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

Winkel, Mark op den; Nagel, Dorothea; Seidensticker, Max; De Toni, Enrico N.; Merz, Jessica; op den Winkel, Jan; Oecal, Osman; Stecher, Stephanie-Susanne; Bourhis, Helene; Malfertheiner, Peter; Mayerle, Julia; Ricke, Jens und Kolligs, Frank T. (2022): Development and External Validation of the Munich Sorafenib Evaluation Score for Hepatocellular Carcinoma. In: Digestive Diseases, Bd. 41, Nr. 2: S. 268-281

Abstract

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 (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 (n = 101) derived from the prospective multicenter 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.

• Dokumententyp: Zeitschriftenartikel
• Fakultät: Medizin
• Themengebiete: 600 Technik, Medizin, angewandte Wissenschaften > 610 Medizin und Gesundheit
• ISSN: 0257-2753
• Sprache: Englisch