Department of Endocrine Neoplasia and Hormonal Disorders, The University of Texas MD Anderson Cancer Center, Houston, TX, USA.
Clinical Cancer Genetics Program, The University of Texas MD Anderson Cancer Center, Houston, TX, USA.
Mol Cell Endocrinol. 2024 Oct 1;592:112344. doi: 10.1016/j.mce.2024.112344. Epub 2024 Aug 31.
Pheochromocytomas and paragangliomas (PPGL) are rare neuroendocrine tumors derived from chromaffin cells in the autonomic nervous system. Depending on their location, these tumors are capable of excessive catecholamine production, which may lead to uncontrolled hypertension and other life-threatening complications. They are associated with a significant risk of metastatic disease and are often caused by an inherited germline mutation. Although surgery can cure localized disease and lead to remission, treatments for metastatic PPGL (mPPGL)-including chemotherapy, radiopharmaceutical agents, multikinase inhibitors, and immunotherapy used alone or in combination- aim to control tumor growth and limit organ damage. Substantial advances have been made in understanding hereditary and somatic molecular signaling pathways that play a role in tumor growth and metastasis. Treatment options for metastatic disease are rapidly evolving, and this paper aims to provide a brief overview of the management of mPPGL with a focus on therapy options.
嗜铬细胞瘤和副神经节瘤(PPGL)是源自自主神经系统嗜铬细胞的罕见神经内分泌肿瘤。根据其位置,这些肿瘤能够过度产生儿茶酚胺,这可能导致不受控制的高血压和其他危及生命的并发症。它们与转移疾病的风险显著相关,并且通常由遗传性种系突变引起。尽管手术可以治愈局限性疾病并导致缓解,但转移性 PPGL(mPPGL)的治疗方法——包括化疗、放射性药物、多激酶抑制剂和免疫疗法单独或联合使用——旨在控制肿瘤生长并限制器官损伤。在理解遗传性和体细胞分子信号通路在肿瘤生长和转移中所起的作用方面已经取得了重大进展。转移性疾病的治疗选择正在迅速发展,本文旨在简要概述 mPPGL 的治疗方法,重点介绍治疗选择。