Overall Survival in Patients with Hepatocellular Carcinoma Treated with Sorafenib: A Polish Experience.

BACKGROUND The mortality caused by hepatocellular carcinoma is expected to rise in the upcoming decade. Sorafenib has become the preferred systemic treatment option in patients with unresectable HCC. This study aimed to present the median overall survival (OS) in a group of patients with advanced HCC, treated with sorafenib in Poland between 2011 and 2019. MATERIAL AND METHODS The analyzed group of patients was qualified for treatment with sorafenib, financed by the National Health Fund, based on the guidelines of the Polish Drug Program. Kaplan-Meier method was used to plot the OS curves, and the log-rank test was used for testing. Multivariate assessment of factors (sex and age) related to the time to death of the patient was done using Cox regression. RESULTS Of the 2072 treated patients, 75% were men (1556) and 25% were women (516). The minimum age of patients in the trial group was 18 years and the maximum age was 90 years. Among the 1556 analyzed cases in males, 27.44% (427) did not end with death (by the date of completing the analysis). The percentage of one-year survival for this population was 58.16%, and the 2-, 3-, and 5-year survival rates were 34.45%, 21.81%, and 9.72%, respectively. The percentage of censored cases in the 516 females was 25.78% (133). The 1-2-, 3-, and 5-year survival for this population was 59.30%, 36.27%, 22.47%, and 11.34%, respectively. Statistical tests did not reveal a significant difference in the curve profiles by sex. There were no associations between OS and age. CONCLUSIONS Systemic treatment with sorafenib in accordance with the presented criteria allows for very good results, comparable to the results of selected groups of patients presented by other authors.

Real-World Evidence of Patient Outcome Following Treatment of Advanced Gastrointestinal Stromal Tumor (GIST) with Imatinib, Sunitinib, and Sorafenib in Publicly Funded Health Care in Poland.

BACKGROUND This study aimed to undertake an analysis of ten years of real-world evidence (RWE) on overall survival (OS) following treatment of advanced gastrointestinal stromal tumor (GIST) with imatinib, sunitinib, and sorafenib using data from the Polish National Health Fund. MATERIAL AND METHODS Data from the Polish National Health Fund, the sole Polish public payer, identified 1,641 patients with advanced GIST who were treated with imatinib (n=1047), sunitinib (n=457), and sorafenib (n=137). The differences in overall survival (OS) were analyzed. RESULTS For patients with advanced GIST, the median follow-up time for patients treated with imatinib was 71 months (95% CI, 64.8-79.2), the median OS was 56.9 months (95% CI, 50.4-61.2), with survival at 12 months (89.5%), 24 months (77.9%), 36 months (66.9%), and 60 months (48.4%). The median follow-up time for patients treated with sunitinib was 41.4 months (95% CI, 34.6-49.3), the median OS was 22.8 months (95% CI, 19.2-26.8), with survival at 12 months (68.2%), 24 months (47.1%), and 36 months (31%). The median follow-up time for patients treated with sorafenib was 17.4 months (95% CI, 14.6-22.9), the median OS was 16.9 months (95% CI, 13.7-24.3), with survival at 12 months (61.9%), at 24 months (36.2%), and at 36 months (16.8%). CONCLUSIONS Real-world data collected in a ten-year period confirmed the effectiveness of the use of imatinib, sunitinib, or sorafenib for the treatment of advanced GIST and was comparable with the findings from clinical trials.