Targeted therapies in primary vaginal cancer

Primary vaginal cancer (PVC) accounts for 1–2% of all gynecological malignancies. Therefore, clinical trials lack and there is a paucity of approved medications beyond radiation or chemoradiation as main first line therapy. The GOG 240 and the Keynote 826 have shown significant improvements in survival in cervical cancer patients. Both concepts are approved medications for the treatment of advanced, recurrent or metastastic cervical cancer. However, the efficacy of Bevacizumab and Pembrolizumab and other Immunotherapies in PVC is mainly unknown. This study evaluates the efficacy and potential of targeted therapies and immunotherapies in patients with metastatic primary vaginal cancer, based on therapeutic strategies established for cervical cancer. In this retrospective study, 6 patients with metastatic PVC were treated with Carboplatin/Paclitaxel/Bevacizumab with or without Pembrolizumab. Due to Her2neu expression one patient was treated with Her2neu targeted therapy and one patient is on Nivolumab at present. 4 patients received Bevacizumab (Bev) in addition to Carboplatin (C) and Paclitaxel (Pac). Duration of response was 6 and 13 month and one patient is still on Bev. One patient progressed after 5 cycles of C/P/Bev. 2 Patients were treated with Pembrolizumab (P) mono after C/Pac/Bev. One out of 2 patients showed a duration of response of 36 month despite late stage metastatic disease. Local progress was controlled by adjunct local radiation therapy. The other patient progressed and died. Two patients were treated with C/Pac/Bev/P. One patient is still on therapy and one patient has died due to progressive disease. One patient is on Nivolumab with good response. These data provide a proof of concept to adopt therapeutic modalities from cervical cancer patients in PVC patients. In addition, our findings underscore the importance of collecting real-world data to guide clinical decision-making in metastatic primary vaginal cancer.