RTI Health Solutions, Research Triangle Park, NC, USA.
Grifols, Inc., Research Commons, NC, USA.
Haemophilia. 2015 May;21(3):310-319. doi: 10.1111/hae.12621. Epub 2015 Feb 16.
Development of inhibitors (alloantibodies to exogenous factor VIII) is the most significant treatment complication in patients with haemophilia A. The only proven way to eradicate inhibitors is through immune tolerance induction (ITI), while bypassing agents are typically employed to treat or prevent bleeds in patients with high titre inhibitors. Costs of these approaches have not been well studied. The aim of this study was to compare lifetime costs of treating patients with severe haemophilia A with inhibitors using on-demand or prophylaxis treatment with bypassing agents and ITI. A decision-analytic model was developed to compare the treatment costs and outcomes. Quantitation of the reduction in bleeding events for patients on prophylaxis and after eradication of inhibitors when on ITI and relapse of inhibitors was derived from published studies. Costs were obtained from standard US costing sources and are reported in 2014 US dollars. Costs and outcomes were discounted 3% per annum. Lifetime costs of treating patients with inhibitors are lower for ITI vs. on-demand or prophylaxis. Patients are also projected to live longer, have greater quality-adjusted life-years, and have fewer bleeding events than patients treated on-demand. Treating patients via ITI to eradicate inhibitors may result in lower lifetime costs and greater life-years and quality-adjusted life-years than treating with bypassing agents.
抑制剂（对外源性因子 VIII 的同种抗体）的产生是 A 型血友病患者治疗中最严重的并发症。消除抑制剂的唯一有效方法是通过免疫耐受诱导（ITI），而旁路制剂通常用于治疗或预防高滴度抑制剂患者的出血。这些方法的成本尚未得到充分研究。本研究旨在比较按需或预防治疗与旁路制剂和 ITI 联合使用治疗有抑制剂的重度 A 型血友病患者的终生成本。开发了一个决策分析模型来比较治疗成本和结果。从已发表的研究中得出了接受预防治疗和 ITI 治疗后抑制剂消除以及抑制剂复发的患者出血事件减少的定量数据。成本来自标准的美国成本源，并以 2014 年美元报告。成本和结果按每年 3%贴现。与按需或预防治疗相比，使用 ITI 治疗抑制剂患者的终生成本更低。与按需治疗的患者相比，接受 ITI 治疗以消除抑制剂的患者预计寿命更长，具有更高的质量调整生命年，且出血事件更少。通过 ITI 治疗消除抑制剂可能会导致较低的终生成本和更多的寿命年和质量调整生命年，而不是使用旁路制剂治疗。
