Golub Robert, Adams Jared, Dave Sundeep, Bennett Charles L
Department of Medicine, Northwestern University Fernberg School of Medicine, Chicago, IL, USA.
Semin Oncol. 2002 Jun;29(3 Suppl 10):28-32. doi: 10.1053/sonc.2002.33758.
Factors that influence the choice of anagrelide, hydroxyurea, or interferon-alfa (IFN-alpha) for treatment of essential thrombocythemia include efficacy, toxicity, and cost. Anagrelide has the US Food and Drug Administration's approval to be used for treating patients with thrombocythemia secondary to chronic myeloproliferative disorders. In contrast, the use of IFN-alpha and hydroxyurea are considered "off-label." We performed an incremental cost-effectiveness analysis to compare anagrelide, hydroxyurea, and IFN-alpha for treating essential thrombocythemia, in terms of estimated impact on life expectancy. The case used for this analysis was of a 40-year-old man with essential thrombocythemia. Clinical assumptions were based on information obtained from nonrandomized clinical trials, and the economic assumptions were derived from information abstracted from observational studies. Lifelong treatment use of anagrelide versus hydroxyurea would cost approximately $72,000 per additional year of life gained, while the use of IFN-alpha was found to be both more costly and less effective than anagrelide. The results were very sensitive to the risk of leukemia caused by hydroxyurea, with an incremental cost-effectiveness of anagrelide compared with hydroxyurea of $156,969 per additional year of life gained if the lifetime leukemia risk drops from a baseline of .08 to.05. Given that many commonly used medical interventions cost in the range of $50,000 to $100,000 per year of life gained, and the generally poor outcome associated with treatment-related leukemia that can result from hydroxyurea, anagrelide could be considered a therapeutic alternative that is clinically effective at an acceptable cost.
影响选择阿那格雷、羟基脲或干扰素-α（IFN-α）治疗原发性血小板增多症的因素包括疗效、毒性和成本。阿那格雷已获美国食品药品监督管理局批准用于治疗慢性骨髓增殖性疾病继发的血小板增多症患者。相比之下，IFN-α和羟基脲的使用被认为是“非适应证用药”。我们进行了一项增量成本效益分析，以比较阿那格雷、羟基脲和IFN-α治疗原发性血小板增多症对预期寿命的估计影响。该分析所采用的案例是一名40岁的原发性血小板增多症男性患者。临床假设基于从非随机临床试验中获得的信息，经济假设则源自从观察性研究中提取的信息。与羟基脲相比，终身使用阿那格雷每多获得一年生命的成本约为72,000美元，而使用IFN-α被发现比阿那格雷成本更高且效果更差。结果对羟基脲所致白血病风险非常敏感，如果终身白血病风险从基线的0.08降至0.05，与羟基脲相比，阿那格雷每多获得一年生命的增量成本效益为156,969美元。鉴于许多常用的医疗干预措施每获得一年生命的成本在50,000美元至100,000美元之间，且羟基脲可能导致与治疗相关的白血病，总体预后较差，阿那格雷可被视为一种在可接受成本下具有临床疗效的治疗选择。
