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用达拉非尼和曲美替尼治疗不可切除的、BRAF突变的分化型甲状腺癌

Treatment of Unresectable , -Mutated Differentiated Papillary Thyroid Cancer With Dabrafenib and Trametinib.

作者信息

Bapat Neha, Ferraro Tatiana, Esper Layal, Joshi Arjun S, Haroun Faysal, Baldwin Chelsey K

机构信息

Department of Endocrinology, The George Washington University School of Medicine & Health Sciences, Washington, DC 20037, USA.

Division of Otolaryngology-Head and Neck Surgery, The George Washington University School of Medicine & Health Sciences, Washington, DC 20037, USA.

出版信息

JCEM Case Rep. 2024 Jul 30;2(8):luae112. doi: 10.1210/jcemcr/luae112. eCollection 2024 Aug.

Abstract

Complete surgical resection of differentiated papillary thyroid cancer (PTC) is associated with an excellent prognosis. However, for locally invasive PTC, disease-specific morbidity and mortality increases when microscopic margin negative resection (R0) or complete macroscopic resection (R1) is not feasible. Neoadjuvant dabrafenib and trametinib (DT) used in -positive, unresectable anaplastic thyroid cancer has allowed for R0 or R1 resection and improved survival rates. We demonstrate feasibility of using neoadjuvant DT in a patient with and -mutated PTC for whom R0/R1 resection was initially aborted due to predicted unacceptable morbidity. The patient was treated with neoadjuvant DT for 5 months, at which time disease was undetectable on imaging with near resolution on final pathology; however, subsequent rapid recurrence after discontinuation of neoadjuvant DT occurred. Neoadjuvant DT offers promise in future cohorts of patients with locally invasive and -mutated PTC for whom neoadjuvant therapy can reduce surgical morbidity while still allowing for R0/R1 resection.

摘要

分化型甲状腺乳头状癌(PTC)的完整手术切除与良好的预后相关。然而,对于局部侵袭性PTC,当显微镜下切缘阴性切除(R0)或完整的肉眼切除(R1)不可行时,疾病特异性发病率和死亡率会增加。用于BRAF V600E阳性、不可切除的间变性甲状腺癌的新辅助达拉非尼和曲美替尼(DT)已实现R0或R1切除并提高了生存率。我们证明了在一名BRAF和NRAS突变的PTC患者中使用新辅助DT的可行性,该患者最初因预计不可接受的发病率而中止了R0/R1切除。该患者接受了5个月的新辅助DT治疗,此时影像学检查未发现疾病,最终病理检查几乎完全缓解;然而,新辅助DT停药后随后迅速复发。新辅助DT在未来局部侵袭性BRAF和NRAS突变的PTC患者队列中具有前景,对于这些患者,新辅助治疗可以降低手术发病率,同时仍允许进行R0/R1切除。

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