Background On November 2007, the FDA approved sorafenib in the treatment of patients with unresectable hepatocellular carcinoma.
Purpose Evaluate the effectiveness and safety of sorafenib in adults diagnosed with hepatocarcinoma.
Material and methods Retrospective study of the effectiveness and safety of sorafenib in patients diagnosed with hepatocarcinoma between January 2012 and December 2016.
The following data were collected from the Electronic Clinical History (SIAS®) and the Abucasis® dispensing program: sex, age, degree of cirrhosis, date of initiation of treatment and duration of treatment, date of exitus, reason for termination of treatment, adverse effects (AEs) and need for dose reduction.
Results Thirty patients were included, 26 males (86.7%) being the mean age of 70 years (55–83). Seventy per cent of the patients had cirrhosis of some type. Stage A was the most common on the Child–Pug scale (81% of cases).
The median of progression-free survival (PFS) was 130 days (31–525) and the median of overall survival (OS) was 240 days (31–981). A significant relationship was observed between bilirubin levels at the initiation of sorafenib treatment and drug effectiveness. Patients with bilirubin ≤1. 5 mg/dL presented a PFS and OS of 189 and 288 days respectively, while those with bilirubin levels>1. 5 mg/dL showed a PFS and OS of 109 and 140 days respectively.
The main AEs observed were: digestive discomfort (70%), asthaenia (70%), anorexia (30%), hand-foot syndrome (20%), haematological toxicity (16.7%), hypertension (16.7%), and neurotoxicity (10%). Four patients (13.3%) required dose reduction and five patients finished treatment because of toxicity.
Conclusion In our study we obtained a PFS similar to pivotal studies, 130 days versus 160, respectively. However, it shows a lower OS (240 days vs. 324) so would be convenient to complete the study with a larger number of patients. It is observed that those patients with bilirubin levels≤1. 5 mg/dL present higher effectiveness rates in PFS and OS.
The safety profile is similar to the pivotal trial and more than half of the patients presented digestive toxicity and asthaenia.
No conflict of interest