Department of Pediatrics, Shin Kong Wu Ho-Su Memorial Hospital, Taipei, Taiwan.
J Formos Med Assoc. 2013 Apr;112(4):221-9. doi: 10.1016/j.jfma.2011.08.020. Epub 2012 Apr 30.
BACKGROUND/PURPOSE: The newly available iron chelator deferasirox (Exjade, Novartis) is expected to provide better long-term clinical outcomes and improved quality of life for patients with thalassemia than its predecessor, deferoxamine (Desferal, Novartis), because of its oral tablet form.
We used the Markov model to estimate total additional lifetime costs and quality-adjusted life years (QALYs) gained with deferasirox versus deferoxamine in patients with transfusion-dependent thalassemia. Patients were assumed to be 2 years of age at initiation of chelation therapy. Clinical outcomes in terms of morbidity and mortality from associated complications and life expectancy for the study population were estimated using the databases of the Bureau of National Health Insurance and the Health and Vital Statistics of Taiwan. Treatment costs were based on analyses of health insurance claims for patients with transfusion-dependent thalassemia. Utilities in terms of quality of life were also included in the model. The incremental cost-utility ratio of deferasirox versus deferoxamine was defined by the ratio of the difference in expected lifetime costs to the difference in QALYs. One-way sensitivity analyses were performed to examine the robustness of the results to key assumptions.
Patients treated with deferasirox are expected to experience a lower incidence of associated complications and obtain 2.3 QALYs (discounted) at an additional lifetime cost of US$36,291 per patient (US$15,596 per QALY). Sensitivity analyses showed that the unit drug cost of deferasirox had the greatest impact on the incremental cost-utility ratio. In addition, the incremental cost-utility ratio will increase by delaying the starting age (2 years of age in our study) of chelation therapy.
Compared with infusional deferoxamine, oral deferasirox improved clinical outcomes and quality of life in terms of iron chelation in transfusion-dependent patients with thalassemia at a reasonable cost from a healthcare perspective.
背景/目的：新型铁螯合剂地拉罗司（Exjade，诺华）因其口服片剂形式，有望为依赖输血的地中海贫血患者提供比其前体去铁胺（Desferal，诺华）更好的长期临床结局和改善生活质量。
我们使用马尔可夫模型来估计地拉罗司与去铁胺相比，在依赖输血的地中海贫血患者中使用时的总额外终生成本和获得的质量调整生命年（QALY）。假设患者在开始螯合治疗时为 2 岁。使用国民健康保险局和台湾卫生与生命统计数据库，估计研究人群的发病率和死亡率以及与相关并发症相关的死亡率以及预期寿命。治疗成本基于对依赖输血的地中海贫血患者的健康保险索赔的分析。模型中还包含了生活质量方面的效用。地拉罗司与去铁胺的增量成本-效用比定义为预期终生成本差异与 QALY 差异的比值。进行了单因素敏感性分析，以检查结果对关键假设的稳健性。
使用地拉罗司治疗的患者预计会经历较低的相关并发症发生率，并获得 2.3 QALY（贴现），每位患者的额外终生成本为 36,291 美元（每 QALY 为 15,596 美元）。敏感性分析表明，地拉罗司的单位药物成本对地拉罗司的增量成本-效用比影响最大。此外，通过延迟螯合治疗的起始年龄（我们研究中的 2 岁），增量成本-效用比将会增加。
从医疗保健角度来看，与输注性去铁胺相比，口服地拉罗司在依赖输血的地中海贫血患者的铁螯合方面改善了临床结局和生活质量，而且具有合理的成本。
