Table 1. Main published trials involving targeted therapy in endometrial cancer.
| Author | Study year | No. of patients | Cancer type | Compound | Pathway target | Clinical response |
|---|---|---|---|---|---|---|
| Ott et al. [17] | 2017 | 24 | Advanced/metastatic | Pembrolizumab | PD-L1 | PR: 3 patients |
| SD: 1 patient | ||||||
| ORR: 13% | ||||||
| PFS: 19% | ||||||
| OS: 68.8% | ||||||
| Makker et al. [18] | 2017 | 23 | Advanced/metastatic | Pembrolizumab | PD-L1 | ORR: 48% |
| Fleming et al. [21] | 2017 | 15 | Advanced | Atezolimumab | PD-L1 | ORR: 13% |
| Oza et al. [36] | 2011 | 34 (25 CT naïve + 29 CT treated) | Recurrent/metastatic | Temsirolimus | mTOR | CT-naïve group |
| PR: 14%, SD: 69%, PD 18% | ||||||
| CT-treated group | ||||||
| PR: 4%, SD: 48%, PD 48% | ||||||
| Slomovitz et al. [37] | 2010 | 28 | Recurrent CT-treated | Everolimus | mTOR | PR: 0% |
| SD: 43% | ||||||
| Oza et al. [40] | 2008 | 33 | Recurrent/metastatic | Erlotinib | EGFR | PR: 12% |
| SD: 46.9% | ||||||
| PD: 40.6% | ||||||
| Leslie et al. [41] | 2013 | 26 | Persistent/recurrent after at least one CT regimen | Gefitinib | EGFR | CR: 3.8% |
| SD: 26.9% | ||||||
| PFS (median): 1.8 months | ||||||
| OS (median): 7.1 months | ||||||
| Coleman et al. [42] | 2015 | 54 | Persistent/recurrent | Selumetinib | MEK 1/2 | CR: 1 patient |
| PR: 2 patients | ||||||
| SD: 13 patients | ||||||
| PFS (median): 2.3 months | ||||||
| OS (median): 8.5 months | ||||||
| Aghajanian et al. [67] | 2011 | 56 | Persistent/recurrent | Bevacizumab | VEGF | CR: 1 patient |
| PR: 6 patients | ||||||
| PFS (median): 4.2 months | ||||||
| OS (median): 10.5 months | ||||||
| Viswanathan et al. [68] | 2014 | 15 | Persistent/recurrent | Bevacizumab + IMRT | VEGF | 1-year PFS: 80% 3-year PFS: 67% |
| 1-year OS: 93% 3-year OS: 80% | ||||||
| Alvarez et al. [69] | 2013 | 49 | Persistent/recurrent | Bevacizumab + Temsirolimus | VEGF/mTOR | CR: 24.5% |
| PFS (median): 5.6 months | ||||||
| OS (median): 16.9 months | ||||||
| Simpkins et al. [70] | 2014 | 15 | Stage III/IV or recurrent EC | Bevacizumab + Paclitaxel/Carboplatin | VEGF | CR: 5 patients |
| PR: 6 patients | ||||||
| PFS (median): 18 months | ||||||
| OS (median): 58 months | ||||||
| Vergote et al. [72] | 2013 | 133 | Metastatic/unresectable endometrial cancer after 1 or 2 prior platinum-based treatments, ≤2 prior chemotherapies, and ECOG PS ≤2. | Lenvatinib | VEGFR1–3, FGFR1–4, RET, KIT, and PDGFRβ | CR + PR: 14.3% |
| PFS (median): 5.4 months | ||||||
| OS (median): 10.6 months | ||||||
| Bender et al. [73] | 2015 | 48 | Second or third line of therapy in EC patients with measurable disease | Cediranib | VEGF, PDGF, FGF | PR: 12.5 % |
| PFS (median): 3.65 months | ||||||
| OS (median): 12.5 months |
CR, clinical response; CT, chemotherapy; EC, endometrial cancer; ECOG Eastern Cooperative Oncology Group; EGFR, epidermal growth factor receptor; FGF, fibroblast growth factor; IMRT, intensity-modulated radiation therapy; MEK, mitogen-activated protein kinase kinase; mTOR, mammalian target of rapamycin; ORR, overall response rate; OS, overall survival; PD, progressive disease; PD-L1, programmed death-ligand 1; PDGF, platelet-derived growth factor; PFS, progression free survival; PR, partial response; PS, performance status; SD, stable disease; VEGF, vascular endothelial growth factor.