无结缔组织型甲状腺癌（ATC）是一种罕见的甲状腺肿瘤，通常发生在老年患者身上。往往存在先前分化型甲状腺癌的病史，暗示着一种生物学进展。其临床特点是快速发病的局部浸润性颈部肿块。50%的患者在诊断时有转移灶。由于其不良预后，迅速确诊至关重要。对于无法手术切除或有转移性疾病的患者，多模式治疗（化疗和外照射放射治疗）的效果不佳，12个月总生存率不到20%。最近，在了解ATC致癌途径方面取得了重要进展，发现BRAF V600E突变是近40%病例中的驱动致癌基因。BRAF抑制剂达来那非（D）和MEK抑制剂曲美替尼（T）的联合应用在BRAF突变型ATC中显示出卓越的应答率，并已被认为是该领域的标准治疗。最近的研究表明，DT新辅助治疗后进行手术可实现80%的24个月总生存率。尽管这些方法改变了ATC的治疗策略，但对于BRAF野生型ATC仍然需要有效的治疗方法，并且对于大部分ATC的治疗缺乏高质量的证据。此外，在实际世界的情况下，及时获得多学科护理、分子测试和靶向治疗仍然是一个挑战。有必要制定健康政策，以确保ATC的专业治疗。对ATC分子生物学的不断扩展的认识，以及正在进行的临床试验，为进一步治疗选择的发展提供了希望。© 2023. The Author(s), under exclusive licence to Springer Science+Business Media, LLC, part of Springer Nature.

Anaplastic thyroid cancer (ATC) is an infrequent thyroid tumor that usually occurs in elderly patients. There is often a history of previous differentiated thyroid cancer suggesting a biological progression. It is clinically characterized by a locally invasive cervical mass of rapid onset. Metastases are found at diagnosis in 50% of patients. Due to its adverse prognosis, a prompt diagnosis is crucial. In patients with unresectable or metastatic disease, multimodal therapy (chemotherapy and external beam radiotherapy) has yielded poor outcomes with 12-month overall survival of less than 20%. Recently, significant progress has been made in understanding the oncogenic pathways of ATC, leading to the identification of BRAF V600E mutations as the driver oncogene in nearly 40% of cases. The combination of the BRAF inhibitor dabrafenib (D) and MEK inhibitor trametinib (T) showed outstanding response rates in BRAF-mutated ATC and is now considered the standard of care in this setting. Recently, it was shown that neoadjuvant use of DT followed by surgery achieved 24-month overall survival rates of 80%. Although these approaches have changed the management of ATC, effective therapies are still needed for patients with BRAF wild-type ATC, and high-quality evidence is lacking for most aspects of this neoplasia. Additionally, in real-world settings, timely access to multidisciplinary care, molecular testing, and targeted therapies continues to be a challenge. Health policies are warranted to ensure specialized treatment for ATC.The expanding knowledge of ATC´s molecular biology, in addition to the ongoing clinical trials provides hope for the development of further therapeutic options.© 2023. The Author(s), under exclusive licence to Springer Science+Business Media, LLC, part of Springer Nature.