malignant tumor

advanced stage cholangiocarcinoma

About

Cholangiocarcinoma originates from bile duct epithelial cells and is divided into intrahepatic cholangiocarcinoma (iCCA) and extrahepatic cholangiocarcinoma (eCCA). About 15-20% of iCCA patients have FGFR2 gene fusion, and about 20% have IDH1 mutations. Core dilemmas: ① Traditional chemotherapy (gemcitabine + cisplatin) has limited efficacy, and the median survival time is only about 12 months;② FGFR2 fusion and IDH1 mutations only cover some patients, and the problem of targeted drug resistance is prominent.

Features & Symptoms

  • No obvious symptoms in the early stage
  • Most of the time of diagnosis is advanced
  • The prognosis of intrahepatic/extrahepatic cholangiocarcinoma is extremely poor
  • Traditional chemotherapy has limited efficacy

Treatment Options

TreatmentDescriptionStagePrice Range
FGFR2 fusion targeting + IDH1 mutation targetingPemigatinib (an FGFR inhibitor) and Ivosidenib (an IDH1 inhibitor) have been approved by the FDA to provide precise treatment options for patients with corresponding mutations.FDA批准$150K - $300K/年
ADC+ immune checkpoint combinationAntibody-conjugated drugs such as Deruxtecan, combined with PD-1/PD-L1 inhibitors, have shown breakthrough efficacy in HER2-positive cholangiocarcinoma.Phase Ib/II$200K - $500K/疗程

Prognosis

Late median survival 6-12 months

Additional Notes

Notes: Genetic testing recommendations: It is recommended to test FGFR2 fusion, IDH1 mutation, HER2 amplification, and BRAF V600E mutation to guide targeted treatment choices; FGFR2 fusion: Pemigatinib and Futibatinib have been approved for FGFR2 fusion in treated bile duct cancer; IDH1 mutation: Ivosidenib has been approved for treated bile duct cancer with IDH1 mutation; liver function assessment: Child-Pugh classification needs to be evaluated before treatment, and treatment options need to be adjusted for patients with severe liver function impairment; biliary drainage: Patients with obstructive jaundice need biliary drainage to improve liver function before considering systematic treatment. Exclusive service commitment: The platform will allocate a dedicated medical team to each patient to assist in dismantling and properly deploying the following precautions throughout the process. When cross-state and cross-border diagnosis and treatment projects or drugs are involved, the platform will make overall arrangements for the entire docking; for disabled and semi-disabled patient groups, the platform will provide rush-free alternative solutions (including remote consultation, door-to-door sampling, direct drug delivery in the cold chain, etc.), to ensure that patients can get the same high-quality diagnosis and treatment resources without having to travel in person.; Study phase: Phase II clinical. Prognosis: Late median survival is 6-12 months.

Frequently Asked Questions

What is advanced stage cholangiocarcinoma?

Cholangiocarcinoma originates from bile duct epithelial cells and is divided into intrahepatic cholangiocarcinoma (iCCA) and extrahepatic cholangiocarcinoma (eCCA). About 15-20% of iCCA patients have FGFR2 gene fusion, and about 20% have IDH1 mutations. Core dilemmas: ① Traditional chemotherapy (gemcitabine + cisplatin) has limited efficacy, and the median survival time is only about 12 months;② FGFR2 fusion and IDH1 mutations only cover some patients, and the problem of targeted drug resistance is prominent.

What are the symptoms of advanced stage cholangiocarcinoma?

No obvious symptoms in the early stage,Most of the time of diagnosis is advanced,The prognosis of intrahepatic/extrahepatic cholangiocarcinoma is extremely poor,Traditional chemotherapy has limited efficacy

How is advanced stage cholangiocarcinoma treated?

FGFR2 fusion targeting + IDH1 mutation targeting: Pemigatinib (an FGFR inhibitor) and Ivosidenib (an IDH1 inhibitor) have been approved by the FDA to provide precise treatment options for patients with corresponding mutations.; ADC+ immune checkpoint combination: Antibody-conjugated drugs such as Deruxtecan, combined with PD-1/PD-L1 inhibitors, have shown breakthrough efficacy in HER2-positive cholangiocarcinoma.

What is the prognosis for advanced stage cholangiocarcinoma?

Late median survival 6-12 months