What is the role of chemotherapy in the treatment of hepatocellular carcinoma (HCC)?

Updated: Jan 31, 2021
  • Author: Luca Cicalese, MD, FACS; Chief Editor: John Geibel, MD, MSc, DSc, AGAF  more...
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Systemic chemotherapy remains the mainstay of therapy for patients with advanced HCC who are not candidates for surgical resection, liver transplantation, or localized tumor ablation. Unfortunately, HCC is minimally responsive to systemic chemotherapy. Resistance to chemotherapy may be caused by the universal expression of the multidrug resistance gene protein on the surface of the malignant cells, leading to active efflux of chemotherapeutic agents.

Chemotherapy is usually not well tolerated and seems to be less efficacious in patients with HCC who have underlying hepatic dysfunction. Younger patients with well-compensated cirrhosis due to chronic hepatitis B or C infections have better outcomes with chemotherapy than older patients with alcoholic cirrhosis and other comorbid diseases.

The most active drugs tested for single-agent therapy have been doxorubicin, cisplatin, and fluorouracil. Response rates are about 10%, and treatment shows no clear impact on overall survival. [59] The combination of gemcitabine and oxaliplatin (GEMOX) helped to shrink large hepatomas to the point where some could be resected, according to a multicenter retrospective study in France. [60]

There is also no apparent benefit to chemotherapy in the adjuvant setting following resection or radiofrequency ablation (RFA). [57]  In an effort to provide care in this difficult population, various hormonal and biologic agents have been tried with minimal success, including tamoxifen, antiandrogens (eg, cyproterone and ketoconazole), interferon, interleukin (IL)-2, and octreotide. [61] Currently, liver-directed therapies (eg, resection, transplantation, and RFA) offer the only genuine hope for extended survival in patients with advanced HCC.

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