Chiche L, Perrin A, Stern L, Kutikova L, Cohen-Nizard S, Lefrère F
Service de médecine interne, centre de compétence PACA ouest pour la prise en charge des cytopénies autoimmunes, hôpital de la Conception, Aix-Marseille université, 13005 Marseille, France.
LA-SER Analytica, 24, West 40th Street, Floor 8, 10018 New York, États-Unis.
Transfus Clin Biol. 2014 May;21(2):85-93. doi: 10.1016/j.tracli.2014.03.007. Epub 2014 May 3.
This analysis compared the response rates and cost per responder associated with romiplostim and rituximab in adult immune thrombocytopenia from the French National Health System payer perspective.
A decision analytic model was developed to estimate the cost per patient and per responder of treating adult immune thrombocytopenia patients with romiplostim versus rituximab over 6 months. A systematic literature review identified phase 3 randomized controlled trials. Published response rates were extracted (response definition: ≥50×10(9) platelets/liter). Resource utilization was based on French and international treatment guidelines, and clinical expert opinion. Unit costs were derived from literature and French reimbursement lists, and included the costs of routine physician visits, treatment administration, and emergency care. Non-responders incurred bleeding-related event costs.
The literature review identified a phase 3 randomized controlled trial for romiplostim with a response rate of 83%. Due to a lack of phase 3 randomized controlled trials for rituximab, a systematic review of studies was selected as the best source, reporting a response rate of 62.5%. Romiplostim and rituximab were associated with similar treatment costs, with an estimated cost per patient for romiplostim of €17,456 and €17,068 for rituximab. Rituximab resulted in a 30% higher cost per responder (€27,308 for rituximab versus €21,031 for romiplostim). Romiplostim use reduced drug administration, intravenous immunoglobulin, and bleeding-related hospitalization costs compared to rituximab.
Due to its high efficacy leading to lower bleeding-related costs, romiplostim represents an efficient use of resources for adult immune thrombocytopenia patients in the French healthcare system.
本分析从法国国家卫生系统支付方的角度，比较了罗米司亭和利妥昔单抗治疗成人免疫性血小板减少症的缓解率及每位缓解者的成本。
建立了一个决策分析模型，以估算在6个月内用罗米司亭与利妥昔单抗治疗成人免疫性血小板减少症患者的每位患者及每位缓解者的成本。通过系统文献回顾确定了3期随机对照试验。提取已发表的缓解率（缓解定义：血小板计数≥50×10⁹/升）。资源利用基于法国和国际治疗指南以及临床专家意见。单位成本源自文献和法国报销清单，包括常规门诊就诊、治疗给药和急诊护理的成本。未缓解者产生与出血相关事件的成本。
文献回顾确定了一项罗米司亭的3期随机对照试验，缓解率为83%。由于缺乏利妥昔单抗的3期随机对照试验，选择了一项研究的系统评价作为最佳来源，报告的缓解率为62.5%。罗米司亭和利妥昔单抗的治疗成本相似，罗米司亭的估计每位患者成本为17,456欧元，利妥昔单抗为17,068欧元。利妥昔单抗导致每位缓解者的成本高出30%（利妥昔单抗为27,308欧元，罗米司亭为21,031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与出血相关的住院成本。
由于罗米司亭疗效高，可降低与出血相关的成本，在法国医疗系统中，它是成人免疫性血小板减少症患者资源的有效利用方式。
