Open AccessA Phase II Randomized Dose Escalation Trial of Sorafenib in Patients With Advanced Hepatocellular Carcinoma
Author(s) -
Rimassa Lorenza,
Pressiani Tiziana,
Boni Corrado,
Carnaghi Carlo,
Rota Caremoli Elena,
Fagiuoli Stefano,
Foa Paolo,
Salvagni Stefania,
Cortesi Enrico,
Chiara Tronconi Maria,
Personeni Nicola,
Bozzarelli Silvia,
Chiara Banzi Maria,
Fanello Silvia,
Romano Lutman Fabio,
Giordano Laura,
Santoro Armando
Publication year - 2013
Publication title -
the oncologist
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 2.176
H-Index - 164
eISSN - 1549-490X
pISSN - 1083-7159
DOI - 10.1634/theoncologist.2012-0221
Subject(s) - medicine , sorafenib , hepatocellular carcinoma , oncology , de escalation , randomized controlled trial , carcinoma
Background. Sorafenib has proven survival benefits in patients with advanced hepatocellular carcinoma (HCC). The viability of continuing sorafenib at a higher dosage in patients who experienced radiologic disease progression was investigated. Methods. Patients who experienced disease progression while on sorafenib 400 mg twice daily were randomized to sorafenib 600 mg twice daily ( n = 49) or best supportive care ( n = 52). The primary end point was progression‐free survival (PFS). Time to progression, overall survival, and safety were also evaluated. Results. The study did not meet its primary end point. The difference in PFS between the sorafenib arm (3.91 months) and the best supportive care arm (2.69 months) did not reach statistical significance ( p = 0.086). Adverse events were mainly grade 1–2 and similar across both groups. In the sorafenib arm, the most frequent events were diarrhea (80%), weight loss (75%), fatigue (67%), hand‐foot‐skin reaction (49%), abdominal pain (37%), and stomatitis (26%). Conclusions. Escalated‐dose sorafenib in patients with advanced HCC who progressed while on sorafenib, failed to provide any clinical benefit. Second‐line treatment still remains an open issue to be explored in appropriate clinical trials.