Table 2.
Response and Follow-Up Data
| Response | Overall |
Mutation Status |
Fisher's Exact P | ||||
|---|---|---|---|---|---|---|---|
| Amplified KIT |
Mutated KIT |
||||||
| No. | % | No. | % | No. | % | ||
| Best overall response | 7 | 29.2 | — | — | 7 | 53.8 | |
| PR | .003 | ||||||
| SD | 5 | 20.8 | 2 | 18.2 | 3 | 23.1 | |
| PD | 12 | 50.0 | 9 | 81.8 | 3 | 23.1 | |
| BORR (CR + PR) | .006 | ||||||
| No | 17 | 70.8 | 11 | 100.0 | 6 | 46.2 | |
| Yes | 7 | 29.2 | — | — | 7 | 53.8 | |
| Disease control (CR + PR + SD) | .01 | ||||||
| No | 12 | 50.0 | 9 | 81.8 | 3 | 23.1 | |
| Yes | 12 | 50.0 | 2 | 18.2 | 10 | 76.9 | |
| Progression | 1 | 4.2 | — | — | 1 | 7.7 | |
| No | .99 | ||||||
| Yes | 23 | 95.8 | 11 | 100.0 | 12 | 92.3 | |
| Survival status | 5 | 20.8 | 1 | 9.1 | 4 | 30.8 | |
| Alive | .33 | ||||||
| Dead | 19 | 79.2 | 10 | 90.9 | 9 | 69.2 | |
| Reason off study | 1 | 4.2 | — | — | 1* | 7.7 | |
| Unacceptable toxicity | .99 | ||||||
| Progressive illness | 21 | 87.5 | 10 | 90.9 | 11 | 84.6 | |
| Intercurrent illness | 1 | 4.2 | — | — | 1† | 7.7 | |
| Withdrew consent | 1 | 4.2 | 1 | 9.1 | — | — | |
NOTE. P values compare amplified versus mutated KIT.
Abbreviations: BORR, best overall response rate; CR, complete response; PD, progressive disease; PR, partial response; SD, stable disease.
*
Patient 2 off study because of grade 3 bleeding/hemorrhage.
†
Patient 14 ended study participation because of preceding and ongoing issues regarding ostomy strictures.