Efficacy of BRAF-MEK Inhibitors in Chinese Papillary Craniopharyngioma Patients: A Retrospective Analysis

Introduction: Papillary craniopharyngioma (PCP), a rare brain tumor with BRAF V600E mutations, presents significant therapeutic challenges. While BRAF-MEK inhibitor therapy has shown promise in American and European populations, data on its effectiveness in Chinese PCP patients is limited. This study aims to evaluate the therapeutic outcomes and safety of BRAF-MEK inhibitors in a cohort of Chinese PCP patients.

Methods: A retrospective analysis was conducted on 30 PCP patients treated with BRAF-MEK inhibitors in China from January 2023 to October 2024. Tumor responses were assessed using changes in the maximum diameters of solid and cystic components at baseline, 1 month, 3 months, and the last available follow-up. Adverse events were recorded and analyzed.

Results: The mean reduction in maximum tumor diameter was 50.37±31.14% at 1 month, with no significant differences observed at 3 months (56.06±23.96%) or at the last follow-up (55.16±19.42%). Solid components showed a greater reduction compared to cystic parts. Neoadjuvant therapy resulted in significantly higher reduction rates (64.5±28.14%) than adjuvant therapy (36.24±28.34%). Adverse events, including fever and lymph node enlargement, were more frequent during the first 3 months but did not increase with longer treatment.

Conclusion : BRAF-MEK inhibitors demonstrated significant efficacy in Chinese PCP patients, particularly in the neoadjuvant setting. While initial tumor shrinkage was substantial, long-term therapy showed diminishing returns, indicating the potential need for subsequent local treatments such as surgery or radiation. Further research is needed to optimize the timing and duration of BRAF-MEK inhibitor therapy in this population.