Table 2.
Main data on treatment with new agent treatment in adults’ patients with DTF.
| Author | Study | Number of cases | Response rate | Toxicity | Outcome |
|---|---|---|---|---|---|
| Chug et al.38 | Phase 2, single arm, trial on imatinib | 51 cases | 6% | G3/4 toxicity: 10% | 12 month PFS: 66% |
| Penel et al.45 | Phase 2, single arm, trial on imatinib | 35 cases | 11% | G3/4 toxicity: 45% | 12 month PFS: 67% |
| Kasper et al.46 | Phase 2, single arm, trial on imatinib | 38 cases | 19% | G3/4 toxicity: 13% | 12 month PAR: 59% |
| Gounder et al.18 | Phase 3, double blind, randomized to sorafenib or placebo | 87 cases | 33% (95% CI: 20-48; sorafenib), 20% (95% CI: 8–38; placebo) | G3 toxicity: 29% (sorafenib), 14% (placebo); G4 toxicity: 4% (sorafenib) | 12 month PFS: 89% (95% CI: 80–99; sorafenib), 46% (95% CI: 32–67; placebo) |
| Agresta et al.8 | Retrospective, comparison between pazopanib and VBL-MTX | 37 cases | 29% (pazopanib), 13% (VBL-MTX) | G3/4 toxicity: 8% | No data on PFS |
| Toulmonde et al.24 | Randomized Phase 2, on pazopanib or VBL-MTX | 72 cases | 37% | G3/4 toxicity: 8% | 12 month PFS: 86% (95% CI: 71–93; pazopanib) and 79% (95% CI: 53–92; VBL-MTX) |
EFS event-free survival, PAR progression arrest rate, PFS progression-free survival, VBL-MTX vinblastine and methotrexate, CI confidential interval, G grade.