Department of Diabetes and Endocrinology, St Vincent's Hospital, Sydney, NSW 2010, Australia.
School of Clinical Medicine, University of New South Wales, Sydney, NSW 2052, Australia.
J Clin Endocrinol Metab. 2024 Oct 15;109(11):2986-2996. doi: 10.1210/clinem/dgae503.
Papillary craniopharyngioma (PCP) and adamantinomatous craniopharyngioma (ACP) are distinct, slow-growing tumors of the suprasellar region. Their location, composition, and biology have historically evaded successful surgical radiation and medical therapy. Meanwhile compromise of critical structures either by tumor or treatments increase morbidity, impacting patient and carer quality of life. There has been a paradigm shift in the management of PCP, stemming from the discovery of BRAFV600E mutation in its tumorigenesis. Such a treatment breakthrough may soon be the case for ACP, changing the landscape of craniopharyngioma management. We use a case of ACP partially responding to ERK inhibitor therapy to demonstrate chronicity of disease progression and discuss modern management strategies highlighting the importance of access to tumor agnostic clinical trials, and future directions.
颅咽管瘤(PCP)和造釉细胞瘤型颅咽管瘤(ACP)是鞍上区具有明显不同特征的缓慢生长的肿瘤。它们的位置、组成和生物学特性一直难以通过手术、放疗和药物治疗获得成功。同时,肿瘤或治疗引起的关键结构的损伤增加了发病率,影响了患者和照顾者的生活质量。PCP 的治疗模式发生了转变,这主要源于在其肿瘤发生过程中发现了 BRAFV600E 突变。对于 ACP 来说,这种治疗突破可能很快就会出现,从而改变颅咽管瘤的治疗格局。我们使用 ACP 对 ERK 抑制剂治疗部分反应的病例来证明疾病进展的慢性,并讨论现代管理策略,强调获得肿瘤不可知临床试验的重要性和未来方向。