| Surgical resection with curative intent, n (%) | 10 (20.0) | 10 (23.8) |
| R0 margin status, n (% of resections) | 8 (80.0) | 8 (80.0) |
| R1 margin status, n (% of resections) | 2 (20.0) | 2 (20.0) |
| CA 19-9 responseb | ||
| Assessable patients with baseline CA 19-9 ≥35 U/ml, n | 38 | 33 |
| Median (range) maximal decrease from baseline, % | −80.7 (+50.0 to −99.9) | −82.3 (+50.0 to −99.9) |
| Mean (SD) maximal decrease from baseline, % | −68.1 (35.4) | −70.9 (34.0) |
| P valuef | 0.0006 | 0.0024 |
| Local progression-free survival, monthsa | ||
| Median (95% CI), uncensored for resection | 9.9 (7.3-12.6) | 9.8 (7.3-12.6) |
| Median (95% CI), patients censored before resection | 9.5 (7.2-11.3) | 9.3 (7.2-11.3) |
| Progression-free survival, monthsa | ||
| Median (95% CI), uncensored for resection | 9.3 (5.7-11.3) | 9.3 (5.8-11.3) |
| Median (95% CI), patients censored before resection | 7.7 (5.7-9.9) | 7.7 (5.8-9.9) |
| Overall survival, months | ||
| Median (95% CI) | 15.2 (11.3-18.8) | 15.5 (11.4-20.1) |
CI, confidence interval; CT, computed tomography; 18F-FDG-PET, [18F]2-fluoro-2-deoxy-d-glucose positron emission tomography; ITT, intention-to-treat population; PP, per protocol population; SD, standard deviation; SULMax, maximum standardized uptake value corrected for lean body mass; SUVMax, maximum standardized uptake value; TLG, total lesion glycolysis.
By central image reader analysis.
Response before surgical resection.
P values for Fisher’s Exact test, comparing the binomial proportion with the null hypothesis proportion of 0.55.
Percentages based on the number of assessable study participants.
Percentages based on the number of all study participants.
P value for paired t-test, percent change from baseline.
n = 47 Patients with evaluable post-baseline scans.