(305) Targeted and Immunotherapy-Based Treatment Strategies for Anaplastic Thyroid Carcinoma: A Systematic Review of Clinical Evidence

Introduction: Anaplastic Thyroid Carcinoma (ATC) is a rare and highly malignant neoplasm with historically poor survival. Advances in molecular profiling and immunotherapy have introduced targeted treatment strategies. Given that ATC is frequently diagnosed and managed by otolaryngologists, a synthesis of prospective evidence is needed to inform care.

Methods: A PRISMA-guided systematic review was performed. Pubmed was searched to identify studies evaluating targeted and immunotherapy-based treatments for ATC. Eligible studies included prospective clinical trials and basket trials reporting ATC clinical outcomes. Excluded studies were preclinical, non-therapeutic, or did not report ATC-specific outcomes. Molecular profile, treatment, sample size, overall survival (OS), progression-free survival (PFS), and overall response rate (ORR) were extracted. Due to study heterogeneity, a qualitative synthesis was performed.

Results: Five studies comprising 99 patients with ATC were included. The predominant molecular profile was BRAF V600E (n=70). Among patients treated with dabrafenib plus trametinib (n=52), 12-month OS ranged from 51.7% to 80%, with a 12-month PFS of 79%. Combination anti–PD-L1 agents (n=42) reported median OS ranging from 6.21 to 43 months and median PFS ranging from 1.35 to 13.93 months. Encorafenib plus binimetinib (n=5) demonstrated a 12-month OS of 81.8% and 12-month PFS of 75%. ORR across treatments ranged from 14% to 80%. ROAR trial patient populations were considered as a single evidence base.

Conclusions: Targeted and immunotherapy-based treatments demonstrate clinically meaningful activity in ATC. While prospective data remain limited, reported survival and response outcomes exceed historical benchmarks and support the integration of molecular testing and multidisciplinary treatment planning.