Table 2.
Primary and secondary efficacy endpoints at week 25
| % (95% CI) | All pexidartinib-treated patients (N = 40) | |
|---|---|---|
| By RECIST v1.1 | By TVS | |
| ORR (CR or PR) at week 25 (primary endpoint) | 22.5 (10.8–38.5) | 47.5 (31.5–63.9) |
| CR | 0 (0–8.8) | 0 (0–8.8) |
| PR | 22.5 (10.8–38.5) | 47.5 (31.5–63.9) |
| Sd | 52.5 (36.1–68.5) | 30.0 (16.6–46.5) |
| PD | 2.5 (0.1–13.2) | 0 (0–8.8) |
| NE | 22.5 (10.8–38.5) | 22.5 (10.8–38.5) |
| Best ORR (CR or PR)a | 30.0 (16.6–46.5) | 47.5 (31.5–63.9) |
| CR | 5.0 (0.6–16.9) | 0 (0–8.8) |
| PR | 25.0 (12.7–41.2) | 47.5 (31.5–63.9) |
| Sd | 50.0 (33.8–66.2) | 32.5 (18.6–49.1) |
| PD | 2.5 (0.1–13.2) | 2.5 (0.1–13.2) |
| NE | 17.5 (7.3–32.8) | 17.5 (7.3–32.8) |
CI, confidence interval; CR, complete response; NE, not evaluable; ORR, overall response rate; PD, progressive disease; PR, partial response; RECIST, Response Evaluation Criteria in Solid Tumors; Sd, stable disease; TVS, tumor volume score
aBest overall response was defined as the best response (in the order of CR, PR, Sd, PD, and NE) among all responses recorded from the start of treatment until the last radiographic tumor assessment