Roman-Gonzalez Alejandro, Jimenez Camilo
aDepartment of Endocrinology, Hospital Universitario San Vicente Fundacion-Universidad de Antioquia, Medellín, Colombia bDepartment of Endocrine Neoplasia and Hormonal Disorders, The University of Texas MD Anderson Cancer Center, Houston, Texas, USA.
Curr Opin Endocrinol Diabetes Obes. 2017 Jun;24(3):174-183. doi: 10.1097/MED.0000000000000330.
Pheochromocytomas and paragangliomas (PPGs) are rare neuroendocrine tumors. Over the last 15 years, substantial progress has been made toward understanding the clinical aspects and molecular origins of this disease. Nevertheless, predicting and managing malignancy remains the biggest challenge in clinical practice. The natural history of patients with malignant PPGs has not yet been described, and their prognosis varies. Currently, the diagnosis of malignant PPGs relies on the presence of metastases, by which time the disease is usually advanced. Better understanding of the clinical and molecular characteristics of patients with malignant PPGs has spurred several prospective clinical trials.
Several molecular targeted therapies, a novel radiopharmaceutical medication that targets the catecholamine transporter, and immunotherapy are under evaluation for the treatment of patients with malignant PPGs. Furthermore, the identification of clinical predictors of malignancy and survival has led to the first TNM staging classification for PPGs.
Prospective clinical trials are providing patients with therapeutic options beyond systemic chemotherapy. The knowledge derived from these trials and from the evaluation of the TNM staging in clinical practice will help to clarify how to most effectively treat malignant PPGs.
嗜铬细胞瘤和副神经节瘤（PPGs）是罕见的神经内分泌肿瘤。在过去15年中，在了解该疾病的临床方面和分子起源方面取得了重大进展。然而，预测和管理恶性肿瘤仍然是临床实践中的最大挑战。恶性PPGs患者的自然病史尚未描述，其预后各不相同。目前，恶性PPGs的诊断依赖于转移灶的存在，而此时疾病通常已进展。对恶性PPGs患者临床和分子特征的更好理解推动了几项前瞻性临床试验。
几种分子靶向疗法、一种靶向儿茶酚胺转运体的新型放射性药物以及免疫疗法正在评估用于治疗恶性PPGs患者。此外，恶性肿瘤和生存的临床预测指标的确定导致了PPGs的首个TNM分期分类。
前瞻性临床试验为患者提供了全身化疗以外的治疗选择。从这些试验以及临床实践中TNM分期评估中获得的知识将有助于阐明如何最有效地治疗恶性PPGs。
