The hallmarks of hepatocellular carcinoma (HCC) are that it is identified clinically at an advanced stage and usually together with cirrhosis. Surgical resection has been considered the optimal treatment approach, but only a small proportion of patients qualify for surgery, and there is a high rate of recurrence. Approaches to prevent recurrence have included chemoembolization before and neoadjuvant therapy after surgery, neither of which has proven to be beneficial. Liver transplantation has been successful in treating limited-stage HCC, affecting cure of both the tumor and underlying cirrhosis. However, only a minority of patients with HCC qualify for transplantation. Recently, chemoembolization has been shown to prolong survival in selected patients who do not qualify for transplantation or resection. Other innovative, relatively noninvasive local ablative therapies have been introduced and have been shown to be effective in reducing tumor size but not in prolonging survival. Standard chemotherapy is poorly tolerated in patients who do not qualify for resection. Both doxorubicin and cisplatin are frequently used, but overall response rates are low, and neither seems to prolong survival. Prospective, randomized controlled trials using current therapies are needed to better define optimal management of this important tumor. Most needed, however, are new therapeutic agents that are effective against HCC, are noncytotoxic, and are tolerated by the typical patient with underlying cirrhosis. Newly emerging agents with promise include 90Y microspheres, antiangiogenesis agents, inhibitors of growth factors and their receptors, and K vitamins.
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