Complete Surgical Resection Following Neoadjuvant Dabrafenib Plus Trametinib in BRAF V600E -Mutated Anaplastic Thyroid Carcinoma
Open accessAbstract
Background: When achieved, complete surgical resection improves outcomes in anaplastic thyroid carcinoma (ATC). However, most ATC patients present with advanced inoperable disease, often with impending airway obstruction, increased hemorrhage risk, and significant dysphagia. Novel treatment strategies are critically needed to improve disease control and decrease locoregional morbidity. The objective of this study was to determine the feasibility and effectiveness of a neoadjuvant regimen by using dabrafenib with trametinib followed by surgical resection in patients with initially unresectable BRAF V600E -mutated ATC. Methods: Case series of six consecutive patients with BRAF V600E -mutated ATC diagnosed between January 2017 and February 2018. Pathologic confirmation of ATC was obtained before treatment. BRAF V600E status was ascertained via immunohistochemistry or sequencing of circulating tumor DNA. All patients received dabrafenib and trametinib (DT) followed by surgical resection and adjuvant chemoradiation. Three patients also received pembrolizumab. Results: Complete surgical resection was achieved in all patients. Histopathologic analyses of resected specimens showed high pathologic response rates with significantly decreased ATC viability and residual papillary thyroid carcinoma components. Overall survival at six months and one year was 100% and 83%, respectively. Locoregional control rate was 100%. Two patients died of distant metastases without evidence of locoregional disease at 8 and 14 months from diagnosis. The remaining four patients had no evidence of disease at the last follow-up. Conclusions: We report the first series in the literature of BRAF V600E -mutated ATC patients with locoregionally advanced disease treated with DT followed by surgical resection. We demonstrated feasibility of complete resection, decreased need for tracheostomy, high pathologic response rates, and durable locoregional control with symptom amelioration.
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Anaplastic thyroid cancer is characterized by median survival of several months, aggressive course, rapid metastasis in up to 50% at admission, and invasion of surrounding tissues.
“It is characterized by a median survival of several months, an aggressive course, rapid metastasis, in up to 50% of patients at the time of hospital admission, and invasion of surrounding tissues. [1,2,17]”
From Photodynamic Therapy for Thyroid Cancer (2025) · cites this paper as [17]
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Research works printed but not linked: 23
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Shonka, D. C., Ho, A., Chintakuntlawar, A. V., Geiger, J. L., Park, J. C., Seetharamu, N., Jasim, S., Abdelhamid Ahmed, A. H., Bible, K. C., Brose, M. S., et al. (2022) · Head & Neck · doi:10.1002/hed.27025 · Cited by 117 · see citing works
Self-citationTetzlaff, M. T., Adhikari, C., Lo, S., Rawson, R. V., Amaria, R. N., Menzies, A. M., Wilmott, J. S., Ferguson, P. M., Ross, M. I., Spillane, A. J., et al. (2020) · Annals of Oncology · doi:10.1016/j.annonc.2020.07.016 · Cited by 37 · see citing works
Photodynamic Therapy for Thyroid Cancer
Inglot, J., Strzelczyk, J. K., Bartusik-Aebisher, D., & Aebisher, D. (2025) · BioMed · doi:10.3390/biomed5010008 · Cited by 2 · see citing works
- Authors
- Wang, J. R., Zafereo, M. E., Dadu, R., Ferrarotto, R., Busaidy, N. L., Lu, C., Ahmed, S., Gule-Monroe, M. K., Williams, M. D., Sturgis, E. M., Goepfert, R. P., Gross, N. D., Lai, S. Y., Gunn, G. B., Phan, J., Rosenthal, D. I., Fuller, C. D., Morrison, W. H., Iyer, P., & Cabanillas, M. E.
- Affiliations
- The University of Texas MD Anderson Cancer Center (US)
- Corresponding author
- Cabanillas, Maria E.
- ORCID
- Wang, Jennifer R.
- Zafereo, Mark E.
- Dadu, Ramona
- Ferrarotto, Renata
- Busaidy, Naifa L.
- Lu, Charles
- Ahmed, Salmaan
- Gule-Monroe, Maria K.
- Williams, Michelle D.
- Sturgis, Erich M.
- Goepfert, Ryan P.
- Gross, Neil D.
- Lai, Stephen Y.
- Gunn, Gary Brandon
- Phan, Jack
- Rosenthal, David I.
- Fuller, Clifton David
- Morrison, William H.
- Iyer, Priyanka
- Cabanillas, Maria E.
- Venue
- Thyroid®
- Publisher
- SAGE Publications
- Published
- 2019-08
- Volume
- 29
- Issue
- 8
- Pages
- 1036-1043
- ISSN
- 1050-7256 (ISSN-L)
- 1557-9077
- PMC
- PMC6707029
- OpenAlex
- W2963699363
- Cited by
- 230
- References
- 0
- Citations by year
- 2026: 19 · 2025: 27 · 2024: 30 · 2023: 38 · 2022: 41 · 2021: 32 · 2020: 35 · 2019: 8
- FWCI
- 23.53
- Type
- journal article
- Language
- en
- Open access
- hybrid
- doi.org
- Licence
- Locations
- Thyroid (publishedVersion, cc-by)
- Europe PMC (PubMed Central) (publishedVersion, cc-by)
- PubMed Central (publishedVersion, cc-by)
- PubMed (publishedVersion)
- Funders
- National Cancer Institute (P30CA016672)
- Topics
- Health Sciences › Medicine › Endocrinology, Diabetes and Metabolism › Thyroid Cancer Diagnosis and Treatment
- Health Sciences › Medicine › Surgery › Thyroid and Parathyroid Surgery
- Health Sciences › Medicine › Surgery › Head and Neck Anomalies
- Keywords
- anaplastic thyroid carcinoma, BRAF V600E mutation, dabrafenib, trametinib, neoadjuvant therapy, R0 resection, unresectable disease, locoregional control, pathological response, pembrolizumab, adjuvant chemoradiotherapy, tracheostomy avoidance
- MeSH terms
- Aged, Antineoplastic Combined Chemotherapy Protocols, Female, Humans, Imidazoles, Male, Middle Aged, Mutation, Neoplasm Recurrence, Local, Neoplasm Staging, Oximes, Pyridones, Pyrimidinones, Survival, Thyroid Neoplasms, Thyroidectomy, Disease-Free Survival, Neoadjuvant Therapy, Neck Dissection, Proto-Oncogene Proteins B-raf, Chemoradiotherapy, Adjuvant, Antibodies, Monoclonal, Humanized, Thyroid Carcinoma, Anaplastic
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