|
| | Study design | Number of patients | Primary endpoint | Main findings |
|
| Demetri et al. [30] | Randomized, double-blind, placebo-controlled, multicenter, international trial comparing sunitinib versus placebo after imatinib failure | 321 (207 sunitinib versus 105 placebo patients) | Tumor progression | Median time to tumor progression: 27.3 weeks in patients receiving sunitinib versus 6.4 weeks with placebo |
|
| MetaGIST [31] | Analysis of two large, randomized, cooperative group studies comparing two doses of IM (400 mg daily versus twice daily) in 1640 patients with advanced GISTs | 1640 (data analysis after 344 and 321 cases of progression or death in each study) | PFS and OS | High-dose imatinib 800 mg daily improved PFS but not OS compared to imatinib 400 mg daily |
|
| Reichardt et al. [32] | Randomized phase III open-label trial comparing nilotinib versus best supportive care with advanced GIST following prior imatinib/sunitinib failure | 248 (2 : 1 randomization nilotinib or best supportive care) | PFS, OS | Subset analysis of patients with one prior regimen each of imatinib and sunitinib showed significant increase in median OS in favor of nilotinib versus best supportive care |
|
| Demetri et al. [33] | Randomized, double-blinded, placebo-controlled, multicenter, international trial comparing regorafenib versus placebo after imatinib/sunitinib failure | 199 (133 regorafenib versus 66 placebo patients) | PFS | Median PFS 4.8 months for regorafenib versus 0.9 months for placebo |
|
| Seifert et al. [34] | Analysis of 85 patients with GISTs to determine expression of immune checkpoint molecules and effects of combination IM + PD-1/PD-L1 blockade in murine GISTs | 85 (blood samples from patients with GISTs) | PD-1 receptor expression in T-cells of human GISTs | The PD-1 inhibitory receptors were upregulated on tumor-infiltrating T-cells compared with T-cells from matched blood |
| T-cell function in mice with GISTs treated with IM and PD-1/PD-L1 inhibitor | PD-1 expression on T-cells was highest in IM-treated human GISTs |
| PD-1/PD-L1 blockade in vivo had no efficacy alone but enhanced antitumor effects of IM by increasing T-cell effector function |
|
