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Published on: February 17, 2019
First report: PLATFORM study for precision treatment of rare tumors in China
Jiawei Zhou1, Peiwen Ma1, Yale Jiang1
1Clinical Cancer Center, National Cancer Center/National Clinical Research Center for Cancer/Cancer Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College, Beijing, China.
Abstract:
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.
Insights
Precision medicine shows promise for rare tumors in China. Targeted therapy demonstrated significant effectiveness, while immunotherapy had limited success in this preliminary study.
Area of Science:
- Oncology
- Precision Medicine
- Clinical Trials
Background:
- Rare tumors present significant challenges in treatment due to their heterogeneity and limited therapeutic options.
- The PLATFORM Study was initiated to investigate novel treatment strategies for advanced rare solid tumors in China.
Purpose of the Study:
- To evaluate the preliminary safety and efficacy of precision treatments, including targeted therapies and immune checkpoint inhibitors, for rare tumors.
- To assess outcomes in patients with advanced rare solid tumors refractory to standard treatments.
Main Methods:
- A phase II, open-label, non-randomized, multi-arm, single-center clinical trial.
- Enrolled patients with advanced rare solid tumors who had failed prior standard therapies.
- Administered targeted therapy for actionable genetic alterations and immune checkpoint inhibitors for those without.
Main Results:
- The study included 64 patients across over 40 rare tumor types, with 26 receiving targeted therapy and 38 immunotherapy.
- Overall objective response rate (ORR) was 7.0% and disease control rate (DCR) was 70%.
- Targeted therapy achieved a higher ORR of 17.8% and 100% DCR, with a median progression-free survival (PFS) of 5 months, compared to immunotherapy's 3-month median PFS.
Conclusions:
- Precision medicine, particularly targeted therapy, shows promising potential for treating rare tumors.
- Monotherapy immune checkpoint inhibitors demonstrated limited efficacy, suggesting challenges in overcoming tumor-specific resistance.
- Further research into combination therapies is crucial to improve outcomes for rare tumor patients.
