Off-label afatinib with tegafur was attempted as a last resort option in a patient with advanced gallbladder cancer, achieving satisfactory outcomes. This study evaluated the benefits and toxicity of afatinib with tegafur in advanced biliary tract cancer (BTC). This retrospective study included patients who received afatinib combined with tegafur after failure of gemcitabine-based systemic therapy between Oct 2017 and Dec 2024 at the First Affiliated Hospital of Anhui Medical University. Progression-free survival (PFS), overall survival (OS), disease control rate (DCR), objective response rate (ORR), and treatment-related adverse events were evaluated. Cox regression analysis and subgroup analysis were used to assess the relationship between different factors and OS. Fifty-eight patients were included. Two patients achieved partial response, 28 achieved stable disease, and 28 achieved progressive disease, indicating a DCR of 51.7% and an ORR of 3.4%. Median PFS and OS were 4.0 and 6.6 months, respectively. The toxicity associated with afatinib and tegafur was generally acceptable. OS was prolonged in six patients with positive HER-2 and four patients with positive KRAS (G13D) mutations (median: 11.0 months) compared with those without mutations (median: 6.30 months) or unknown genetic status (median: 6.30 months) (P = 0.013). The multivariable Cox analysis showed that no factors were independently associated with OS (all P > 0.05). Afatinib with tegafur had an acceptable safety profile for advanced BTC after gemcitabine-based systemic therapy failure. The outcome of patients with low performance status and HER-2 or KRAS (G13D) mutations may be better than patients without the mutation.
Li et al. (Sun,) studied this question.