Tamoxifen is not effective in good prognosis patients with hepatocellular carcinoma.

Abstract

BACKGROUND:

Large randomised clinical trials and systematic reviews substantiate that tamoxifen is ineffective in improving survival of patients with hepatocellular carcinoma (HCC). However, a recent report suggested that the drug might prolong survival among patients with well preserved liver function. The aim of this paper is to validate this hypothesis.

METHODS:

We used the updated database of the phase 3 randomised CLIP-1 trial that compared tamoxifen with supportive therapy. Primary endpoint was overall survival. Treatment arms were compared within strata defined according to the Okuda stage and the CLIP-score. Survival differences were tested by the Log-rank test.

RESULTS:

Tamoxifen was not effective in prolonging survival in Okuda I-II subgroup (p = 0.501). Median survival times were equal to 16.8 (95%CI 12.7-18.5) months for tamoxifen and 16.8 (95%CI 13.5-22.4) months for the control arms; 1-year survival probabilities were equal to 58.8% (95%CI 51.7-65.8) and 59.4 (95%CI 52.5-66.2), respectively. Similar results were observed in the better CLIP subgroup (score 0/1), without evidence of difference between the two treatment arms (p = 0.734). Median survival times were equal to 29.2 (95%CI 20.1-36.4) months with tamoxifen and 29.0 (95%CI 23.3-35.2) months without; 1-year survival probabilities were equal to 80.9% (95%CI 72.5-89.3) with tamoxifen and 77.1% (95%CI 68.6-85.7) for the control arm.

CONCLUSION:

The recent suggestion that tamoxifen might be effective in the subgroup of patients with better prognosis is not supported by a reanalysis of the CLIP-1 trial. Tamoxifen should no longer be considered for the treatment of HCC patients and future trials of medical treatment should concentrate on different drugs.

• Authors: Ciro Gallo, Ermelinda De Maio, Massimo Di Maio, Giuseppe Signoriello, Bruno Daniele, Sandro Pignata, Annalisa Annunziata, Francesco Perrone, CLIP (Cancer of the Liver Italian Programme) Investigators
• Journal: BMC cancer (BMC Cancer) Vol. 6 Pg. 196 (Jul 24 2006) ISSN: 1471-2407 [Electronic] England
• PMID: 16863588 (Publication Type: Journal Article, Multicenter Study, Randomized Controlled Trial, Research Support, Non-U.S. Gov't)

Topics

• Adult
• Aged
• Aged, 80 and over
• Carcinoma, Hepatocellular (drug therapy, pathology)
• Female
• Follow-Up Studies
• Humans
• Liver Neoplasms (drug therapy, pathology)
• Male
• Middle Aged
• Neoplasm Staging
• Prognosis
• Survival Analysis
• Treatment Outcome