Phase 2 open-label study of single-agent sorafenib in treating advanced hepatocellular carcinoma in a hepatitis B-endemic Asian population: presence of lung metastasis predicts poor response.
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.
Yau
Thomas
T
Department of Medicine, The University of Hong Kong, Queen Mary Hospital, Hong Kong, China.
Chan
Pierre
P
Ng
Kelvin K
KK
Chok
Sin Ho
SH
Cheung
Tan To
TT
Fan
Sheung Tat
ST
Poon
Ronnie T
RT
D000328:Adult; D000368:Aged; D000970:Antineoplastic Agents / Q000627:therapeutic use*; D044466:Asian People; D001557:Benzenesulfonates / Q000009:adverse effects / Q000627:therapeutic use*; D006528:Carcinoma, Hepatocellular / Q000188:drug therapy* / Q000209:etiology / Q000473:pathology; D005260:Female; D006509:Hepatitis B / Q000150:complications*; D006801:Humans; D008103:Liver Cirrhosis / Q000150:complications; D008113:Liver Neoplasms / Q000188:drug therapy* / Q000209:etiology / Q000473:pathology; D008175:Lung Neoplasms / Q000556:secondary*; D008297:Male; D008875:Middle Aged; D009536:Niacinamide / Q000031:analogs & derivatives; D010671:Phenylurea Compounds; D047428:Protein Kinase Inhibitors / Q000627:therapeutic use*; D011725:Pyridines / Q000009:adverse effects / Q000627:therapeutic use*; D000077157:Sorafenib
D000970:Antineoplastic Agents; D001557:Benzenesulfonates; D010671:Phenylurea Compounds; D047428:Protein Kinase Inhibitors; D011725:Pyridines; D009536:Niacinamide; D000077157:Sorafenib