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Introduction: The management of hepatocellular carcinoma (HCC) is a well-known public health problem, accounting for increasing mortality and high costs. Quality parameters able to measure clinical outcomes are still lacking. Aim of our study (Value Based Medicine in Hepatology, VBMH)was to identify and test outcome indicators (OIs) in HCC, in light of their potential use in policy decision models. Methods: A panel of experts identified a list of OIs using a modified Delphi method, according to experience and published evidence; four OIs with the highest RAND/UCLA score were tested in a prospective multicenter observational study. During 18 months, 711 HCC patients were enrolled and prospectively followed. 677 patients (95%) had at least one follow-up consultation. Median follow-up time was 14 months. Results: The first OI was 1–3–5 years survival stratified for BCLC stage or treatment (OI#1). One-year survival for BCLC stage 0/A–D was 93%, 86%, 50%, 26% respectively. One-year survival of 288 patients treated for the first time during the study was 88% for liver transplantation, 97% surgical resection, 100% ablation and 89% for TACE. The other three OIs evaluated the appropriateness of treatments: worsening of BCLC and/or CPT score after three months from a loco-regional therapy or surgical resection (OI#2), occurred in 16% of cases (76% after TACE). Recurrence of HCC within 6 months after curative treatments (OI#3) happened in 15% of patients treated, mainly after ablation (20%). Presence of severe morbidity three months after loco-regional procedure or resection, evaluated as grade≥3 according to Clavien-Dindo classification (OI#3), was found in 3% of patients treated. Conclusions: The outcome indicators identified in the VBMH study proved their feasibility in a large cohort of patients and could stand as a benchmark for healthcare providers to move towards a value-based approach in the management of HCC.

Introduction: Liver Cirrhosis (LC) is responsible for high morbidity, mortality and costs, with increasing need to improve quality of care. Aim of our study (Value Based Medicine in Hepatology Study, VBMH) was to identify outcome indicators (OIs) able to measure quality in liver diseases, including compensated (CC) and decompensated (DC) LC. Methods: A panel of hepatologists identified a list of 7 OIs for LC according to experience and published evidence, using a modified Delphi method and a standard 9-pointRANDappropriateness scale. Then, these 7 OIs were tested in clinical practice in a prospective multicenter observational study involving three tertiary centers in Italy, using a web-based electronic medical record. 1751 LC patients were enrolled in 18 months: 1004 CC and 747 DC. 92% of patients had at least two consultations in 17 months median follow-up. Results: Annual rate of decompensation in CC was 12% (OI#1). Annual incidence of 1st variceal bleeding (VB) was 2% for low-risk and 3% for high-risk varices (OI#2) indicating a significant success of primary profilaxis. Annual incidence of HCC in CC was 4.3%, with 81% patients found at early stage (OI#3), underlining the accuracy of oncologic surveillance. One-year survival after the first decompensation episode (ascites in 74% of cases) was 96%, 81% and 59% stratified for CPT score A, B, C respectively (OI#4), and 94%, 56% stratified for MELD below or above 15 (OI#5). 4% of DC patients had an episode of VB with 90% survival after 6 weeks, and 33% recurrence (OI#6). Similarly, 3% ofDCpatients had spontaneous bacterial peritonitis with 6 weeks survival of 86% and 13% recurrence (OI#7). Conclusions: The value-based OIs generated in this study performed well in a large cohort of consecutive patients, and could represent a reference tool for healthcare providers to improve care in LC.

OBJECTIVES: Liver diseases (LDs) have a high impact on morbidity, mortality and health-related quality of life (HRQoL). LDs may have different impact on patients’ HRQoL. The aim of our study was to evaluate HRQoL in the major liver conditions: hepatitis B(HBV), hepatitis C(HCV), cirrhosis(compensated and decompensated), hepatocellular carcinoma(HCC), autoimmune hepatitis(AIH), primary biliary cirrhosis(PBC), primary sclerosing cholangitis(PSC), NAFLD/NASH, patients in the liver transplant list and post-transplant METHODS: a naturalistic, prospective, multicenter study has been conducted to generate and validate a set of health care outcomes indicators for the major liver conditions. LDs patients (age>18 years) were enrolled in 3 major Italian medical centers and are still being followed up (median f-up:13 months). Within this study, socio-demographic, clinical and HRQoL were collected using the EQ-5D-3L. The HRQoL data was analyzed dividing the patients in sub-groups according to the most recently diagnosed and most severe condition. RESULTS: we enrolled 3,217 patients, 64.8% male, aged 19-91 (median=61) years; 95.0% of them filled in the EQ-5D at baseline visit. Patients in the HCC group were 22.6%; while in the AIH group were 1.6%. The highest percentage of problems in Mobility dimension (39.2%) was reported by decompensated cirrhosis sub-group, the highest percentage in Self-care (22.6%) and Usual Activities (47.1%) by patients in liver transplant list, in Pain/Discomfort (59.2%) by AIH and in Anxiety/Depression (57.8%) by PBC. The HBV sub-group reported the best HRQoL with a mean EQ-5D VAS of 77.8; while AIH and listed for liver transplant patients reported the worst HRQoL levels (mean EQ-5D VAS=67.7 and 63.5, respectively). CONCLUSIONS: our study shows how HRQoL is different between LDs and how it is negatively related with the clinical severity. Understanding the different impact of LDs on the patients’ HRQoL could help physicians and decision makers to better estimate the burden of these conditions and to improve the quality of care.

monotherapy groups. The radiologic assessment was performed according to modified RECIST. Results: Cox proportional hazard regression analysis showed that vitamin K dosing improved PFS (p =0.003, hazard ratio:0.193). Furthermore, vitamin K and BCAA improved OS (p =0.013, hazard ratio:0.189 and p=0.016, hazard ratio:0.157, respectively). The median time of PFS was prolonged in vitamin K group compared with sorafenib monotherapy one (2.5 mo vs. 8.5 mo, respectively, Log-rank test: p = 0.003). The median time of OS was also extended (15.5 mo vs. 31.5 mo, p = 0.027). The BCAA improved OS compared with sorafenib monotherapy (median survival time: 17.0 mo vs. 36.5 mo, respectively, p = 0.030). Conclusions: These nontoxic agents could markedly improve the prognosis of sorafenib-treated patients with BCLC-B.