Retrospective Study
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World J Gastroenterol. Sep 21, 2014; 20(35): 12581-12587
Published online Sep 21, 2014. doi: 10.3748/wjg.v20.i35.12581
Indicators of sorafenib efficacy in patients with advanced hepatocellular carcinoma
Masayoshi Yada, Akihide Masumoto, Kenta Motomura, Hirotaka Tajiri, Yusuke Morita, Hideo Suzuki, Takeshi Senju, Toshimasa Koyanagi
Masayoshi Yada, Akihide Masumoto, Kenta Motomura, Hirotaka Tajiri, Yusuke Morita, Hideo Suzuki, Takeshi Senju, Toshimasa Koyanagi, Department of Hepatology, Iizuka Hospital, Iizuka, Fukuoka 820-8505, Japan
Author contributions: Yada M, Masumoto A, Motomura K, Tajiri H, Morita Y, Suzuki H, Senju T and Koyanagi T designed and performed the research; Yada M analyzed data; Yada M and Masumoto A wrote the paper.
Correspondence to: Akihide Masumoto, MD, PhD, Department of Hepatology, Iizuka Hospital, 3-83 Yoshio-machi, Iizuka, Fukuoka 820-8505, Japan. amasumotoh1@aih-net.com
Telephone: +81-948-22-3800 Fax: +81-948-29-5744
Received: February 19, 2014
Revised: April 10, 2014
Accepted: June 21, 2014
Published online: September 21, 2014
Processing time: 212 Days and 1.9 Hours
Abstract

AIM: To determine significant indicators for the efficacy of sorafenib in patients with advanced hepatocellular carcinoma (HCC).

METHODS: A total of 46 patients with Barcelona Clinic Liver Cancer stage C who received sorafenib for more than 30 d at the Iizuka Hospital from June 2009 to December 2012 were enrolled in this study. Multivariate and univariate analyses were performed to evaluate the associations of hepatic function according to Child-Pugh grade, location and size of the largest tumor and adverse events of sorafenib treatment, such as hand-foot syndrome (HFS), hypertension, diarrhea, and alopecia, with the efficacy of treatment, as measured by overall survival (OS) and time to progression (TTP).

RESULTS: Patients included 39 men and 7 women whose ages ranged from 48 to 85 years (70.6 ± 9.6 years). HCC was classified according to etiology as follows: hepatitis C virus (n = 26), hepatitis B virus (n = 9), and other (n = 11). Liver function in patients was categorized as Child-Pugh grade A (n = 30) or B (n = 16). Tumors were categorized by size [< 5 cm (n = 33) or >5 cm (n = 13)] and the location of the largest tumor was used to categorize patients with intrahepatic (n = 28) or extrahepatic (n = 18) HCC. HFS, hypertension, diarrhea, and alopecia were present in 22 (47.8%), 19 (41.3%), 15 (32.6%) and 7 patients (15.2%), respectively. The median OS of all patients was 373 d and the median TTP was 112 d. The etiology of HCC did not correlate with the median OS and TPP. The median OS of patients with tumors < 5 cm was significantly longer than those with larger tumors (496 vs 245 d; HR = 0.19, 95%CI: 0.07-0.48; P < 0.01). According to the results of a multivariate analysis, the size of the largest tumor affected OS (HR = 0.22, 95%CI: 0.08-0.59; P < 0.01). The median TTP was significantly longer in patients with extrahepatic compared to intrahepatic major HCC (224 vs 98 d; HR = 0.32; 95%CI: 0.14-0.67; P < 0.01). The median TTP of patients with HFS was significantly longer than those without it (195 d vs 83 d; HR = 0.41, 95%CI: 0.20-0.82; P < 0.05), and the median TTP was significantly longer in patients with hypertension (195 d vs 84 d; HR = 0.43, 95%CI: 0.21-0.84; P < 0.05). According to the results of the multivariate analysis, extrahepatic major HCC (HR = 0.36, P < 0.01) and HFS (HR = 0.44, P < 0.05) prolonged TTP.

CONCLUSION: Extrahepatic major HCC and HFS are associated with prolonged TTP and are useful indicators for judging the efficacy of sorafenib treatment.

Keywords: Hand-foot syndrome; Hepatocellular carcinoma; Indicator of efficacy; Sorafenib; Time to progression

Core tip: Clinical factors influencing the efficacy of sorafenib for treating patients with advanced hepatocellular carcinoma (HCC) were evaluated in this study. As only the size of the largest tumor correlated significantly with overall survival, analyses were focused on factors affecting time to progression (TTP). Sorafenib was more effective for treating patients with extrahepatic compared to intrahepatic advanced HCC. The occurrence of hand-foot syndrome as an adverse event significantly correlated with increased TTP and may therefore be a useful indicator for adjusting the dose of sorafenib to improve its effectiveness and the health-related quality of life of patients with advanced HCC.