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.
Authors
- Zhen Ye
- Ye Zhao (ORCID: https://orcid.org/0000-0001-5969-1203)
- Nidan Qiao (ORCID: https://orcid.org/0000-0001-5478-3555)
- Xiaoyun Cao (ORCID: https://orcid.org/0000-0002-7174-4731)
- Wenqiang He (ORCID: https://orcid.org/0000-0002-7475-8485)
- Hongying Ye (ORCID: https://orcid.org/0000-0002-9435-164X)
- Haixia Cheng (ORCID: https://orcid.org/0000-0002-1601-8673)
- Zhengyuan Chen (ORCID: https://orcid.org/0000-0001-9074-1678)
- Yuechao Zhao (ORCID: https://orcid.org/0000-0002-2732-2689)
- Xufang Gu (ORCID: https://orcid.org/0000-0001-7670-829X)
- Boyuan Yao (ORCID: https://orcid.org/0000-0001-6448-2588)
- Hong Chen (ORCID: https://orcid.org/0000-0001-5528-3923)
- Qilin Zhang (ORCID: https://orcid.org/0000-0002-0122-3201)
- Xiaorong Yan (ORCID: https://orcid.org/0009-0000-7619-5703)
- Rui Han (ORCID: https://orcid.org/0000-0003-2281-7129)
- Zengyi Ma (ORCID: https://orcid.org/0000-0002-6948-9049)
- Zhaoyun Zhang (ORCID: https://orcid.org/0000-0001-5366-1353)
- Buqing Liang
- Boyu Cai
- Ruofan Huang
- Yifei Yu (ORCID: https://orcid.org/0009-0006-2986-1682)
- Yongfei Wang
Institutions
- Brigham and Women's Hospital (US)
- Fujian Medical University (CN)
- Fudan University (CN)
- Loma Linda University (US)
- Massachusetts General Hospital (US)
- Shanghai Cancer Institute (CN)
- First Affiliated Hospital of Fujian Medical University (CN)
- Huashan Hospital (CN)
- Shanghai Center for Brain Science and Brain-Inspired Technology (CN)
- State Key Laboratory of Medical Neurobiology
Publication Details
- Journal
- Molecular Therapy Oncology
- Published
- 2025-11-06
- DOI
- https://doi.org/10.1016/j.omton.2025.201085
- Primary Topic
- Pituitary Gland Disorders and Treatments
- Type
- article
- Field-Weighted Citation Impact
- 0.00