Efficacy and tolerability of low-dose thalidomide as first-line systemic treatment of patients with advanced hepatocellular carcinoma.
OBJECTIVE
The systemic treatment of advanced hepatocellular carcinoma (HCC) has produced disappointing results thus far. HCC is a hypervascular tumor with over-expression of angiogenic factors such as vascular endothelial growth factor. Thalidomide is an anti-neoplastic agent with anti-angiogenic and other mechanisms of action. We aim to evaluate the efficacy and toxicity of low-dose (100 mg) thalidomide as the first-line treatment of advanced HCC.


METHODS
Between August 2003 and March 2007, 45 patients who had received thalidomide 100 mg daily as first-line treatment of advanced HCC were reviewed retrospectively. Advanced HCC was defined as either metastatic or not amenable to surgical or locoregional therapies. Diagnosis of HCC was based on clinical, biochemical and radiological grounds. Survival was analyzed by the Kaplan-Meier method.


RESULTS
Thirty-eight patients were evaluable for response and toxicity. Two (5%) patients had partial response and 8 (21%) had stable disease. The overall median survival of patients in this cohort was 3.2 months (95% CI: 2.8-3.7 months). The common toxicities were somnolence (13%), peripheral neuropathy (11%) and ankle edema (8%), with no grade 3 or 4 toxicities and treatment-related deaths.


CONCLUSIONS
Our study shows that a single agent, low-dose thalidomide has a modest clinical activity with good tolerability in treating advanced HCC patients.
Yau
Thomas
T
Department of Medicine, University of Hong Kong Medical Centre, Queen Mary Hospital, Hong Kong, China.
Chan
Pierre
P
Wong
Hilda
H
Ng
Kelvin K
KK
Chok
Siu-Ho
SH
Cheung
Tan-To
TT
Lam
Vincent
V
Epstein
Richard J
RJ
Fan
Sheung-Tat
ST
Poon
Ronnie T P
RT
D000328:Adult; D000368:Aged; D000369:Aged, 80 and over; D020533:Angiogenesis Inhibitors / Q000008:administration & dosage* / Q000009:adverse effects* / Q000494:pharmacology; D000842:Ankle; D006528:Carcinoma, Hepatocellular / Q000188:drug therapy* / Q000556:secondary; D004487:Edema / Q000139:chemically induced; D005260:Female; D006801:Humans; D053208:Kaplan-Meier Estimate; D008113:Liver Neoplasms / Q000188:drug therapy* / Q000473:pathology; D008297:Male; D008875:Middle Aged; D009367:Neoplasm Staging; D010523:Peripheral Nervous System Diseases / Q000139:chemically induced; D012189:Retrospective Studies; D012894:Sleep Stages / Q000187:drug effects; D013792:Thalidomide / Q000008:administration & dosage* / Q000009:adverse effects* / Q000494:pharmacology
D020533:Angiogenesis Inhibitors; D013792:Thalidomide