Abstract
Sorafenib is recommended for the treatment of advanced-stage hepatocellular carcinoma (HCC). Nonetheless, it is expensive, effective in few patients, and may cause significant adverse effects. Therefore, accurate selection of patients is needed. In a previous study, we constructed a simple scoring system to predict patients' outcomes based on the occurrence of sorafenib adverse effects. The present study aimed to validate this scoring system in a real-life cohort of HCC patients. Clinical records of 279 outpatients treated with sorafenib in eight Italian centers were retrospectively analyzed. Adverse effects considered to calculate the score were skin toxicity, diarrhea, and arterial hypertension, occurring during the first month of therapy. For each adverse effect, 1 point was assigned if present; and 0 points if absent (resulting in a total score between 0 and 3). Median overall survival (OS) was 10.8months and median time to progression (TTP) was 5.1months. At multivariate analysis, performance status, α-fetoprotein (AFP), and Child-Pugh score were independently associated with TTP and OS. A progressive increase of OS and TTP was observed in patients with scores from 0 to 3 (p<0.001). Six-, 12-, and 24-month survival probabilities were 55.1, 24.5, and 7.9% in score 0 patients, and 100, 80.9, and 46.2% in score 3 patients, respectively. Complete response was observed in one patient (0.4%), partial responses in 41 (15.2%), and stable disease in 117 (43.5%). The disease control rate in patients with scores of 0, 1, 2, and 3 was 34.3, 51.6, 80.9, and 96.3%, respectively (p<0.001). Complete or partial responses were not observed in score 0 patients. We have validated a useful scoring system to predict outcomes in sorafenib-treated HCC patients. This score is easy to calculate and suitable for implementation in daily clinical practice.
Highlights
Hepatocellular carcinoma (HCC) is one of the deadliest cancers
Sorafenib is recommended for the treatment of advanced hepatocellular carcinoma; neverthless it is difficult to manage
Most cases develop in the setting of chronic liver inflammation that has progressed to cirrhosis, the main risk factor in 70– 90% of patients
Summary
Hepatocellular carcinoma (HCC) is one of the deadliest cancers. it represents only approximately 4% of all new cancer cases diagnosed worldwide, HCC is the fourth leading cause of cancer death in males and the sixth in females [1]. Knowledge of the risk factors for HCC provides an opportunity for the development and implementation of preventative strategies aiming to decrease the worldwide burden of the disease [4]. For this purpose, national and international associations have issued guidelines on surveillance strategies for early diagnosis of HCC, based on the use of periodic ultrasound examinations [5,6,7]. Sorafenib is recommended for the treatment of advanced-stage hepatocellular carcinoma (HCC). It is expensive, effective in few patients, and may cause significant adverse effects. We constructed a simple scoring system to predict patients’ outcomes based on the occurrence of sorafenib adverse effects
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