Abstract
Background. Advanced HCC is a clinical challenge with limited treatment options. The multikinase inhibitor sorafenib is the first and only agent showing a survival benefit in these patients. In this study we evaluate the efficacy and tolerability of sorafenib in an unselected patient population. Furthermore we explore the role of alpha-fetoprotein (αFP) as a potential biomarker for treatment efficacy and correlation to survival. Methods. Seventy-six patients with advanced HCC, not eligible for locoregional therapy, treated with sorafenib between 2007 and 2009 were included. Followup was until 2011. Results. Patients in PS 0-1 had a median overall survival (mOS) of 6.2 months, compared to 1.8 months in patients with poorer PS (P = 0.005). Child-Pugh A patients had a mOS of 6.6 months versus 3.6 months among patients in Child-Pugh B or C (P = 0.0001). Serum αFP ≥ 200 at baseline was prognostic for a shorter survival. All patients with radiologically verified tumor response and baseline αFP ≥ 200 experienced a significant decline in αFP within the first four weeks of treatment. Conclusion. The survival of patients with advanced HCC treated with sorafenib is dependent on performance status and liver function. Treatment of patients with compromised liver function and poor performance status cannot be recommended. The correlation between αFP and objective tumor response warrants further investigation.
Highlights
Until recently treatment options for advanced or unresectable hepatocellular carcinoma (HCC) have been limited as chemotherapy in general is ineffective [1]
Signi cant progress in the treatment of HCC was made with the approval of the multikinase inhibitor sorafenib for this indication [2]. e approval was based upon two placebocontrolled randomi ed trials which for the rst time could demonstrate a survival bene t in HCC patients treated with sorafenib [3, 4]. e majority of patients included in these studies were in ECOG performance status (PS) 0 or 1 and had an ade uate liver function classi ed as ChildPugh A (CP-A)
Seventy-eight per cent of the patients were in PS 0-1, and 57% had a well preserved liver function (CP-A)
Summary
Until recently treatment options for advanced or unresectable hepatocellular carcinoma (HCC) have been limited as chemotherapy in general is ineffective [1]. Signi cant progress in the treatment of HCC was made with the approval of the multikinase inhibitor sorafenib for this indication [2]. E approval was based upon two placebocontrolled randomi ed trials which for the rst time could demonstrate a survival bene t in HCC patients treated with sorafenib [3, 4]. Despite the lack of evidence of a survival bene t, many HCC patients with Child-Pugh B and even C liver cirrhosis are treated with sorafenib [6]. All patients with radiologically veri ed tumor response and baseline ααFP ≥ 200 experienced a signi cant decline in ααFP within the rst four weeks of treatment. E survival of patients with advanced HCC treated with sorafenib is dependent on performance status and liver function. Treatment of patients with compromised liver function and poor performance status cannot be recommended. e correlation between ααFP and objective tumor response warrants further investigation
Sign-in/Register to view highlights & summary Sign-in/Register to access full text options 
Free) 
Talk to us
Join us for a 30 min session where you can share your feedback and ask us any queries you have
Schedule a call
