Thyroid : official journal of the American Thyroid Association 2017 11 22() doi 10.1089/thy.2017.0285
Patients with anaplastic thyroid cancer (ATC) have a dismal prognosis despite systemic cytotoxic chemotherapy. Our objective was to study the efficacy and safety of targeted therapy in ATC patients when used outside of a clinical trial.
This is a retrospective review from April 2015 to May 2016 at a single academic institution where we studied 16 ATC patients receiving targeted therapy outside of a clinical trial. Ten patients (8 BRAF wild-type and 2 BRAF V600E mutant) were started on lenvatinib and 6 with BRAF V600E mutated tumors received the combination of dabrafenib plus trametinib. Best response evaluated by RECIST v1.1, progression-free survival (PFS) and overall survival (OS). Adverse events were evaluated for safety.
The majority of patients were men (63%) and all had distant metastases or radiation-resistant primary disease at the time of treatment. Responses were as follows: In the entire cohort, 6/16 (38%) had partial response, 6/16 (38%) stable disease and 2/16 (12%) progressive disease. 2 patients (12%) died before restaging. Median follow up time was 11.8 months. Median PFS was 3.7 months (95% CI = 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 (95% CI = 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.
Targeted therapies, lenvatinib and dabrafenib plus trametinib (for BRAF V600E mutants), may provide clinical benefit in ATC patients who are unable to participate in clinical trials, with manageable toxicities.