Neuroendocrine Tumour Unit, ENETS Centre of Excellence, Royal Free Hospital, London, United Kingdom,
Neuroendocrine Tumour Unit, ENETS Centre of Excellence, Royal Free Hospital, London, United Kingdom.
Neuroendocrinology. 2019;109(4):287-298. doi: 10.1159/000499497. Epub 2019 Mar 12.
At present there is no clinical guideline or standardised protocol for the treatment of metastatic or invasive phaeochromocytoma and paraganglioma (collectively known as PPGL) due to the rarity of the disease and the lack of prospective studies or extended national databases. Prognosis is mainly determined by genetic predisposition, tumour burden, rate of disease progression, and location of metastases. For patients with progressive or symptomatic disease that is not amenable to surgery, there are various palliative treatment options available. These include localised therapies including radiotherapy, radiofrequency, or cryoablation, as well as liver-directed therapies for those patients with hepatic metastases (e.g., transarterial chemoembolisation) and systemic therapies including chemotherapy or molecular targeted therapies. There is currently intense research interest in the value of radionuclide therapy for neuroendocrine tumours, including phaeochromocytoma and paraganglioma, with either iodine-131 (131I)-radiolabelled metaiodobenzylguanidine or very recently peptide receptor radionuclide therapy (PRRT), and the most important contemporary clinical studies will be highlighted in this review. The studies to date suggest that PRRT may induce major clinical, biochemical, and radiological changes, with 177Lu-DOTATATE being most efficacious and presenting less toxicity than 90Y-DOTATATE. Newer combination therapies with combined radioisotopes, or combinations with chemotherapeutic agents, also look promising. Given the favourable efficacy, logistic, and safety profiles, we believe that PRRT will probably become the standard treatment for inoperable metastatic PPGL in the near future, but we await data from definitive randomised controlled trials to understand its role.
目前，由于疾病的罕见性以及缺乏前瞻性研究或扩展的国家数据库，尚无治疗转移性或侵袭性嗜铬细胞瘤和副神经节瘤（统称为 PPGL）的临床指南或标准化方案。预后主要取决于遗传易感性、肿瘤负担、疾病进展速度和转移部位。对于进展性或有症状且无法手术的患者，有多种姑息治疗选择。这些包括局部治疗，包括放射治疗、射频或冷冻消融，以及对于肝转移患者的肝导向治疗（例如，经动脉化疗栓塞）和全身治疗，包括化疗或分子靶向治疗。目前，对于放射性核素治疗神经内分泌肿瘤（包括嗜铬细胞瘤和副神经节瘤）的价值存在强烈的研究兴趣，包括碘-131（131I）标记的间碘苄胍或最近的肽受体放射性核素治疗（PRRT），本综述将重点介绍最重要的当代临床研究。迄今为止的研究表明，PRRT 可能诱导重大的临床、生化和影像学变化，其中 177Lu-DOTATATE 最有效，且毒性比 90Y-DOTATATE 小。新型联合治疗方案，包括联合放射性同位素，或联合化疗药物，也有前途。鉴于其有利的疗效、后勤和安全性，我们相信 PRRT 可能会在不久的将来成为不可手术的转移性 PPGL 的标准治疗方法，但我们仍在等待来自明确的随机对照试验的数据，以了解其作用。
