1Division of Medical Oncology, Department of Medicine.
2Department of Neurologic Surgery.
J Natl Compr Canc Netw. 2020 Dec 2;18(12):1590-1595. doi: 10.6004/jnccn.2020.7624. Print 2020 Dec.
Craniopharyngiomas are rare tumors that arise in the suprasellar region of the brain and are known for their aggressive nature despite their WHO grade I. This is due to the complex neuroanatomy of the sellar/suprasellar region and their proximity to the optic nerve apparatus, hypothalamic-pituitary tract, and other critical neuroanatomical structures. Definitive treatment is based on a multidisciplinary approach and often involves a combination of surgical, radiation, and medical therapy. However, there is high morbidity associated with surgery and RT due to the complex neuroanatomy of this region. Recently, BRAFV600E somatic mutation has been identified in most papillary craniopharyngiomas. This discovery has led to the novel use of RAF pathway inhibitors to treat these tumors. We report the successful use of dabrafenib (BRAF inhibitor) and trametinib (mitogen-activated protein kinase kinase inhibitor) in the neoadjuvant setting followed by definitive stereotactic radiosurgery. We propose an algorithm based on available literature on the integration of targeted therapy in the management of papillary craniopharyngiomas. Our observations, together with prior case reports, advocate the incorporation of targeted therapy for unresectable craniopharyngiomas and reinforce that treatment with dual-targeted therapy is safe and effective.
颅咽管瘤是一种罕见的肿瘤，起源于脑的鞍上区，尽管其组织学分级为 I 级，但具有侵袭性。这是由于鞍上区的复杂神经解剖结构及其与视神经装置、下丘脑-垂体束和其他关键神经解剖结构的接近。明确的治疗方法基于多学科方法，通常涉及手术、放疗和药物治疗的联合应用。然而，由于该区域的复杂神经解剖结构，手术和放疗相关的发病率很高。最近，大多数乳头状颅咽管瘤中都发现了 BRAFV600E 体细胞突变。这一发现导致了 RAF 通路抑制剂用于治疗这些肿瘤的新方法。我们报告了在新辅助治疗中使用达布拉非尼（BRAF 抑制剂）和曲美替尼（丝裂原活化蛋白激酶激酶抑制剂），然后进行确定性立体定向放射外科治疗的成功案例。我们根据现有文献提出了一种关于将靶向治疗整合到颅咽管瘤管理中的算法。我们的观察结果与之前的病例报告一起，提倡将靶向治疗用于不可切除的颅咽管瘤，并强调双重靶向治疗是安全有效的。
