Carosi Francesca, Nigro Maria Concetta, Siepe Giambattista, Repaci Andrea, Locati Laura Deborah, Nannini Margherita
Department of Medical and Surgical Sciences, University of Bologna, Bologna 40138, Italy.
Radiation Oncology Unit, IRCCS Azienda Ospedaliero-Universitaria di Bologna, Bologna 40138, Italy.
JCEM Case Rep. 2025 Jul 28;3(9):luaf160. doi: 10.1210/jcemcr/luaf160. eCollection 2025 Sep.
Anaplastic thyroid cancer (ATC) is an aggressive and lethal malignancy with limited therapeutic options and poor prognosis. In recent years, the therapeutic arsenal of locally advanced or metastatic ATC has been expanded, with V-Raf murine sarcoma viral oncogene homolog B (mitogen-activated protein kinase kinase-MAPKK inhibitors for the subset of -mutant ATC. For wild-type ATC and without other actionable alterations, the most promising strategy is certainly immune checkpoint inhibitors, which have shown activities both in monotherapy or in combination regimens. However, access to novel treatments is heterogeneous worldwide for ATC patients, and activity of immunotherapy as a single agent is limited. We report the case of a patient with locally advanced wild-type ATC who achieved a near-complete and durable response following a multimodal treatment approach combining chemotherapy (carboplatin and paclitaxel), immunotherapy (pembrolizumab), and external beam radiotherapy. Pembrolizumab monotherapy was continued as maintenance therapy, and disease control was maintained for over 1 year. This case highlights the potential efficacy of chemo-immunotherapy in wild-type ATC, especially when a rapid tumor reduction is required. It supports the use of immune checkpoint inhibitors combined with cytotoxic agents as a viable therapeutic option in this aggressive tumor subtype.
间变性甲状腺癌(ATC)是一种侵袭性致死性恶性肿瘤,治疗选择有限且预后较差。近年来,局部晚期或转移性ATC的治疗手段有所扩展,对于特定突变型ATC亚群,可使用V-Raf鼠肉瘤病毒癌基因同源物B(丝裂原活化蛋白激酶激酶 - MAPKK抑制剂)。对于野生型ATC且无其他可靶向改变的患者,最有前景的策略当属免疫检查点抑制剂,其在单药治疗或联合治疗方案中均显示出活性。然而,全球范围内ATC患者获得新型治疗的机会存在差异,免疫疗法作为单一药物的活性有限。我们报告了一例局部晚期野生型ATC患者的病例,该患者在采用化疗(卡铂和紫杉醇)、免疫疗法(帕博利珠单抗)和外照射放疗的多模式治疗方法后,实现了近乎完全且持久的缓解。帕博利珠单抗单药治疗作为维持治疗持续进行,疾病控制维持了1年以上。该病例突出了化学免疫疗法在野生型ATC中的潜在疗效,尤其是在需要快速缩小肿瘤的情况下。它支持将免疫检查点抑制剂与细胞毒性药物联合使用,作为这种侵袭性肿瘤亚型的一种可行治疗选择。