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Ref Type Journal Article
PMID (36855200)
Authors Lang M, Longerich T, Anamaterou C
Title Targeted therapy with vemurafenib in BRAF(V600E)-mutated anaplastic thyroid cancer.
Journal Vol Issue Date Pages Journal Short Title
Thyroid research 16 1 2023 Mar 01 5 Thyroid Res
URL
Abstract Text 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%.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.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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Molecular Profile Treatment Approach
Gene Name Source Synonyms Protein Domains Gene Description Gene Role
Therapy Name Drugs Efficacy Evidence Clinical Trials
Drug Name Trade Name Synonyms Drug Classes Drug Description
Gene Variant Impact Protein Effect Variant Description Associated with drug Resistance
Molecular Profile Indication/Tumor Type Response Type Therapy Name Approval Status Evidence Type Efficacy Evidence References
BRAF V600E anaplastic thyroid carcinoma predicted - sensitive Vemurafenib Case Reports/Case Series Actionable In a clinical case study, first-line adjuvant therapy with Zelboraf (vemurafenib) resulted in a partial remission of the tumor and lymphatic and pulmonary metastases after 36 days of treatment in a patient with analplastic thyroid carcinoma harboring BRAF V600E, with a near-complete regression observed within 5 months of treatment initiation (PMID: 36855200). 36855200