Background/Objectives: The recent discovery of BRAF mutation in papillary craniopharyngiomas opened new avenues for targeted therapies to control tumour growth, decreasing the need for invasive treatments and relative complications. The aim of this systematic review was to summarize the recent scientific data dealing with the use of targeted therapies in papillary craniopharyngiomas, as adjuvant and neoadjuvant treatments.Methods: The PRISMA guidelines were followed with searches performed in Scopus, MEDLINE, and Embase, following a dedicated PICO approach.Results: We included 21 pertinent studies encompassing 53 patients: 26 patients received BRAF inhibitors (BRAFi) as adjuvant treatment, while 25 received them as neoadjuvant treatment. In the adjuvant setting, BRAFi were used to treat recurrent tumours after surgery or adjuvant radiation therapy. The most common regimen combined dabrafenib (BRAFi) with trametinib (MEK1 and 2 inhibitor) in 81% of cases. The mean treatment length was 8.8 months (range 1.6 to 28 months) and 32% were continuing BRAFi. A reduction of tumour volume variable from 24% to 100% was observed at cerebral MRI during treatment and volumetric reduction ≥80% was described in 64% of cases. Once the treatment was stopped, adjuvant treatments were performed to stabilize patients in remission in 11 cases (65%) or when a progression was detected in three cases (12%). In four cases no further therapies were administered (16%). Mean follow-up after the end of targeted therapy was 17.1 months. As neoadjuvant regimen, 36% of patients were treated with dabrafenib and trametinib with a near complete radiological response in all the cases with a mean treatment of 5.7 months. The neoadjuvant use of verumafenib (BRAFi) and cometinib (MEK1 inhibitor) induced a near complete response in 15 patients (94%), with a median volumetric reduction between 85% and 91%. Ten patients did not receive further treatments. Side effects varied among studies. The optimal timing, sequencing, and duration of treatment of these new therapies should be established. Moreover, questions remain about the choice of specific BRAF/MEK inhibitors, the optimal protocol of treatment, and the strategies for managing adverse events.Conclusions: Treatment is shifting to a wider multidisciplinary management, where a key role is played by targeted therapies, to improve outcomes and quality of life for patients with BRAF-mutated craniopharyngiomas. Future, larger comparative trials will optimize their protocol of use and integration into multimodal strategies of treatment.
背景/目的：近期在乳头型颅咽管瘤中发现BRAF突变，为靶向治疗控制肿瘤生长开辟了新途径，从而减少了对侵入性治疗及相关并发症的需求。本系统综述旨在总结近期关于靶向治疗作为辅助及新辅助治疗在乳头型颅咽管瘤中应用的科学数据。 方法：遵循PRISMA指南，采用专门的PICO框架在Scopus、MEDLINE和Embase数据库中进行检索。 结果：共纳入21项相关研究，涵盖53例患者：其中26例患者接受BRAF抑制剂（BRAFi）作为辅助治疗，25例作为新辅助治疗。在辅助治疗中，BRAFi用于治疗手术后或辅助放疗后复发的肿瘤。最常见的方案是达拉非尼（BRAFi）联合曲美替尼（MEK1和2抑制剂），占81%的病例。平均治疗时长为8.8个月（范围1.6至28个月），32%的患者仍在继续接受BRAFi治疗。治疗期间脑部MRI显示肿瘤体积缩小幅度从24%到100%不等，其中64%的病例体积缩小≥80%。停止治疗后，11例（65%）患者为稳定缓解状态接受了辅助治疗，3例（12%）在检测到进展时进行了治疗。4例（16%）未接受进一步治疗。靶向治疗结束后的平均随访时间为17.1个月。在新辅助治疗方案中，36%的患者接受达拉非尼联合曲美替尼治疗，所有病例均达到接近完全的影像学缓解，平均治疗时长为5.7个月。新辅助使用维莫非尼（BRAFi）和考美替尼（MEK1抑制剂）在15例患者（94%）中诱导了接近完全缓解，中位体积缩小率在85%至91%之间。10例患者未接受进一步治疗。各研究报道的副作用存在差异。这些新疗法的最佳治疗时机、顺序和持续时间尚待确定。此外，关于特定BRAF/MEK抑制剂的选择、最佳治疗方案以及不良事件管理策略等问题仍有待解决。 结论：治疗正转向更广泛的多学科综合管理，其中靶向治疗在改善BRAF突变颅咽管瘤患者预后和生活质量方面发挥关键作用。未来更大规模的对比试验将优化其使用方案，并促进其融入多模式治疗策略。
肿瘤(癌症)患者之家