Boost: A phase 3 trial of sorafenib vs. best supportive care in first line treatment of hepatocellular carcinoma in patients with deteriorated liver function

Gennaro Daniele, Clorinda Schettino, Laura Arenare, Domenico Bilancia, Fabio Farinati, Piera Federico, Stefano Tamberi, Gino Crivellari, Sandro Barni, Raffaella Tortora, Francesco Izzo, Antonio Frassoldati, Luigi Cavanna, Claudia Mucciarini, Luigi Bolondi, Angelo Dinota, Filippo Pelizzaro, Maria Carmela Piccirillo, Piera Gargiulo, Massimo Di MaioCiro Gallo, Francesco Perrone, Bruno Daniele

Research output: Contribution to journalArticlepeer-review

Abstract

Aim: Only patients with good liver function {[Child-Pugh (CP)] A class} were eligible for trials testing sorafenib as first-line treatment of hepatocellular carcinoma (HCC); nevertheless, the drug was authorized without restrictions based on liver function. Therefore, we planned to test sorafenib efficacy and safety in patients with HCC and deteriorated liver function (CP-B). Methods: This was an open-label, multicenter, randomized phase 3 trial. Patients with HCC, no previous systemic therapy, and CP-B score 7-9 were assigned 1:1 to best supportive care alone (control arm) or with standard dose sorafenib (experimental arm). Overall survival (OS) was the primary endpoint. To detect a 0.70 HR of death, with 80% power, and two-tailed α error 0.05, 234 events were required. The study closed prematurely because of slow accrual. Descriptive analyses are reported. Results: From 2012 to 2017, 13 Italian centers randomized 35 patients. In total, 28 deaths were recorded, 12 without and 16 with sorafenib; median OS was 4.9 (95%CI: 1.2-5.6) and 3.5 months (95%CI: 1.3-5.3), respectively. At least one severe adverse event was reported in 2/15 (13.3%) without and 9/17 (52.9%) patients with sorafenib. Conclusions: This trial failed its planned enrolment goal, showing the difficulty in performing clinical trials with drugs already registered with a label broader than what available evidence supports.

Original languageEnglish
Article number61
JournalHepatoma Research
Volume7
DOIs
Publication statusPublished - 2021

Keywords

  • Child-pugh B class
  • Hepatocellular carcinoma
  • Sorafenib

ASJC Scopus subject areas

  • Hepatology
  • Oncology

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