Cooper Nichola, Guterres Sebastian, Pochopień Michał, Wilson Koo, James Sam, Toumi Mondher, Tytuła Anna, Rich Carly, Eriksson Daniel
Faculty of Medicine, Department of Immunology and Inflammation, Imperial College London, London SW7 2AZ, UK.
Swedish Orphan Biovitrum Ltd., Cambridge CB21 6AD, UK;
J Mark Access Health Policy. 2025 Mar 24;13(2):11. doi: 10.3390/jmahp13020011. eCollection 2025 Jun.
Thrombopoietin receptor agonists-romiplostim, eltrombopag and avatrombopag-are commonly used as second-line treatments for immune thrombocytopenia (ITP).
A Markov model was developed to estimate the cost effectiveness of the three TPO-RAs in adults with insufficient response to previous treatment from the perspective of the UK National Health Service (NHS). The model considered the effects of bleeding events, concomitant ITP medications, rescue therapies and treatment related adverse events over a lifetime horizon. Model inputs for effectiveness were based on a network meta-analysis and other published literature on ITP management. Other model inputs included costs (e.g., drug acquisition and administration) and healthcare resource utilisation.
Avatrombopag was associated with higher quality-adjusted life-years (QALYs) (10.979) than romiplostim (10.628) and eltrombopag (10.085), producing incremental QALYs of -0.351 and -0.894, respectively. Avatrombopag was associated with lower total costs (GBP £319,334) compared with romiplostim (GBP 406,361 [cost saving of GBP 87,027]) and higher total costs compared with eltrombopag (GBP 313,987 [incremental cost of GBP 5347]). Avatrombopag therefore dominated romiplostim (more effective and less expensive) and was cost-effective versus eltrombopag (incremental cost-effectiveness ratio of GBP 5982 per QALY).
Avatrombopag is a cost-effective treatment compared with romiplostim and eltrombopag for the second-line treatment of adults with ITP from the perspective of the UK NHS.
血小板生成素受体激动剂——罗米司亭、艾曲泊帕和阿伐曲泊帕——通常用作免疫性血小板减少症（ITP）的二线治疗药物。
建立了一个马尔可夫模型，从英国国家医疗服务体系（NHS）的角度评估这三种血小板生成素受体激动剂（TPO-RAs）在既往治疗反应不足的成年患者中的成本效益。该模型考虑了出血事件、ITP伴随用药、挽救治疗以及一生时间范围内与治疗相关的不良事件的影响。有效性的模型输入基于一项网状荟萃分析以及其他关于ITP管理的已发表文献。其他模型输入包括成本（如药物采购和给药）以及医疗资源利用情况。
与罗米司亭（10.628）和艾曲泊帕（10.085）相比，阿伐曲泊帕的质量调整生命年（QALYs）更高（10.979），分别产生了-0.351和-0.894的增量QALYs。与罗米司亭（406,361英镑[节省成本87,027英镑]）相比，阿伐曲泊帕的总成本更低（319,334英镑），与艾曲泊帕（313,987英镑[增量成本5347英镑]）相比总成本更高。因此，阿伐曲泊帕优于罗米司亭（更有效且成本更低），与艾曲泊帕相比具有成本效益（每QALY的增量成本效益比为5982英镑）。
从英国NHS的角度来看，对于成人ITP的二线治疗，与罗米司亭和艾曲泊帕相比，阿伐曲泊帕是一种具有成本效益的治疗方法。
