Statistics Department, University of Barcelona, Barcelona, Spain.
Clin Ther. 2012 Aug;34(8):1774-87. doi: 10.1016/j.clinthera.2012.06.029. Epub 2012 Jul 25.
Chronic graft-versus-host disease (cGvHD) is the leading cause of late nonrelapse mortality (transplant-related mortality) after hematopoietic stem cell transplant. Given that there are a wide range of treatment options for cGvHD, assessment of the associated costs and efficacy can help clinicians and health care providers allocate health care resources more efficiently.
The purpose of this study was to assess the cost-effectiveness of extracorporeal photopheresis (ECP) compared with rituximab (Rmb) and with imatinib (Imt) in patients with cGvHD at 5 years from the perspective of the Spanish National Health System.
The model assessed the incremental cost-effectiveness/utility ratio of ECP versus Rmb or Imt for 1000 hypothetical patients by using microsimulation cost-effectiveness techniques. Model probabilities were obtained from the literature. Treatment pathways and adverse events were evaluated taking clinical opinion and published reports into consideration. Local data on costs (2010 Euros) and health care resources utilization were validated by the clinical authors. Probabilistic sensitivity analyses were used to assess the robustness of the model.
The greater efficacy of ECP resulted in a gain of 0.011 to 0.024 quality-adjusted life-year in the first year and 0.062 to 0.094 at year 5 compared with Rmb or Imt. The results showed that the higher acquisition cost of ECP versus Imt was compensated for at 9 months by greater efficacy; this higher cost was partially compensated for (€517) by year 5 versus Rmb. After 9 months, ECP was dominant (cheaper and more effective) compared with Imt. The incremental cost-effectiveness ratio of ECP versus Rmb was €29,646 per life-year gained and €24,442 per quality-adjusted life-year gained at year 2.5. Probabilistic sensitivity analysis confirmed the results. The main study limitation was that to assess relative treatment effects, only small studies were available for indirect comparison.
ECP as a third-line therapy for cGvHD is a more cost-effective strategy than Rmb or Imt.
慢性移植物抗宿主病（cGvHD）是造血干细胞移植后晚期非复发相关死亡率（移植相关死亡率）的主要原因。鉴于 cGvHD 有广泛的治疗选择，评估相关成本和疗效可以帮助临床医生和医疗保健提供者更有效地分配医疗保健资源。
本研究旨在从西班牙国家卫生系统的角度评估与利妥昔单抗（Rmb）和伊马替尼（Imt）相比，体外光分离术（ECP）在 cGvHD 患者 5 年后的成本效益。
该模型通过使用微模拟成本效益技术，评估了 ECP 与 Rmb 或 Imt 相比，对 1000 名假想患者的增量成本效益/效用比。模型概率来自文献。治疗途径和不良事件在考虑临床意见和已发表报告的基础上进行了评估。当地成本（2010 年欧元）和卫生保健资源利用数据由临床作者进行了验证。概率敏感性分析用于评估模型的稳健性。
与 Rmb 或 Imt 相比，ECP 的更高疗效导致第一年获得 0.011 至 0.024 个质量调整生命年，第 5 年获得 0.062 至 0.094 个质量调整生命年。结果表明，与 Imt 相比，ECP 较高的购置成本在 9 个月时被更高的疗效所弥补；到第 5 年，与 Rmb 相比，这一较高的成本部分得到了弥补（517 欧元）。9 个月后，与 Imt 相比，ECP 更具成本效益（更便宜、更有效）。与 Rmb 相比，ECP 每增加 1 个生命年的增量成本效益比为 29646 欧元，每增加 1 个质量调整生命年的增量成本效益比为 24442 欧元，在第 2.5 年。概率敏感性分析证实了这些结果。本研究的主要局限性是，为了评估相对治疗效果，只能对小样本量的研究进行间接比较。
作为 cGvHD 的三线治疗方法，ECP 比 Rmb 或 Imt 更具成本效益。
