First report: PLATFORM study for precision treatment of rare tumors in China

The purpose of this study was to present the preliminary results of the PLATFORM Study, which aimed to evaluate the effectiveness of precision treatment for rare tumors in China. This study involved a phase II, open-label, non-randomized, multi-arm, single-center clinical trial. Patients with advanced rare solid tumors, who had not responded to standard treatment, were enrolled. The primary objective was to assess the safety and efficacy of targeted therapies in patients with actionable genetic alterations and immune checkpoint inhibitors in patients lacking actionable genetic alterations. Out of the 922 cases screened, 107 patients underwent mutation detection, with a final enrollment of 64 cases for the study. Among these, 26 cases received targeted therapy, and 38 cases underwent immunotherapy. The study encompassed over 40 types of rare tumors. The overall objective response rate (ORR) was 7.0%, with a disease control rate (DCR) of 70%. Targeted therapy showed a higher ORR of 17.8% and a DCR of 100%. The median progression-free survival (PFS) was 4 months overall, with targeted therapy showing a median PFS of 5 months and immunotherapy showing a median PFS of 3 months. In conclusion, from this preliminary analysis, targeted therapy within the precision medicine framework demonstrated promising therapeutic potential for rare tumors. However, monotherapy immunotherapy exhibited limited efficacy, highlighting the challenges in overcoming tumor-specific variations. These findings underscore the importance of further research and the exploration of combination therapies to improve outcomes for patients with rare tumors.