BRAF Inhibition in BRAFV600E-Positive Anaplastic Thyroid Carcinoma

• Published in: Journal of the National Comprehensive Cancer Network Vol. 14; no. 3; p. 249
• Main Authors: Lim, Annette M, Taylor, Graham R, Fellowes, Andrew, Cameron, Laird, Lee, Belinda, Hicks, Rodney J, McArthur, Grant A, Angel, Christopher, Solomon, Benjamin, Rischin, Danny
• Format: Journal Article
• Language: English
• Published: United States 01.03.2016
• Subjects: Aged; Antineoplastic Agents - therapeutic use; Biopsy, Fine-Needle; Disease Progression; Fatal Outcome; Female; Humans; Image-Guided Biopsy; Imidazoles - administration & dosage; Imidazoles - therapeutic use; Male; Middle Aged; Molecular Targeted Therapy; Mutation; Off-Label Use; Oximes - administration & dosage; Oximes - therapeutic use; Palliative Care; Positron-Emission Tomography; Protein Kinase Inhibitors - therapeutic use; Proto-Oncogene Proteins B-raf - antagonists & inhibitors; Proto-Oncogene Proteins B-raf - genetics; Thyroid Carcinoma, Anaplastic - drug therapy; Thyroid Carcinoma, Anaplastic - genetics; Thyroid Carcinoma, Anaplastic - pathology; Thyroid Neoplasms - drug therapy; Thyroid Neoplasms - genetics; Thyroid Neoplasms - pathology
• ISSN: 1540-1413
• DOI: 10.6004/jnccn.2016.0030

The efficacy of targeted monotherapy for BRAF(V600E)-positive anaplastic thyroid carcinomas (ATC) is not established. We report 2 cases of BRAF(V600E)-positive ATC treated with a BRAF inhibitor. A 49-year-old woman with a T4bN1bM0 ATC manifested symptomatic metastatic disease 8 weeks after radical chemoradiotherapy. Within 1 month of BRAF inhibitor monotherapy, a complete symptomatic response was observed, with FDG-PET scan confirming metabolic and radiologic response. Treatment was terminated after 3 months because of disease progression. The patient died 11 months after primary diagnosis. A 67-year-old man received first-line BRAF inhibitor for a T4aN1bM0 ATC. Within 10 days of treatment his pain had stabilized and his tumor had clinically halved in size. Stable disease was achieved for 11 weeks but the patient died 11 months after diagnosis because of disease progression. BRAF inhibitor monotherapy in ATC may obtain clinical benefit of short duration. Upfront combination therapy should be investigated in this patient subgroup.