Supplementary Material for: Development and external validation of the Munich Sorafenib Evaluation Score for hepatocellular carcinoma

Introduction: In recent years increasing options for systemic HCC treatment have become available. The development of therapy-specific prognostic scores has been encouraged. Tailoring therapy to individual patients requires prognostic scores for treatment success in addition to the Barcelona-Clinic-Liver-Cancer (BCLC) classification. We have developed and validated a prognostic score for patients treated with sorafenib. Methods: Prognostic factors identified in a multivariate analysis of 108 sorafenib patients were used to construct the Munich-Sorafenib-Evaluation score (M-SE). M-SE and 9 established HCC prognostic systems were ranked according to concordance-index and AIC. External M-SE-validation was performed in an independent HCC sorafenib cohort (n=101) derived from the prospective multicentre randomized controlled SORAMIC trial. Results: Ascites (p<0.0001; HR 2.923), tumor burden ≥50% of the liver (p=0.0033; HR 1.946) and GOT (p<0.0001; HR 1.716) were identified as independent prognostic parameters. All three M-SE stages were characterized by significantly different survival times (p<0.0001). M-SE stage A patients had a median OS of 18.7 months (95% CI 15.6-21.8), patients in stage B and C showed a significantly shorter survival of 5.7 (2.7-8.7) and 2.0 months (1.6-2.4), respectively. M-SE (c-index 0.70; AIC 621) outperformed all other prognostic systems. External validation in a prospective cohort confirmed its superior prognostic performance. Conclusion: The M-SE score allows classification of sorafenib-patients in three distinct prognostic stages. Provided that M-SE successfully passes prospective validation, it can help to predict the outcome of patients evaluated for sorafenib treatment.