Table 3.
Results of clinical trials evaluating immunotherapeutic approaches in GIST.
| Description | Phase | Number of GISTs | ORR (RECIST) | Median PFS | mOS | Notes | Reference |
|---|---|---|---|---|---|---|---|
| Peg-IFNα2b + imatinib followed by imatinib maintenance | II | 8 | 100% | NR (> 3years) | NR | New PR achieved after reintroduction of peg-IFNα2 in a patient who progressed on imatinib maintenance therapy | Chen et al, 2012 (66) |
| Dasatinib + Ipilimumab in advanced GIST and other sarcomas | Ib | 20 | 0% | 2.8M | mOS: 13,5M | 7/13 evaluable GISTs had PR by CHOI criteria | D’Angelo et al, 2017 (67) |
| Pembrolizumab + Cyclophosphamide in advanced STS | II | 9 | 0% | 6M-PFS: 11% | – | 63% of GISTs showed a high IDO expression | Toulmonde et al, 2018 (52) |
| Nivolumab +/- ipilimumab in advanced GIST refractory to imatinib | II | N: 15 | N: 0% | N: 8.57w | – | – | Singh AS et al, 2018 (68) |
| N+ I: 12 | N+I: 8.3% | N+I: 9.1w | |||||
| Nivolumab +/- Ipilimumab in advanced STS | II | N: 9 | N : 0% | N : 1.5M | N: 9.1M | - | Chen et al, 2020 (69) |
| N + I: 9 | N+I : 0% | N+I : 2.9M | N+I :12.1M |
ORR, objective response rate; median PFS, median progression-free survival; mOS, median overall survival; STS, soft-tissue sarcoma; 6M-PFS, 6 month progression-free survival; N, nivolumab; N+I, nivolumab+ipilimumab; M, months; w, weeks.