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EMICIZUMAB DOSING IN CHILDREN AND ADULTS WITH HEMOPHILIA A: SIMULATING A USER-FRIENDLY AND COST-EFFICIENT REGIMEN
Thrombosis and Haemostasis ( IF 6.7 ) Pub Date : 2021-05-04 , DOI: 10.1055/a-1499-0030
Laura H Bukkems 1 , Kathelijn Fischer 2 , Idske Kremer-Hovinga 2 , Anouk A M Donners 3 , Karin Fijnvandraat 4 , Roger E G Schutgens 5 , Marjon H Cnossen 6 , Ron A A Mathôt 1
Affiliation  

Background: When emicizumab is dosed according to label, clinicians are obligated to discard or overdose medication due to discrepancies between calculated dose and vial content. The aim of this study was to compose a cost-efficient emicizumab maintenance dosing regimen using Monte Carlo simulation based on vial size, patient friendly intervals and patient characteristics, while striving for similar plasma concentrations as observed in clinical trials. Methods: Monte Carlo simulations were used to investigate alternative dosing regimens in patients weighing 3 to 150 kg. Simulated regimens were targeted to achieve median emicizumab plasma concentrations at steady state (Cav,ss) of 40-60 (90% range: 25-95) µg/ml. The cost-efficiency of the alternative dosing regimen was calculated in mg and costs saved per patient per year. Results: The developed alternative dosing regimen achieved similar emicizumab Cav,ss levels compared to the registered dosing regimen with a median deviation of less than 2 µg/mL in 78 % of the bodyweight categories. A dose of 60 mg every 3 weeks was advised for children weighing 12–16 kg, while adults weighing 76–85 kg can receive 120 mg emicizumab every week. Compared to the registered weekly dosing of 1.5 mg/kg, alternative dosing saved €35,434 per year in children weighing between 12 and 16 kg. For patients weighing 76–85 kg the median saving was €29,529 (range: €0 – €59,057). Conclusion: This alternative maintenance dosing scheme - applicable in patients with hemophilia A receiving emicizumab prophylaxis - reduces financial costs, avoids medication spillage and is patient friendly without loss of efficacy.

中文翻译:

在患有血友病 A 的儿童和成人中使用 EMICIZUMAB:模拟一种用户友好且具有成本效益的方案

背景:根据标签给予 emicizumab 时,由于计算的剂量和药瓶内容物之间的差异,临床医生有义务丢弃或过量用药。本研究的目的是根据药瓶大小、患者友好间隔和患者特征,使用 Monte Carlo 模拟制定具有成本效益的 emicizumab 维持给药方案,同时努力获得与临床试验中观察到的相似的血浆浓度。方法:蒙特卡罗模拟用于研究体重 3 至 150 kg 患者的替代给药方案。模拟方案的目标是在稳态 (Cav,ss) 达到 40-60(90% 范围:25-95)μg/ml 的中位 emicizumab 血浆浓度。替代给药方案的成本效益以毫克计算,每位患者每年节省的成本。结果:与注册的给药方案相比,开发的替代给药方案达到了相似的 emicizumab Cav,ss 水平,在 78% 的体重类别中,中位偏差小于 2 µg/mL。建议体重 12-16 公斤的儿童每 3 周服用 60 毫克,而体重 76-85 公斤的成年人每周可以服用 120 毫克 emicizumab。与注册的每周剂量 1.5 毫克/公斤相比,替代剂量每年可为体重在 12 至 16 公斤之间的儿童节省 35,434 欧元。对于体重 76-85 公斤的患者,中位节省为 29,529 欧元(范围:0 - 59,057 欧元)。结论:这种替代维持给药方案 - 适用于接受 emicizumab 预防的血友病 A 患者 - 降低了财务成本,避免了药物溢出,并且对患者友好而不会失去疗效。
更新日期:2021-06-25
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