Molecular analysis for refractory rare cancers: Sequencing battle continues – learnings for the MOSCATO-01 study

Background: For patients with metastatic rare cancers, treatments are limited. How systematic tumor sequencing can improve therapeutic possibilities in this population? Patients and methods: Patients with rare cancer were identified in the MOSCATO-01 trial. Patients’ outcome was measured by progression-free survival (PFS) and overall survival (OS). Results: The most frequently identified histologic subypes were ovarian adenocarcinoma (N = 13), carcinoma of unknown primary (N = 11), and leiomyosarcoma (N = 10). Ninety-nine (39%) of them had at least one targetable cancer molecular alteration Forty-nine patients (50%) received the therapy proposed by the molecular tumor board, and 13 patients (26%, 95%CI 15–41%) achieved a PFS2/PFS1 > 1.3. The median PFS2 on matched treatment subgroup was 2.3 months (95% CI 1.8–3.6) and the median OS was 11.4 months (95% CI 9–15.5). Conclusions: The molecular screening of patients with refractory, metastatic rare cancers might increase the therapeutic options. Facilitating access strategy to molecular-driven clinical trials or agnostic-approved treatment is crucial.