Researchers discovered that pemigatinib, a second-line treatment for cholangiocarcinoma, is associated with longer progression-free survival compared to systemic therapy, as published in Precision Oncology.

Cholangiocarcinoma is the most common primary malignancy of the bile duct and carries a poor prognosis. Most patients are diagnosed too late to be eligible for surgical resection. In such cases, the first-line systemic therapy is a combination of gemcitabine and cisplatin chemotherapy.

“Among patients with [biliary tract cancer], fusions or rearrangements involving the FGFR2 gene occur almost exclusively in patients with [intrahepatic cholangiocarcinoma],” the authors of the study wrote. The relationship between FGFR alterations and clinical outcomes has not been fully explored.

Continue Reading

Read more about cholangiocarcinoma complications

Bibeau and colleagues hence decided to recruit patients with advanced/metastatic cholangiocarcinoma harboring FGFR2 fusions or rearrangements who received first- or second-line systemic therapy. In addition, they also recruited patients with no FGF/FGFR genomic alterations or no FGR/FGFR genomic alterations. 

The primary endpoint of this study was progression-free survival. The researchers wanted to investigate the effects of systemic cancer therapy on progression-free survival. In addition, they compared the clinical outcomes of these therapies with pemigatinib, which is an FGFR 1-3 inhibitor.

The results demonstrated a median progression-free survival of 6.9 months in patients with FGFR2 fusions receiving pemigatinib as a second-line or later therapy. This is significantly higher than other second-line systemic therapies, which have a mean progression-free survival of 4.2 months. Other second-line chemotherapy regimens perform even more poorly, with some having a progression-free survival of just 3.2 months.

“Further research is warranted to prospectively characterize the disease course and response to chemotherapy in patients with and without FGFR2 fusions and, in the real-world setting, to characterize the second-line treatment outcomes of patients with FGFR2 alterations outside of a clinical trial,” Bibeau and colleagues concluded.


Bibeau K, Féliz L, Lihou CF, Ren H, Abou-Alfa GK. Progression-free survival in patients with cholangiocarcinoma with or without FGF/FGFR alterations: a FIGHT-202 post hoc analysis of prior systemic therapy responseJCO Precis Oncol. 2022;6:e2100414. doi:10.1200/PO.21.00414