Value-based approach to hepatocellular carcinoma: outcome indicators tested in a large multicenter study (VBMH study)

Background and Aims: Hepatocellular carcinoma (HCC) is a worldwide public health problem, accounting for increasing mortality and high costs. Aim of our study 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; three of these OIs with the highest RAND/UCLA scores were tested in a prospective multicenter observational study (Value Based Medicine in Hepatology, VBMH). During 18 months, 711 HCC patients were enrolled and prospectively followed. Median follow-up time was 14 months. Results: The first OI was survival after 1–3–5 years stratified for BCLC stage or treatment (OI#1). One-year survival for BCLC stage 0/A, B, C, D was 93%, 86%, 50%, 26% respectively. One-year survival of 288 patients who had the first treatment during the study time was 88% for liver transplantation, 97% surgical resection, 100% ablation and 89% for TACE. The remaining two OIs meant to evaluate the appropriateness of treatments. Worsening of BCLC and/or CPT score after loco-regional therapy or surgical resection recorded at three months (OI#2), occurred in 16% of cases (76% after TACE). Very early HCC recurrence (within 6 months) after curative treatments (OI#3) happened in 15% of patients treated (20% after ablation). Conclusions: This set of outcome indicators proved their feasibility in a large cohort of patients and could serve as a benchmark for healthcare providers to move towards a value-based approach in the management of HCC.