Development and External Validation of the Munich Sorafenib Evaluation Score for Hepatocellular Carcinoma

Abstract

<b><i>Introduction:</i></b> 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. <b><i>Methods:</i></b> Prognostic factors identified in a multivariate analysis of 108 sorafenib patients were used to construct the Munich Sorafenib Evaluation (M-SE) score. 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 (<i>n</i> = 101) derived from the prospective multicenter randomized controlled SORAMIC trial. <b><i>Results:</i></b> Ascites (<i>p</i> &#x3c; 0.0001; HR 2.923), tumor burden ≥50% of the liver (<i>p</i> = 0.0033; HR 1.946), and GOT (<i>p</i> &#x3c; 0.0001; HR 1.716) were identified as independent prognostic parameters. All three M-SE stages were characterized by significantly different survival times (<i>p</i> &#x3c; 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. <b><i>Conclusion:</i></b> 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.