Phase 2 open‐label study of single‐agent sorafenib in treating advanced hepatocellular carcinoma in a hepatitis B–endemic Asian population

Abstract

BACKGROUND: The current study was a phase 2 open–label study to evaluate the efficacy and tolerability of single‐agent sorafenib in the treatment of advanced HCC patients in a hepatitis B–endemic Asian population. METHODS: Patients with advanced hepatocellular carcinoma (HCC) received sorafenib at a dose of 400 mg twice daily in 4‐week cycles. Tumor response was assessed every 3 cycles using Response Evaluation Criteria in Solid Tumors criteria. RESULTS: Fifty‐one patients were enrolled in the study and were treated with sorafenib for at least 12 weeks. The median age was 56 years (range, 28‐79 years). Approximately 90% had hepatitis B virus–related HCC. Thirty‐six (71%) patients had underlying Child‐Pugh A cirrhosis, 13 (26%) Child‐Pugh B, and 2 (3%) Child‐Pugh C. Four (8%) patients achieved partial responses, and 9 (18%) patients had stable disease for at least 12 weeks. The median overall survival was 5 months (range, 4‐17 months). Patients without extrahepatic spread, particularly without lung metastasis (P<.01), are more likely to benefit from sorafenib treatment. The most common toxicities were diarrhea (67%), malaise (55%), and hand‐foot‐skin reaction (54%). The majority of patients had transient liver function derangement. Patients with and without underlying portal vein thrombosis had similar therapeutic benefits and likewise shared a similar treatment‐related toxicity profile with sorafenib treatment. CONCLUSIONS: Single‐agent sorafenib demonstrates good efficacy and acceptable tolerability in treating an advanced HCC patient population in a hepatitis B–endemic area. The presence of lung metastasis predicts poor response to sorafenib in advanced HCC patients. Cancer 2009. © 2009 American Cancer Society.

Prognostic factors in patients with advanced hepatocellular carcinoma treated with sorafenib

“…Indeed, it is possible that an analysis of outcomes according to each BCLC stage of disease with respect to Child-Pugh class would provide us with further details of the true benefits of sorafenib in each of these patient subsets, but the low patient numbers prevented such an analysis in this study. Nevertheless, the alignment of our findings in CP-A patients with those reported in SHARP, 9 and in CP-B patients with previously reported prospective and retrospective studies, 8,9,13,25,26 support the validity of our findings.…”

Sorafenib Continuation after First Disease Progression Could Reduce Disease Flares and Provide Survival Benefits in Patients with Hepatocellular Carcinoma: a Pilot Retrospective Study

“…Therefore, even under the ideal conditions of clinical trials with sorafenib alone or in combination with TACE, 19.5%-71.4% of patients discontinued treatment (JM et al, 2008;Pinter et al, 2009;Worns et al, 2009;Yau et al, 2009;Dufour et al, 2010). In clinical practice, where the cost of sorafenib treatment may influence patients' compliance, the discontinuation rate may be even higher.…”

The Significance of Early Alpha-Fetoprotein Level Changes in Predicting Clinical and Survival Benefits in Advanced Hepatocellular Carcinoma Patients Receiving Sorafenib

“…Twenty percent drop in AFP was chosen as the cutoff point because of prior data suggesting a significant prognostic value of this level of AFP drop in patients undergoing chemotherapy or anti-angiogenic therapy for HCC [8,12]. Between November 2006 and January 2008, a phase II, open-label, single-arm sorafenib study for patients with advanced HCC was conducted at Queen Mary Hospital, The University of Hong Kong [13]. Written consents were obtained from the patients before enrollment.…”