Pembrolizumab in patients with rare ovarian cancers : analysis from AcSé Pembrolizumab phase II multicentric basket trial
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Rare ovarian cancer is a heterogeneous group of diseases with significant unmet medical needs and low chemotherapy sensitivity. In this context, the AcSé Pembrolizumab (NCT03012620) multicentric, non-randomized, phase II basket trial, assessing pembrolizumab monotherapy in rare tumors has been conducted. We report here the results from the rare ovarian cancer cohort. Primary outcome was the objective response rate (ORR) at week 12. Secondary endpoints included best ORR, clinical benefit rate, survival and safety. ORR at week 12 was 3.2% (95%CI, 0.4% to 11.2%), with one partial (PR) and one complete response (CR) in a patient with a teratoma with malignant transformation remaining in CR six years after initiating pembrolizumab. Stable disease (SD) was observed in 34% of patients, including one patient with carcinosarcoma maintaining a SD for 33 months. No new safety concerns and no deaths related to pembrolizumab were observed. Pembrolizumab in rare ovarian tumor subtypes showed limited ORR but promising long-term benefit in selected patients were observed.