Jimenez Camilo, Baudrand Rene, Uslar Thomas, Bulzico Daniel
Department of Endocrine Neoplasia and Hormonal Disorders, The University of Texas MD Anderson Cancer Center, 1400 Pressler Street, Unit 1461, Houston, TX 77030, USA.
Department of Endocrinology, Pontificia Universidad Catolica de Chile, Santiago, Chile.
Ther Adv Med Oncol. 2024 Nov 21;16:17588359241301359. doi: 10.1177/17588359241301359. eCollection 2024.
Pheochromocytomas and paragangliomas (PPGLs) are orphan tumors with the potential to spread to distant organs such as the lymph nodes, the skeleton, the lungs, and the liver. These metastatic tumors exhibit high rates of morbidity and mortality due to their frequently large tumor burden, the progression of the disease, and the excessive secretion of catecholamines that lead to cardiovascular disease and gastrointestinal dysmotility. Several molecular drivers responsible for the development of PPGLs have been described over the last 30 years. Although therapeutic options are limited, substantial progress has been made in the recognition of effective systemic therapies for these tumors. Successful clinical trials with radiopharmaceuticals such as high-specific-activity meta-iodobenzylguanidine and tyrosine kinase inhibitors such as cabozantinib and sunitinib have been recently published. This review will discuss the results of these studies and their impact on current clinical practices. In addition, this review will provide valuable information on how to design clinical trials to treat patients with metastatic PPGLs with novel medications.
嗜铬细胞瘤和副神经节瘤（PPGLs）是罕见肿瘤，有扩散至远处器官的可能，如淋巴结、骨骼、肺和肝脏。这些转移性肿瘤由于其通常较大的肿瘤负荷、疾病进展以及儿茶酚胺过度分泌导致心血管疾病和胃肠动力障碍，发病率和死亡率很高。在过去30年里，已描述了几种导致PPGLs发生的分子驱动因素。尽管治疗选择有限，但在识别这些肿瘤的有效全身治疗方面已取得了重大进展。最近发表了使用高比活度间碘苄胍等放射性药物以及卡博替尼和舒尼替尼等酪氨酸激酶抑制剂的成功临床试验。本综述将讨论这些研究的结果及其对当前临床实践的影响。此外，本综述将提供有关如何设计临床试验以用新型药物治疗转移性PPGLs患者的有价值信息。
