BRAF/MEK 抑制剂的靶向治疗 (TT) 已成为乳头状颅咽管瘤 (PCP) 的潜在治疗方法。然而，缺乏大群体的标准化数据。我们的研究旨在评估 BRAF/MEK 抑制剂对 PCP 患者的现实疗效和安全性。一项回顾性法国多中心研究，涉及 2019 年 4 月至 2023 年 7 月期间接受 BRAF/MEK 抑制剂组合达拉非尼和曲美替尼治疗的 BRAF V600E 突变 PCP 患者. 3 个月后以及 TT 期间最后一次随访时评估客观反应以及临床和安全性结果。 16 名患者（8 名女性，平均年龄 50.5 ± 15.75 岁），接受针对不可切除肿瘤的新辅助治疗 (NEO) (n = 6)、术后辅助治疗 (ADJ；n = 8) 或多模式治疗失败后的姑息治疗 (PAL) (n = 2)。 最后一次随访时（平均 7.6 ± 5.3）月），12 名患者表现出部分缓解，3 名患者表现出部分缓解，1 名患者病情保持稳定。 NEO、ADJ 和 PAL 组的平均体积减少分别为 88.9 ± 4.4%、73.3 ± 23.4% 和 91.8 ± 4.3%。靶向治疗解决了 5/5 名患者的头痛和 6/9 名患者的视力障碍； 2/3患者神经症状改善，1/4体重减轻，2/14内分泌功能恢复。62.5%的病例耐受性良好；不良事件导致 5 名患者停止治疗，3 名患者最终停止治疗。在这项研究中，94% 的患者对 TT 表现出部分缓解或更好。不良事件是可以接受的。需要进一步研究来建立标准化协议；然而，这些结果主张在侵入性 PCP 中采用 NEO 方法。© 作者 2024。由牛津大学出版社代表欧洲内分泌学会出版。版权所有。如需商业重复使用，请联系 reprints@oup.com 获取转载和转载的翻译权。所有其他权限都可以通过我们网站文章页面上的权限链接通过我们的 RightsLink 服务获得 - 如需了解更多信息，请联系journals.permissions@oup.com。

Targeted therapy (TT) with BRAF/MEK inhibitors has emerged as a potential treatment in papillary craniopharyngiomas (PCPs). However, standardized data on large cohorts are lacking. Our study aimed to assess real-life efficacy and safety of BRAF/MEK inhibition in patients with PCPs.Retrospective French multicenter study involving BRAF V600E-mutated PCP patients, treated with BRAF/MEK inhibitor combination dabrafenib and trametinib, from April 2019 to July 2023.Objective response and clinical and safety outcomes were assessed after 3 months and at the last available follow-up during TT.Sixteen patients (8 females, mean age 50.5 ± 15.75 years), receiving either neoadjuvant therapy (NEO) for non-resectable tumors (n = 6), post-surgical adjuvant therapy (ADJ; n = 8), or palliative therapy (PAL) following failure of multimodal treatment (n = 2), were included.At the last follow-up (mean 7.6 ± 5.3 months), 12 patients showed subtotal response, 3 exhibited partial response, and 1 maintained stable disease. Mean volume reduction was 88.9 ± 4.4%, 73.3 ± 23.4%, and 91.8 ± 4.3% in the NEO, ADJ, and PAL groups, respectively.Targeted therapy resolved headaches in 5/5 patients and visual impairment in 6/9; 2/3 patients had improved neurological symptoms, 1/4 presented weight loss, and 2/14 recovered endocrine function.Targeted therapy was well-tolerated in 62.5% of cases; adverse events led to treatment discontinuation in 5 patients and definitive discontinuation in 3 cases.In this study, 94% of patients showed partial response or better to TT. Adverse events were acceptable. Further research is needed to establish standardized protocols; however, these results advocate for a NEO approach in invasive PCPs.© The Author(s) 2024. Published by Oxford University Press on behalf of European Society of Endocrinology. All rights reserved. For commercial re-use, please contact reprints@oup.com for reprints and translation rights for reprints. All other permissions can be obtained through our RightsLink service via the Permissions link on the article page on our site—for further information please contact journals.permissions@oup.com.