Targeted therapy with vemurafenib in BRAF(V600E)-mutated anaplastic thyroid cancer.

IF 1.9 Q3 ENDOCRINOLOGY & METABOLISM Thyroid Research Pub Date : 2023-03-01 DOI:10.1186/s13044-023-00147-7

Matthias Lang, Thomas Longerich, Chrysanthi Anamaterou

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引用次数: 3

Abstract

Background: Anaplastic thyroid cancer (ATC) is one of the most aggressive malignancies, representing less than 5% of all thyroid carcinomas. Τhe median survival is limited to months due to the resistance of ATC to surgery, radioiodine therapy, radiotherapy and chemotherapy. This review will cover novel agents involving several cellular signaling pathways including the BRAF pathway. The BRAF inhibitor vemurafenib improves survival among patients with metastatic melanoma, hairy-cell leukemia and intracranial neoplasms with BRAF gene mutations. The frequency of a BRAF (V600E) mutation in ATC is about 25%.

Case presentation: We report the first case of a marked partial response to adjuvant first line monotherapy with vemurafenib in BRAF V600E-mutated ATC. The 78-year-old man showed a sustained response for 7 months, thereafter scans revealed progressive disease and the patient died 10 months after first diagnosis. This case report is accompanied by a comprehensive review of current strategies and tools for ATC treatment.

Conclusions: This case and the review of current data confirm the benefit of BRAF inhibition in BRAF-mutated ATC, limited by acquired resistance to targeted therapy.

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vemurafenib靶向治疗BRAF(V600E)突变的间变性甲状腺癌。

背景:间变性甲状腺癌(ATC)是最具侵袭性的恶性肿瘤之一，占所有甲状腺癌的不到5%。Τhe由于ATC对手术、放射性碘治疗、放疗和化疗的耐药性，中位生存期限制为数月。本综述将涵盖涉及几种细胞信号通路的新型药物，包括BRAF通路。BRAF抑制剂vemurafenib可提高BRAF基因突变的转移性黑色素瘤、毛细胞白血病和颅内肿瘤患者的生存率。ATC中BRAF (V600E)突变的频率约为25%。病例介绍:我们报告了第一例BRAF v600e突变ATC患者对vemurafenib辅助一线单药治疗有显著部分反应的病例。这名78岁的男子在7个月的时间里表现出持续的反应，随后的扫描显示疾病进展，患者在首次诊断后10个月死亡。本病例报告附有对目前ATC治疗策略和工具的全面审查。结论:该病例和对现有数据的回顾证实了BRAF抑制对BRAF突变的ATC的益处，受靶向治疗获得性耐药的限制。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

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