López M Fernanda, Mingot María Eva, Valcárcel David, Vicente García Vicente, Perrin Allison, Campos Tapias Ignasi
Complejo Hospitalario Universitario de A Coruña, A Coruña, España.
Hospital Regional Universitario Carlos Haya, Málaga, España.
Med Clin (Barc). 2015 May 8;144(9):389-96. doi: 10.1016/j.medcli.2013.11.035. Epub 2014 Feb 22.
Romiplostim, a thrombopoietin-receptor agonist, is approved for second-line use in idiopathic thrombocytopenic purpura (ITP) patients where surgery is contraindicated. Anti-CD20 rituximab, an immunosuppressant, is currently used off-label. This analysis compared the cost per responder for romiplostim versus rituximab in Spain.
A decision analytic model was constructed to estimate the 6-month cost per responding patient (achieving a platelet count≥50×10(9)/l) according to the most robust published data. A systematic literature review was performed to extract response rates from phase 3 randomized controlled trials. Romiplostim patients received weekly injections; rituximab patients received 4 weekly intravenous infusions. Medical resource costs were obtained from Spanish reimbursement lists. Treatment non-responders incurred bleeding-related event (BRE) management costs as reported in clinical trials. Medical resource utilization and clinical practice were based on Spanish treatment guidelines and validated by local clinical experts.
The literature review identified phase 3 romiplostim trials with a response rate of 83%. Due to a lack of phase 3 controlled rituximab trials, a systematic review of studies was selected as the best source, reporting a response rate of 62.5%. The mean cost per patient for romiplostim was €16,289 and €13,459 for rituximab. Rituximab resulted in a 10% higher cost per responder (€21,535 versus €19,625 for romiplostim). Romiplostim use reduced drug administration, intravenous immunoglobulin, and bleeding-related costs compared to rituximab.
Due to its high level of efficacy leading to lower BRE costs, romiplostim represents an efficient use of resources for adult ITP patients in the Spanish Healthcare System.
罗米司亭是一种血小板生成素受体激动剂，已被批准用于手术禁忌的特发性血小板减少性紫癜（ITP）患者的二线治疗。抗CD20利妥昔单抗是一种免疫抑制剂，目前为非标签用药。本分析比较了西班牙罗米司亭与利妥昔单抗的每位缓解者成本。
根据已发表的最可靠数据构建决策分析模型，以估计每位缓解患者（血小板计数≥50×10⁹/L）的6个月成本。进行系统的文献综述，以从3期随机对照试验中提取缓解率。罗米司亭患者每周注射一次；利妥昔单抗患者接受4次每周一次的静脉输注。医疗资源成本来自西班牙报销清单。治疗无反应者产生了临床试验中报告的出血相关事件（BRE）管理成本。医疗资源利用和临床实践基于西班牙治疗指南，并经当地临床专家验证。
文献综述确定3期罗米司亭试验的缓解率为83%。由于缺乏3期利妥昔单抗对照试验，选择了一项系统综述研究作为最佳来源，报告缓解率为62.5%。罗米司亭每位患者的平均成本为16,289欧元，利妥昔单抗为13,459欧元。利妥昔单抗导致每位缓解者的成本高出10%（罗米司亭为19,625欧元，利妥昔单抗为21,535欧元）。与利妥昔单抗相比，使用罗米司亭可降低药物给药、静脉注射免疫球蛋白和出血相关成本。
由于罗米司亭疗效高，可降低BRE成本，在西班牙医疗保健系统中，它是成人ITP患者资源的有效利用方式。
