Efficacy and safety of BRAF-MEK inhibitors in treatment of Chinese patients with papillary craniopharyngioma: A retrospective cohort study

Previous research supports the efficacy of BRAF-MEK inhibitor therapy for papillary craniopharyngiomas (PCPs). This retrospective study of 30 Chinese PCP patients evaluated tumor response and safety outcomes during treatment. All patients maintained 100% progression-free and overall survival throughout the follow-up. Tumor measurements revealed consistent reductions over time, with maximum diameter decreasing by 45.81% (31.01%-66.67%) at 1 month, 56.26% (46.67%-66.67%) at 3 months, and 59.50% (46.84%-69.94%) at the final follow-up. Similar patterns emerged for both solid and cystic components. Notably, neoadjuvant therapy demonstrated superior efficacy (60.35% reduction) compared to adjuvant approaches (42.90% reduction) at 1 month, particularly for solid tumor portions. Adverse events occurred more frequently during the initial 3-month period. These findings highlight the clinical value of BRAF-MEK inhibitor combinations for PCP treatment while suggesting potential limitations of extended targeted therapy duration.