Mormando Marilda, Lauretta Rosa, Puliani Giulia, Bianchini Marta, Spoltore Maria Elena, Appetecchia Marialuisa
Oncological Endocrinology Unit, IRCCS Regina Elena National Cancer Institute, Via Elio Chianesi 53, 00144 Rome, Italy.
Section of Medical Pathophysiology, Food Science and Endocrinology, Department of Experimental Medicine, Sapienza University of Roma, 00185 Rome, Italy.
Biomedicines. 2024 Dec 23;12(12):2923. doi: 10.3390/biomedicines12122923.
: The current possible treatments of advanced medullary carcinoma (MTC) include different drugs belonging to the class of tyrosine kinase inhibitors (TKIs): vandetanib, cabozantinb, and selpercatinib. Although the effects of these TKIs have been well described in clinical trials, the real-practice evidence of the effectiveness and safety of these treatment is scant. This real-world case series aims to describe a niche of patients with advanced MTC treated with more than one TKI by focusing on treatment responses and any reported adverse events (AEs) and to provide additional insight on the individualized approach to the management of metastatic MTC. : Five patients with a diagnosis of metastastic MTC, treated with at least two different molecules of TKIs, were retrospectively selected. : Three patients obtained a partial response (one with cabozantinb, one with selpercatinib, and one with vandetanib), and two patients obtained disease stability (both of them treated with all three TKIs, the first two lines discontinued for AEs). The AE profile agreed with the known clinical trials AEs except for non-neoplastic ascites related to selpercatinib and lung cavitations of non-neoplastic tissue related to cabozantinb. The latter was an AE never described so far in patients receiving TKIs. : The best management of MTC relies on an individualized approach, keeping in mind and dealing with the potential toxicity in order to minimize the treatment withdrawal.
目前晚期髓样癌(MTC)的可能治疗方法包括不同的酪氨酸激酶抑制剂(TKIs)类药物:凡德他尼、卡博替尼和塞尔帕替尼。尽管这些TKIs的疗效在临床试验中已有详细描述,但这些治疗方法有效性和安全性的实际应用证据却很少。这个真实世界病例系列旨在通过关注治疗反应和任何报告的不良事件(AE)来描述接受不止一种TKI治疗的晚期MTC患者的情况,并为转移性MTC的个体化管理方法提供更多见解。
回顾性选择了5例诊断为转移性MTC且接受至少两种不同TKIs分子治疗的患者。
3例患者获得部分缓解(1例使用卡博替尼,1例使用塞尔帕替尼,1例使用凡德他尼),2例患者病情稳定(均接受了所有三种TKIs治疗,前两线治疗因AE停药)。AE情况与已知的临床试验AE一致,除了与塞尔帕替尼相关的非肿瘤性腹水和与卡博替尼相关的非肿瘤组织肺空洞。后者是接受TKIs治疗的患者中迄今为止从未描述过的AE。
MTC的最佳管理依赖于个体化方法,牢记并处理潜在毒性,以尽量减少治疗中断。