PMID- 19877181 OWN - NLM STAT- MEDLINE DCOM- 20100115 LR - 20181201 IS - 1527-3350 (Electronic) IS - 0270-9139 (Linking) VI - 51 IP - 1 DP - 2010 Jan TI - Use of sorafenib in patients with hepatocellular carcinoma before liver transplantation: a cost-benefit analysis while awaiting data on sorafenib safety. PG - 165-73 LID - 10.1002/hep.23260 [doi] AB - The role of bridging therapies for patients with hepatocellular carcinoma (HCC) on the waiting list for liver transplantation (LT) remains controversial. There is strong evidence to support the effectiveness of sorafenib in extending the time to progression of HCC. Using a Markov model, we compared two strategies: one using sorafenib as neoadjuvant therapy before LT (Strategy A), and the other using no bridging therapy in the first 6 months (Strategy B). Reference case: T2 HCC patient with compensated cirrhosis. The benefit of sorafenib in delaying time to HCC progression was expressed as the hazard ratio (HR) and taken from recently published randomized trials. The endpoints considered were: survival benefit measured in quality-adjusted life days (QALDs), transplant probability, costs (C) in euro, willingness to pay (WTP), and net health benefit (NHB), where NHB = survival benefit - C/WTP. The calculated WTP of sorafenib in Italy was 346 euro per QALD. Probabilistic sensitivity analysis showed a median survival benefit of 94 QALDs (10% percentile = 38, 90% percentile = 210). In the base-case scenario (HR = 0.47, monthly dropout probability = 5%, median time to LT = 3 months), the gain in LT probability due to sorafenib was 5% and it increased proportionally with increasing median times to LT and decreasing HR. In the cost-benefit analysis, the incremental NHB of Strategy A versus Strategy B was 37 QALDs; it increased as sorafenib HR decreased and when median times to LT were shorter than 6 months, whereas for longer times it gradually dropped, particularly when Strategy B included effective locoregional treatments. CONCLUSION: Sorafenib neoadjuvant therapy is cost-effective by comparison with no therapy for T2-HCC patients waiting for LT, particularly for median times to LT under 6 months. FAU - Vitale, Alessandro AU - Vitale A AD - Unita di Chirurgia Oncologica, Istituto Oncologico Veneto, IRCCS, Padova, Italy. alessandro.vitale@unipd.it FAU - Volk, Michael L AU - Volk ML FAU - Pastorelli, Davide AU - Pastorelli D FAU - Lonardi, Sara AU - Lonardi S FAU - Farinati, Fabio AU - Farinati F FAU - Burra, Patrizia AU - Burra P FAU - Angeli, Paolo AU - Angeli P FAU - Cillo, Umberto AU - Cillo U LA - eng PT - Journal Article PL - United States TA - Hepatology JT - Hepatology (Baltimore, Md.) JID - 8302946 RN - 0 (Antineoplastic Agents) RN - 0 (Benzenesulfonates) RN - 0 (Phenylurea Compounds) RN - 0 (Pyridines) RN - 25X51I8RD4 (Niacinamide) RN - 9ZOQ3TZI87 (Sorafenib) SB - IM CIN - Hepatology. 2010 Jan;51(1):12-5. PMID: 20034046 CIN - Hepatology. 2010 Jun;51(6):2232-3. PMID: 20041410 CIN - Hepatology. 2010 Sep;52(3):1171-2. PMID: 20812364 CIN - J Hepatol. 2012 Mar;56(3):723-5. PMID: 22127280 MH - Antineoplastic Agents/*therapeutic use MH - Benzenesulfonates/*therapeutic use/toxicity MH - Carcinoma, Hepatocellular/drug therapy/economics/*therapy MH - Cost-Benefit Analysis MH - Humans MH - Liver Transplantation MH - Markov Chains MH - Models, Theoretical MH - Monte Carlo Method MH - Niacinamide/analogs & derivatives MH - Phenylurea Compounds MH - Pyridines/*therapeutic use/toxicity MH - Quality-Adjusted Life Years MH - Sorafenib MH - Treatment Outcome MH - Waiting Lists EDAT- 2009/10/31 06:00 MHDA- 2010/01/16 06:00 CRDT- 2009/10/31 06:00 PHST- 2009/10/31 06:00 [entrez] PHST- 2009/10/31 06:00 [pubmed] PHST- 2010/01/16 06:00 [medline] AID - 10.1002/hep.23260 [doi] PST - ppublish SO - Hepatology. 2010 Jan;51(1):165-73. doi: 10.1002/hep.23260.