Department of Emergency and Organ Transplantation, Section of Internal Medicine, Endocrinology, Andrology and Metabolic Diseases, University of Bari Aldo Moro, Bari, Italy.
Endocrinology Unit, IRCCS Ospedale Policlinico San Martino, Genova, Italy.
Front Endocrinol (Lausanne). 2021 Apr 14;12:667784. doi: 10.3389/fendo.2021.667784. eCollection 2021.
Medullary thyroid carcinoma is a rare neuroendocrine neoplasm that originates from thyroid C cells. Surgery, with complete resection of the tumor, is the only curative approach. However, in most cases, the tumor recurs at locoregional or metastatic level. In this setting, the management remains challenging. In recent years, the immune checkpoint inhibitors have provided promise for changing the cancer treatment paradigm through the application of new approaches that enhance the body's natural antitumor defenses. The aim of this review is to summarize and discuss available data on efficacy and safety of the Food and Drug Administration-approved immune checkpoint inhibitors in patients with medullary thyroid carcinoma. After an extensive search, we found 7 useful data sources (one single-case report, one short article with very preliminary data, five ongoing registered clinical trials). Despite the lack of published evidence regarding the use of immune check point inhibitors, it must be considered that all the ongoing registered clinical trials saw first light in the last three years, thus indicating a growing interest of researchers in this field. Results coming from these trials, and hopefully, in the next future, from additional trials, will help to clarify whether this class of drugs may represent a new weapon in favor of patients with medullary thyroid carcinoma.
甲状腺髓样癌是一种罕见的神经内分泌肿瘤，起源于甲状腺 C 细胞。手术切除肿瘤是唯一的治愈方法。然而，在大多数情况下，肿瘤会在局部或远处转移水平复发。在这种情况下，治疗仍然具有挑战性。近年来，免疫检查点抑制剂通过应用新的方法增强了机体的天然抗肿瘤防御，为改变癌症治疗模式提供了希望。本综述的目的是总结和讨论美国食品和药物管理局批准的免疫检查点抑制剂在甲状腺髓样癌患者中的疗效和安全性数据。经过广泛搜索，我们找到了 7 个有用的数据源（一个单一病例报告，一个初步数据非常有限的短文，五个正在进行的注册临床试验）。尽管关于免疫检查点抑制剂的使用缺乏已发表的证据，但必须考虑到所有正在进行的注册临床试验都是在过去三年中首次进行的，这表明研究人员对这一领域的兴趣日益浓厚。这些试验的结果，以及希望在未来的试验中，将有助于阐明这类药物是否可能成为支持甲状腺髓样癌患者的新武器。
