Reed Shelby D, Anstrom Kevin J, Ludmer Jennifer A, Glendenning G Alastair, Schulman Kevin A
Center for Clinical and Genetic Economics, Duke Clinical Research Institute, Duke University Medical Center, Durham, North Carolina, USA.
Cancer. 2004 Dec 1;101(11):2574-83. doi: 10.1002/cncr.20694.
Despite a lack of long-term data, imatinib has become standard therapy for patients with newly diagnosed chronic-phase chronic myeloid leukemia (CML) who are not candidates for allogeneic stem cell transplantation. In the current study, the authors estimated the incremental cost-effectiveness of imatinib versus interferon-alpha plus low-dose cytarabine (IFN+LDAC) as first-line therapy for these patients.
Data from the International Randomized Interferon versus STI571 Study and the literature were used to estimate lifetime costs, survival, and quality-adjusted survival. Survival estimates were based on published survival curves for patients who achieved and those who did not achieve a complete cytogenetic response after treatment with interferon-alpha.
The mean estimated survival with first-line imatinib therapy was 15.30 years, compared with 9.07 years with IFN+LDAC. Undiscounted lifetime costs were approximately $424,600 with imatinib and $182,800 with IFN+LDAC. Using a 3% discount rate, the incremental survival gain with imatinib was 3.93 life-years and 3.89 quality-adjusted life-years (QALYs). Incremental discounted lifetime costs were found to be $168,100 higher with imatinib, resulting in incremental cost-effectiveness ratios of $43,100 per life-year saved (95% confidence interval [95% CI], $37,600-51,100) and $43,300 per QALY (95% CI, $38,300-49,100).
The results of the current study demonstrate that compared with IFN+LDAC, imatinib is a cost-effective first-line therapy in patients with newly diagnosed chronic-phase CML.
尽管缺乏长期数据,但伊马替尼已成为不适于接受异基因干细胞移植的新诊断慢性期慢性髓性白血病(CML)患者的标准治疗方法。在本研究中,作者评估了伊马替尼与α干扰素加小剂量阿糖胞苷(IFN+LDAC)作为这些患者一线治疗的增量成本效益。
采用国际随机干扰素与STI571研究及文献中的数据来估计终生成本、生存率和质量调整生存率。生存率估计基于已发表的α干扰素治疗后达到和未达到完全细胞遗传学缓解的患者的生存曲线。
一线伊马替尼治疗的平均估计生存期为15.30年,而IFN+LDAC治疗为9.07年。伊马替尼的未贴现终生成本约为424,600美元,IFN+LDAC为182,800美元。采用3%的贴现率,伊马替尼的增量生存获益为3.93生命年和3.89质量调整生命年(QALY)。发现伊马替尼的增量贴现终生成本高出168,100美元,导致增量成本效益比为每挽救1生命年43,100美元(95%置信区间[95%CI],37,600 - 51,100美元)和每QALY 43,300美元(95%CI,38,300 - 49,100美元)。
本研究结果表明,与IFN+LDAC相比,伊马替尼是新诊断慢性期CML患者具有成本效益的一线治疗方法。