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Updated: Feb 18, 2026
An Orthotopic Mouse Model of Anaplastic Thyroid Carcinoma
Published on: April 17, 2013
Real-World Experience with Targeted Therapy for the Treatment of Anaplastic Thyroid Carcinoma
Priyanka C Iyer1,2, Ramona Dadu1, Renata Ferrarotto3
11 Department of Endocrine Neoplasia and Hormonal Disorders, The University of Texas MD Anderson Cancer Center , Houston, Texas.
Background:
Patients with anaplastic thyroid cancer (ATC) have a dismal prognosis, despite systemic cytotoxic chemotherapy. The objective of this study was to investigate the efficacy and safety of targeted therapy in ATC patients when used outside of a clinical trial.
Methods:
This is a retrospective review from April 2015 to May 2016 at a single academic institution where 16 ATC patients receiving targeted therapy outside of a clinical trial were studied. Ten patients (eight BRAF wild type and two BRAFV600E mutant tumors) were started on lenvatinib, and six with BRAFV600E-mutated tumors received a combination of dabrafenib plus trametinib. Best response evaluated by RECIST v1.1, progression-free survival, and overall survival were determined. Adverse events were evaluated for safety.
Results:
The majority of patients (63%) were men, and all had distant metastases or radiation-resistant primary disease at the time of treatment. In the entire cohort, 6/16 (38%) had a partial response, 6/16 (38%) had stable disease, and 2/16 (12%) had progressive disease. Two (12%) patients died before restaging. Median follow-up time was 11.8 months. Median progression-free survival was 3.7 months [confidence interval 1.8-7.6] in the entire cohort, 2.7 months for lenvatinib, and 5.2 months for dabrafenib plus trametinib. Median OS was 6.3 months [confidence interval 1.8-7.6] for the entire cohort, 3.9 months for lenvatinib, and 9.3 months for dabrafenib plus trametinib. Adverse events were as expected and manageable.
Conclusions:
Targeted therapies, lenvatinib, and dabrafenib plus trametinib (for BRAFV600E mutants) may provide clinical benefit in ATC patients who are unable to participate in clinical trials, and toxicities are manageable.
Insights
Targeted therapies like lenvatinib and dabrafenib plus trametinib show clinical benefit for anaplastic thyroid cancer (ATC) patients outside clinical trials. These treatments offer manageable toxicities and potential survival improvements for this difficult-to-treat cancer.
Area of Science:
- Oncology
- Pharmacology
- Clinical Medicine
Background:
- Anaplastic thyroid cancer (ATC) presents a poor prognosis despite conventional chemotherapy.
- There is a critical need for effective treatments for ATC patients, particularly those ineligible for clinical trials.
Purpose of the Study:
- To evaluate the efficacy and safety of targeted therapies in anaplastic thyroid cancer patients treated outside of clinical trials.
- To assess the outcomes of lenvatinib and dabrafenib plus trametinib in a real-world ATC cohort.
Main Methods:
- Retrospective review of 16 ATC patients receiving targeted therapy between April 2015 and May 2016.
- Patients received either lenvatinib (BRAF wild type or V600E mutant) or dabrafenib plus trametinib (BRAF V600E mutant).
- Outcomes assessed included best response (RECIST v1.1), progression-free survival (PFS), overall survival (OS), and adverse events.
Main Results:
- 38% of patients achieved partial response, and 38% had stable disease.
- Median PFS was 3.7 months (lenvatinib: 2.7 months; dabrafenib/trametinib: 5.2 months).
- Median OS was 6.3 months (lenvatinib: 3.9 months; dabrafenib/trametinib: 9.3 months); adverse events were manageable.
Conclusions:
- Targeted therapies, including lenvatinib and dabrafenib plus trametinib, demonstrate clinical benefit in ATC patients outside of clinical trials.
- These treatments offer a viable option for anaplastic thyroid cancer patients with manageable toxicities.
- BRAF V600E mutation status influences treatment selection and potential outcomes with targeted agents.
