Background Sorafenib is a multikinase inhibitor approved for the treatment of hepatocellular carcinoma (HCC). In clinical trials sorafenib treatment resulted in a median overall survival of 9.2 months and a median time to progression of 5.5 months (SHARP study).
Purpose To describe the results of sorafenib treatment for HCC in terms of progression-free survival (PFS), toxicity and compliance in clinical practice.
Material and methods Retrospective, descriptive, real-world data-based study including patients with HCC treated with sorafenib between January 2011 and May 2017 at a regional reference hospital.
Initial registered variables: age, sex, Child–Pugh status.
Follow-up variables: discontinuation and reason of discontinuation (progression, death, worsening of clinical condition, unacceptable toxicity, lack of adherence, patient decision, loss of follow-up).
Median PFS and PFS at 1 year were measured.
All the data was extracted from the clinical practice registries: electronic clinical records (SAP®) and pharmacy dispensation records (Silicon®). The statistical data was obtained from the SPSS®program applying Kaplan–Meier analysis.
Results A total of 55 patients aged 63.4±14 were included (85% males). Child–Pugh score was A, B or C in 35 (64%), 14 (25%) and six (11%) patients respectively. Twenty-two of them (40%) were excluded from the follow-up analysis because they did not reach a minimum of 45 days of treatment: nine (16%) presented unacceptable toxicity, seven (13%) died prematurely, four (7%) worsened their clinical condition and two (4%) were lost. The most frequent toxicity was asthaenia 18/55 (32.7%)
Among the remaining 33 patients, 16 (48.5%) stopped the treatment for death, six (18.2%) for unacceptable toxicity and six (18.2%) for worsening in their clinical situation and progression. The other five (15.1%) continues with active treatment. Compliance among these patients was 90%.
The median of PFS for the 33 patients in the follow-up phase was 209±53 days and the PFS at 1 year was 15%±7%.
Conclusion In more than one-third of our HCC patients who started sorafenib, the drug could be deemed ineffective and harmful. In the patients who survived the initial phase of 45 days, PFS yielded slightly better results than expected from clinical trials. Limitations of the study include lack of data on patient-related outcomes.
No conflict of interest
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