Personalized treatment of 95 poorly differentiated thyroid cancer/anaplastic thyroid cancer in 2019-2023.

[BACKGROUND] Poorly differentiated thyroid cancer (PDTC) and anaplastic thyroid cancer (ATC) are both rare and aggressive thyroid cancers. Advances in targeted therapy and immunotherapy have changed treatment strategies and improved prognosis in these patients.

[METHODS] This single-center cohort study included patients diagnosed with locally advanced or metastatic PDTC/ATC at Fudan University Shanghai Cancer Center (FUSCC) between 2019 and 2023. Patients were either enrolled in clinical trials or received treatment based on clinical guidelines and expert consensus. Gene testing was conducted using next-generation sequencing of clinical samples.

[RESULTS] 95 patients were analyzed (PDTC = 34, ATC = 61). Median overall survival (OS) was 19.7 months for PDTC and 9.5 months for ATC (P = 0.478). Among 82 patients who underwent gene testing, the most frequent gene alterations in PDTC were BRAF (50.0%), TERT promoter (39.3%), and TP53 (25.0%) mutations; in ATC, they were TERT promoter (55.6%), BRAF (42.6%), and TP53 (25.9%) mutations. Compared with ATC patients, PDTC patients were more likely to receive best supportive care and less likely to be enrolled in clinical trials or treated with PD-1 inhibitors. The 1-year OS rates for PDTC/ATC patients receiving neoadjuvant therapy + surgery, systemic treatment, and supportive care only were 83.3, 51.2, and 5.7%, respectively (P < 0.001). After adjusting for covariates, neoadjuvant therapy + surgery (hazard ratio = 0.216, 95% confidence interval: 0.647-0.718, P = 0.012) was an independent predictor of superior OS.

[CONCLUSION] Despite the generally poor prognosis, aggressive treatment, particularly neoadjuvant therapy, has been shown to improve survival. Personalized treatment is crucial for optimizing treatment strategies for PDTC/ATC.