Use of Sorafenib in Patients With Hepatocellular Carcinoma Before Liver Transplantation: A Cost-Benefit Analysis While Awaiting Data on Sorafenib Safety
Use of Sorafenib in Patients With Hepatocellular Carcinoma Before Liver Transplantation: A Cost-Benefit Analysis While Awaiting Data on Sorafenib Safety
复制标题
DOI：
10.1002/hep.23260
复制
发表时间：
2010-01-01
期刊：
影响因子：
13.5
通讯作者：
Cillo, Umberto
中科院分区：
文献类型：
--
作者：
Vitale, Alessandro;Volk, Michael L.;Cillo, Umberto
桥接疗法在肝移植（LT）等候名单上的肝细胞癌（HCC）患者中的作用仍存在争议。有强有力的证据支持索拉非尼延长HCC进展时间的有效性。使用马尔可夫模型，我们比较了两种策略：一种是在LT前使用索拉非尼B作为新辅助治疗（策略A），另一种是在前6个月不使用桥接治疗（策略B）。参考病例：T2 HCC伴代偿性肝硬化患者。索拉非尼在延迟HCC进展时间方面的获益以风险比（HR）表示，并来自最近发表的随机试验。考虑的终点为：以定量调整5天（QALDs）衡量的生存效益、移植概率、C中的成本（C）、支付意愿（WTP）和净健康效益（NHB），其中NHB =生存效益- C/WTP。索拉非尼在意大利的WTP计算值为346（原文如此）/QALD。概率敏感性分析显示中位生存获益为94 QALD（10%百分位数= 38，90%百分位数= 210）。在基本情况下，（HR = 0.47，每月脱落概率= 5%，中位LT时间= 3个月），索拉非尼B导致的LT概率增加为5%，且随着中位LT时间的增加和HR的降低而成比例增加。在成本效益分析中，策略A与策略B相比，NHB增量为37 QALD;随着索拉非尼B HR降低和中位至LT时间短于6个月，其增加，而对于较长时间，其逐渐下降，特别是当策略B包括有效的局部治疗时。结论：索拉非尼新辅助治疗对于等待肝移植的T2-HCC患者，尤其是对于中位肝移植时间低于6个月的患者，与未治疗相比具有成本效益。（肝脏学2010，51：165-173。）
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 quatity-adjusted fife days (QALDs), transplant probability, costs (C) in C, willingness to pay (WTP), and net health benefit (NHB), where NHB = survival benefit - C/WTP. The calculated WTP of sorafenib in Italy was 346 (sic) 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. (HEPATOLOGY 2010,51:165-173.)