Abacus International, 6 Talisman Business Centre, Talisman Road, Bicester, Oxfordshire, OX26 6HR, UK.
Pharmacoeconomics. 2013 Sep;31(9):807-22. doi: 10.1007/s40273-013-0076-z.
Patients with β-thalassaemia major experience chronic iron overload due to regular blood transfusions. Chronic iron overload can be treated using iron-chelating therapies such as desferrioxamine (DFO), deferiprone (DFP) and deferasirox (DFX) monotherapy, or DFO-DFP combination therapy.
This study evaluated the relative cost effectiveness of these regimens over a 5-year timeframe from a UK National Health Service (NHS) perspective, including personal and social services.
A Markov model was constructed to evaluate the cost effectiveness of the treatment regimens over 5 years. Based on published randomized controlled trial evidence, it was assumed that all four treatment regimens had a comparable effect on serum ferritin concentration (SFC) and liver iron concentration (LIC), and that DFP was more effective for reducing cardiac morbidity and mortality. Published utility scores for route of administration were used, with subcutaneously administered DFO assumed to incur a greater quality of life (QoL) burden than the oral chelators DFP and DFX. Healthcare resource use, drug costs (2010/2011 costs), and utilities associated with adverse events were also considered, with the effect of varying all parameters assessed in sensitivity analysis. Incremental costs and quality-adjusted life-years (QALYs) were calculated for each treatment, with cost effectiveness expressed as incremental cost per QALY. Assumptions that DFP conferred no cardiac morbidity, mortality, or morbidity and mortality benefit were also explored in scenario analysis.
DFP was the dominant strategy in all scenarios modelled, providing greater QALY gains at a lower cost. Sensitivity analysis showed that DFP dominated all other treatments unless the QoL burden associated with the route of administration was greater for DFP than for DFO, which is unlikely to be the case. DFP had >99 % likelihood of being cost effective against all comparators at a willingness-to-pay threshold of £20,000 per QALY.
In this analysis, DFP appeared to be the most cost-effective treatment available for managing chronic iron overload in β-thalassaemia patients. Use of DFP in these patients could therefore result in substantial cost savings.
患有重型β地中海贫血症的患者由于定期输血而经历慢性铁过载。慢性铁过载可以通过铁螯合疗法来治疗，例如去铁胺（DFO）、地拉罗司（DFX）单药治疗或 DFO-DFP 联合治疗。
本研究从英国国家医疗服务体系（NHS）的角度评估了这些方案在 5 年内的相对成本效益，包括个人和社会服务。
构建了一个 Markov 模型来评估治疗方案在 5 年内的成本效益。基于已发表的随机对照试验证据，假设所有四种治疗方案对血清铁蛋白浓度（SFC）和肝脏铁浓度（LIC）的效果相当，且地拉罗司在降低心脏发病率和死亡率方面更为有效。使用了已发表的给药途径效用评分，假设皮下给予 DFO 比口服螯合剂地拉罗司和地拉罗司的生活质量（QoL）负担更大。还考虑了医疗资源的使用、药物成本（2010/2011 年成本）以及与不良事件相关的效用，通过敏感性分析评估了所有参数变化的影响。为每种治疗方案计算了增量成本和质量调整生命年（QALY），并以增量成本每 QALY 表示成本效益。还在情景分析中探讨了地拉罗司不具有心脏发病率、死亡率或发病率和死亡率获益的假设。
在地拉罗司在所有建模的情景中均为占主导地位的策略，以较低的成本提供了更大的 QALY 获益。敏感性分析表明，除非地拉罗司的给药途径 QoL 负担大于 DFO，否则地拉罗司将主导所有其他治疗方案，而这不太可能。在地拉罗司相对于其他所有比较剂的意愿支付阈值为 20,000 英镑/QALY 的情况下，地拉罗司具有超过 99%的可能性具有成本效益。
在这项分析中，地拉罗司似乎是治疗β地中海贫血症患者慢性铁过载的最具成本效益的治疗方法。在这些患者中使用地拉罗司可能会带来显著的成本节约。
