Suzuki Eiichiro, Kaneko Shuichi, Okusaka Takuji, Ikeda Masafumi, Yamaguchi Kensei, Sugimoto Rie, Aramaki Takeshi, Asagi Akinori, Yasui Kohichiroh, Sano Keiji, Hosokawa Ayumu, Kato Naoya, Ishii Hiroshi, Sato Tosiya, Furuse Junji
Department of Gastroenterology, Graduate School of Medicine, Chiba University, Chiba.
Department of Gastroenterology, Kanazawa University School of Medical Science, Kanazawa.
Jpn J Clin Oncol. 2018 Apr 1;48(4):317-321. doi: 10.1093/jjco/hyy010.
To evaluate prospectively the efficacy and safety of sorafenib, which has been the first-line treatment for advanced hepatocellular carcinoma (HCC), in Japanese HCC patients (pts) with not only Child-Pugh (C-P) A class but also C-P B class.
Sorafenib was administered orally at the dose of 400 mg twice daily for pts with HCC and liver function of C-P score of 5-8. Administration was continued until the detection of disease progression or appearance of unacceptable toxicity. The primary endpoint was time to progression (TTP), and toxicity and the secondary endpoints included objective response, overall survival (OS).
Forty C-P A pts and 12 C-P B pts were enrolled. The median TTP in the C-P A pts and C-P B pts was 3.3 months and 3.2 months, respectively. Among the pts with C-P A, complete response, partial response, and stable disease were achieved for 2.5%, 7.5% and 47.5%. Among the pts with C-P B, there were no treatment responses, 66.7% of pts had stable disease. The median OS in the C-P A pts and C-P B pts was 13.4 months and 7.4 months, respectively. With regard to toxicities, fewer C-P A pts experienced Grade 3/4 toxicities than C-P B pts (77.5% vs. 91.6%). There were no treatment-related deaths in either group of patients.
This study shows sorafenib has similar effectiveness in the recent post-approval studies and is well-tolerated in Japanese pts with HCC and Child Pugh A class. Sorafenib should be used with great care for Child Pugh class B pts.
前瞻性评估索拉非尼对日本肝细胞癌（HCC）患者的疗效和安全性。索拉非尼已成为晚期HCC的一线治疗药物，这些日本患者不仅包括Child-Pugh（C-P）A类，还包括C-P B类。
对于C-P评分为5-8分的HCC和肝功能患者，口服索拉非尼，剂量为400mg，每日两次。持续给药直至检测到疾病进展或出现不可接受的毒性。主要终点是进展时间（TTP），毒性和次要终点包括客观缓解率、总生存期（OS）。
纳入40例C-P A类患者和12例C-P B类患者。C-P A类患者和C-P B类患者的中位TTP分别为3.3个月和3.2个月。在C-P A类患者中，完全缓解、部分缓解和病情稳定的患者分别为2.5%、7.5%和47.5%。在C-P B类患者中，没有治疗反应，66.7%的患者病情稳定。C-P A类患者和C-P B类患者的中位OS分别为13.4个月和7.4个月。关于毒性，C-P A类患者发生3/4级毒性的人数少于C-P B类患者（77.5%对91.6%）。两组患者均无治疗相关死亡。
本研究表明，索拉非尼在近期的批准后研究中具有相似的有效性，并且在日本HCC和Child Pugh A类患者中耐受性良好。对于Child Pugh B类患者，应谨慎使用索拉非尼。
