How effective is pemetinib in patients with advanced cholangiocarcinoma?

Cholangiocarcinoma is a rare but extremely aggressive cancer, and the treatment of advanced patients has always faced many challenges. Pemetinib, a potent and selective oral inhibitor targeting FGFR subtype 1/2/3, has shown significant efficacy in the treatment of advanced cholangiocarcinoma.

Pemetinib has demonstrated a high objective response rate (ORR) and significant improvement in survival when treating patients with advanced cholangiocarcinoma. The ORR confirmed based on the IRRC assessment reached 37%, including 4 cases had complete remission (CR=3.7%) and 36 cases had partial remission (PR=33.3%). This data brings new hope for treatment of patients with advanced cholangiocarcinoma.

After treatment with pemetinib, most patients showed signs of tumor shrinkage. The median duration of response (DOR) was 8.1 months, and the median progression-free survival (PFS) was 7.0 months, and the median overall survival (OS) was 17.5 months. These data are significantly better than traditional chemotherapy regimens, indicating that pemetinib has obvious advantages in controlling tumor growth and prolonging patient survival.

Pemetinib treatment has a good safety profile. The most common adverse event is hyperphosphatemia, but it is mostly mild to moderate and can be alleviated by adjusting dietary habits or taking anti-nausea drugs. Other adverse events such as joint pain, stomatitis, hyponatremia, etc. were also relatively few, and there were no treatment-related deaths.

For patients with advanced cholangiocarcinoma with FGFR2 fusions or rearrangements, the efficacy of pemetinib is more significant. After such patients receive pemetinib treatment, the ORR can reach 35.5%, the median PFS is 6.9 months, and the median OS is as high as 21.1 months, far exceeding the traditional chemotherapy regimen.