Department of Endocrine Neoplasia and Hormonal Disorders, The University of Texas MD Anderson Cancer Center, Houston, Texas, USA.
Department of Endocrinology and Diabetes, Universidad de Antioquia, Medellin, Colombia.
Endocr Relat Cancer. 2020 Jul;27(7):R239-R254. doi: 10.1530/ERC-20-0043.
Metastatic pheochromocytomas and paragangliomas are rare, highly vascular tumors that spread primarily to the lymph nodes, skeletal tissue, lungs, and liver. Tumor morbidity is related to their size, location, hormonal activity, vascular nature, and rate of progression. Systemic therapies for this indication are limited. Only high-specific-activity iodine-131 metaiodobenzylguanidine is approved in the Unites States for treatment of these patients, and not all patients are candidates for this radiopharmaceutical. Antiangiogenic medications are currently being evaluated in prospective clinical trials for patients with metastatic pheochromocytomas and paragangliomas, and preliminary results have been encouraging. Antiangiogenic medications frequently offer antineoplastic effects with sometimes durable responses. However, cardiovascular toxicity and the development of tumor resistance may limit their efficacy. Experience derived from clinical trials is being used to identify mechanisms to effectively improve drug toxicity and possibly prevent the emergence of resistance. Therefore, antiangiogenic medications represent a therapeutic option for patients with metastatic pheochromocytomas and paragangliomas. Furthermore, in the world of oncology, there is strong scientific interest in the development of clinical trials that combine antiangiogenic medications with other modalities such as immunotherapy, radiopharmaceuticals, and hypoxia inhibitors since these combinations may substantially enhance clinical outcomes, including survivorship. In this review, we examine the progress made to date on antiangiogenic treatments for patients with metastatic pheochromocytomas and paragangliomas.
转移性嗜铬细胞瘤和副神经节瘤是罕见的高度血管肿瘤,主要扩散到淋巴结、骨骼组织、肺部和肝脏。肿瘤的发病率与肿瘤的大小、位置、激素活性、血管性质和进展速度有关。针对这种适应症的系统治疗方法有限。只有高比活度碘-131 间碘苄胍在美国被批准用于治疗这些患者,但并非所有患者都适合使用这种放射性药物。抗血管生成药物目前正在前瞻性临床试验中评估用于转移性嗜铬细胞瘤和副神经节瘤患者,初步结果令人鼓舞。抗血管生成药物通常具有抗肿瘤作用,有时可获得持久缓解。然而,心血管毒性和肿瘤耐药性的发展可能限制其疗效。从临床试验中获得的经验正在用于确定有效改善药物毒性并可能预防耐药性出现的机制。因此,抗血管生成药物代表了转移性嗜铬细胞瘤和副神经节瘤患者的一种治疗选择。此外,在肿瘤学领域,科学界强烈关注将抗血管生成药物与免疫疗法、放射性药物和缺氧抑制剂等其他方法相结合的临床试验的开发,因为这些组合可能显著提高临床结果,包括生存率。在这篇综述中,我们检查了迄今为止在转移性嗜铬细胞瘤和副神经节瘤患者的抗血管生成治疗方面取得的进展。