Abstract / Summary
Abstract Objective: To analyze the clinical characteristics of immune checkpoint inhibitor-related thyroid dysfunction (irTD) in patients with biliary and ampullary malignancies, explore its pathogenesis, diagnostic difficulties and treatment strategies, and thereby provide references for the early recognition, accurate diagnosis and standardized management of this disease. Subjects and Methods: This is a retrospective study that collected clinical data of patients with biliary and ampullary tumors who received immune checkpoint inhibitor therapy at Xuhui District Central Hospital in Shanghai from January 2024 to December 2025, aiming to identify potential influencing factors of irTD. In addition, by integrating the clinical data of two typical cases of irTD, the clinical characteristics, diagnosis, treatment process, and prognosis of this disease were elaborated in detail. Results: A total of 15 patients with abnormal thyroid function were included in the study, and a case-control cohort was established at a 1:2 ratio. It was found that there were no significant differences in age, gender, tumor type, tumor surgical condition, tumor stage, drug type, diabetes, baseline free triiodothyronine, and baseline free thyroxine (P > 0.05). In contrast, statistically significant differences were observed in thyroid stimulating hormone (TSH, P = 0.009), anti-thyroid peroxidase antibody (TPOAb, P < 0.001), and anti-thyroglobulin antibody (TgAb, P = 0.001). Univariate and multivariate logistic regression analyses indicated that TPOAb was the most important independent risk factor affecting irTD. (P < 0.05). Abnormalities could significantly increase the risk of thyroid function abnormalities in patients receiving ICIs treatment. Analysis of two typical cases—one with drug-induced hypothyroidism complicated with pericardial effusion and the other with drug-induced hyperthyroidism complicated with hyperthyroid heart disease—demonstrated that the dynamic change trend of TPOAb could reflect the degree of thyroid damage and prognosis. It could also serve as an early warning indicator for irTD and a reference indicator for adjusting treatment regimens, which further optimizes the monitoring and treatment strategies for irTD Conclusion: The results of this study provide significant clinical implications for the management of irTD. In clinical practice, it is crucial to pay attention to TPOAb. For high-risk individuals, the frequency of monitoring thyroid function should be increased simultaneously, and early intervention should be carried out to avoid serious complications. During treatment, close monitoring of TPOAb changes provides a reliable reference for timely adjustment of treatment regimens, thereby improving the standardized management level of irTD in clinical practice.