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

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Last updated: May 19, 2025View editorial policy

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From the Guidelines

Neoadjuvant chemotherapy is not recommended as a standard treatment approach for hepatocellular carcinoma (HCC) due to its limited efficacy and potential harm to patients with underlying liver disease. The current standard of care for resectable HCC focuses on upfront surgical resection when possible, or liver transplantation for selected patients, as outlined in the EASL-EORTC clinical practice guidelines 1. For patients with advanced disease, systemic therapy options include targeted therapies such as sorafenib or lenvatinib, or immunotherapy combinations like atezolizumab plus bevacizumab.

In specific cases where downstaging is needed to enable surgical intervention, locoregional therapies such as transarterial chemoembolization (TACE) or radioembolization are preferred over systemic neoadjuvant chemotherapy, as they have shown better response rates and outcomes in patients with HCC 1. The limited efficacy of traditional chemotherapy in HCC relates to the tumor's inherent chemoresistance and the compromised liver function in many patients with underlying cirrhosis, which restricts the tolerability of cytotoxic agents.

Some studies have explored the use of neoadjuvant therapy in combination with liver transplantation for HCC, with promising results in selected patients 1. However, this approach is not widely recommended and requires further research to establish its efficacy and safety. The most recent study on the topic, published in 2021, highlights the importance of liver transplantation in the treatment of HCC, but does not support the use of neoadjuvant chemotherapy as a standard approach 1.

Key points to consider in the treatment of HCC include:

  • Liver transplantation is a viable option for patients with HCC who meet the Milan criteria, with a 4-year patient survival rate of 75% and a recurrence-free survival rate of 83% 1
  • Systemic therapy options, such as targeted therapies and immunotherapy combinations, are available for patients with advanced disease
  • Locoregional therapies, such as TACE and radioembolization, are preferred over systemic neoadjuvant chemotherapy for downstaging and bridging to surgery or transplantation
  • Neoadjuvant therapy in combination with liver transplantation may be considered in selected patients, but requires further research to establish its efficacy and safety.

From the Research

Neoadjuvant Chemotherapy for Hepatitis Cellular Carcinoma

  • The use of neoadjuvant chemotherapy for hepatocellular carcinoma (HCC) has been explored in various studies, with mixed results 2, 3.
  • According to a 2004 study, approaches to prevent recurrence, including neoadjuvant therapy, have not proven to be beneficial 2.
  • However, a 2018 study found that hepatic arterial infusion chemotherapy (HAIC) may improve survival compared to sorafenib in patients with advanced HCC 4.
  • Another study from 2018 discussed the current status and future perspectives of chemotherapy for HCC, highlighting the importance of finding new therapeutic agents that are effective against HCC and can be tolerated by patients with underlying cirrhosis 3.

Treatment Options for HCC

  • The Barcelona Clinic Liver Cancer (BCLC) staging system is used to allocate treatment for HCC, with transarterial chemoembolization (TACE) being the first-line treatment for patients with intermediate stage HCC 5.
  • Other treatment options for HCC include surgical resection, radiofrequency ablation (RFA), and liver transplantation 2, 6.
  • Emerging therapies for HCC, such as gene- and immune-based therapies, are being explored and may offer new hope for patients with this disease 6.

Chemotherapy for HCC

  • Chemotherapy is one of the most important treatment modalities for advanced HCC, with sorafenib being the standard therapeutic agent 3.
  • Other chemotherapy agents, such as regorafenib and lenvatinib, have shown promise in clinical trials and may offer alternative treatment options for patients with HCC 3.
  • Hepatic arterial infusion chemotherapy (HAIC) has been shown to improve survival compared to sorafenib in patients with advanced HCC, and may be a viable treatment option for some patients 4.

Professional Medical Disclaimer

This information is intended for healthcare professionals. Any medical decision-making should rely on clinical judgment and independently verified information. The content provided herein does not replace professional discretion and should be considered supplementary to established clinical guidelines. Healthcare providers should verify all information against primary literature and current practice standards before application in patient care. Dr.Oracle assumes no liability for clinical decisions based on this content.

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