Mutation-based, neoadjuvant treatment for advanced anaplastic thyroid carcinoma

Area of Science:

• Oncology
• Thyroid Cancer Research
• Translational Medicine

Background:

• Anaplastic thyroid carcinoma (ATC) has a poor prognosis, necessitating novel treatment strategies.
• Mutation-based targeted therapies and immune checkpoint inhibitors (ICI) are emerging options for advanced ATC.

Purpose of the Study:

• To evaluate the efficacy of neoadjuvant mutation-based therapy in converting unresectable ATC to a resectable state.
• To optimize local tumor control and improve overall survival in advanced ATC patients.

Main Methods:

• Patients with advanced ATC received either lenvatinib plus pembrolizumab (BRAFV600E-negative) or dabrafenib plus trametinib (BRAFV600E-positive) for 4-6 weeks.
• Re-staging with FDG-PET/CT assessed tumor response; surgical resection was performed if regression was observed.
• Adjuvant mutation-based systemic therapy was continued post-surgery.

Main Results:

• Twelve patients were treated; eight showed tumor regression after neoadjuvant therapy.
• Surgical resection was performed in nine patients, with R0/R1 resection achieved in eight.
• Median progression-free survival was 3 months, and median overall survival was 9 months.

Conclusions:

• Neoadjuvant therapy is a promising approach for a subset of advanced ATC patients.
• Initial results warrant further investigation into its role in multimodal ATC management.