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Published on: May 17, 2024
Systemic Treatment: Expecting Further Success
Alessia Gazzola1, Maria Reig, Roberto Di Donato
1Barcelona Clinic Liver Cancer (BCLC) Group, Liver Unit, Hospital Clinic, IDIBAPS, University of Barcelona, Barcelona, Spain.
Abstract:
From its approval in 2008, sorafenib is the recommended treatment option for advanced-stage patients and its safety and efficacy has been confirmed by several studies. However, its mechanism of action is not completely understood and many efforts have been dedicated to investigating possible treatment response predictors. Dermatological adverse events occurring within the first 2 months of treatment are predictors of longer survival, while the same role for hypertension and diarrhea still needs a prospective confirmation. This association is opposite to the strategy of starting at a low dose as it may imply suboptimal drug exposure. In case of radiological progression, the appearance of new extrahepatic metastasis or vascular invasion significantly worsens life expectancy if compared to other patterns of progression. To date no genetic or biologic marker is available to predict response, even if some encouraging results have been reported by the study of polymorphism of VEGF and its receptor. Currently, data are conflicting about the possible predictive role of α-fetoprotein. Due to failure or the progression of therapies for earlier evolutionary stages (BCLC B) some patients in such a clinical profile may be treated with sorafenib. Indeed, almost 50% of the sorafenib-treated patients belong to this class. Patients with severely decompensated liver disease (jaundice, ascites in need of intense diuretic therapy/paracentesis) may not benefit from treatment. The use of sorafenib in the waiting list for liver transplantation is controversial, while its use at an advanced age requires careful evaluation of existing comorbidities that may increase the risk of adverse events. Many strides have been made in the field of hepatocellular carcinoma systemic therapy, and many remain to be realized. Considering the disappointing results of the trials conducted on new agents, a more dynamic interpretation of events together with the development of new strategies is key to enriching new and hopefully more successful trials.
Insights
Sorafenib is a recommended treatment for advanced liver cancer, but its effectiveness can be predicted by early side effects like skin issues. Further research is needed for other predictors and treatment strategies.
Area of Science:
- Hepatocellular Carcinoma Research
- Oncology
- Pharmacology
Background:
- Sorafenib, approved in 2008, is a standard treatment for advanced hepatocellular carcinoma (HCC).
- Its mechanism of action and reliable predictors of treatment response remain areas of active investigation.
- Current research explores various factors, including adverse events and biomarkers, to personalize HCC therapy.
Purpose of the Study:
- To review the current understanding of sorafenib's role in advanced HCC treatment.
- To identify potential predictors of treatment response and survival.
- To discuss challenges and future directions in HCC systemic therapy.
Main Methods:
- Review of existing literature on sorafenib in advanced HCC.
- Analysis of factors influencing treatment outcomes, including adverse events and biomarkers.
- Discussion of patient selection criteria and treatment strategies.
Main Results:
- Early dermatological adverse events within two months predict longer survival.
- Hypertension and diarrhea require further prospective validation as survival predictors.
- Radiological progression patterns, particularly new extrahepatic metastasis or vascular invasion, significantly impact prognosis.
- No definitive genetic or biologic markers currently predict response, though VEGF pathway polymorphisms show promise.
- Alpha-fetoprotein's predictive role remains conflicting.
- Patients with severely decompensated liver disease may not benefit from sorafenib.
- Use in liver transplant candidates and elderly patients requires careful consideration.
Conclusions:
- Sorafenib remains a key therapy for advanced HCC, but personalized treatment requires better predictive markers.
- Early identification of predictive factors, like specific adverse events, can guide treatment decisions.
- Future research should focus on developing novel biomarkers and dynamic treatment strategies to improve outcomes in HCC.
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