Division of Neurosurgery, Department of Neurosciences, Reproductive and Odontostomatological Sciences, Università degli Studi di Napoli "Federico II", Naples, Italy; Skull Base and Pituitary Unit, Department of Neurosurgery B, Neurological Hospital Pierre-Wertheimer, Bron, 69677 Lyon, France.
Department of Neurosurgery, Helsinki University Central Hospital, Helsinki University, Meilahden tornisairaala, Haartmaninkatu 4 Rakennus 1, 00290 Helsinki, Finland.
Ann Endocrinol (Paris). 2023 Dec;84(6):727-733. doi: 10.1016/j.ando.2023.10.003. Epub 2023 Oct 19.
Craniopharyngioma (CP) is a neurosurgical challenge, due to location and to the substantial risk of morbidity associated with surgical resection. Recent advances in molecular research have identified a mutation signature in papillary craniopharyngiomas: BRAF V600E. This has led to targeted therapy, yielding positive results. Despite numerous studies of the pathophysiology of adamantinomatous craniopharyngioma, treatment options for molecular-based therapy are still lacking. The objective of our study was to provide an illustrative review of the literature on possible molecular targets in adamantinomatous craniopharyngioma and to report the case of a patient harboring an adamantinomatous craniopharyngioma deemed unsuitable for surgical resection, in which an anti-VEGF antibody was used to achieve tumor control.
An 84-year-old-man was referred to our department with a history of visual loss caused by recurrent infundibular adamantinomatous craniopharyngioma. A first surgical attempt to reduce the cystic portion of the tumor compressing the optic pathway failed. Due to rapid worsening of visual function, adjuvant therapy with bevacizumab was initiated before radiotherapy.
Neuroradiological and ophthalmological follow-up showed a decrease in tumor volume and improvement in visual function as early as 6 weeks after commencing therapy. These results were confirmed 3 months after commencement of chemotherapy. Radiotherapy was scheduled for long-term tumor control.
To the best of our knowledge, our case is the first in the literature in which targeted therapy using anti-VEGF was successfully used as a single agent to treat adamantinomatous craniopharyngioma, with favorable outcome in terms of tumor shrinkage and clinical improvement. These preliminary results may open new perspectives for the management of adamantinomatous craniopharyngioma. Validation of this approach requires additional clinical evidence.
颅咽管瘤（CP）是神经外科的一个挑战，由于其位置以及与手术切除相关的大量发病率风险。最近在分子研究方面的进展已经确定了乳头状颅咽管瘤的突变特征：BRAF V600E。这导致了靶向治疗，取得了积极的结果。尽管对成釉细胞瘤型颅咽管瘤的病理生理学进行了大量研究，但针对分子的治疗选择仍然缺乏。我们的研究目的是提供一个关于成釉细胞瘤型颅咽管瘤中可能的分子靶标的文献综述，并报告一例被认为不适合手术切除的成釉细胞瘤型颅咽管瘤患者的病例，该患者使用抗 VEGF 抗体来控制肿瘤。
一名 84 岁男性因复发性漏斗部成釉细胞瘤型颅咽管瘤导致视力丧失而被转至我们科室。第一次手术试图切除压迫视路的囊性部分肿瘤，但失败了。由于视力功能迅速恶化，在放射治疗前开始辅助贝伐单抗治疗。
神经放射学和眼科随访显示，在开始治疗后 6 周时肿瘤体积减小，视力功能改善。化疗开始 3 个月后证实了这些结果。为了长期控制肿瘤，计划进行放射治疗。
据我们所知，我们的病例是文献中首例成功使用抗 VEGF 作为单一药物治疗成釉细胞瘤型颅咽管瘤的病例，在肿瘤缩小和临床改善方面取得了良好的结果。这些初步结果可能为成釉细胞瘤型颅咽管瘤的治疗开辟新的视角。需要更多的临床证据来验证这种方法。
