Tremblay Gabriel, Dolph Mike, Bhor Menaka, Said Qayyim, Elliott Brian, Briggs Andrew
Department of Health Economics, Purple Squirrel Economics, New York, NY, USA,
Department of Health Economics and Outcomes Research, Novartis Pharmaceuticals, East Hanover, NJ, USA.
Clinicoecon Outcomes Res. 2018 Nov 1;10:705-713. doi: 10.2147/CEOR.S177324. eCollection 2018.
Thrombopoietin-receptor agonists eltrombopag (EPAG) and romiplostim (ROMI) are treatment options for adults with chronic immune thrombocytopenia (cITP) who have had an insufficient response to corticosteroids or immunoglobulins.
A cost-consequence model was developed to evaluate the costs relative to treatment success of EPAG, ROMI, and watch and rescue (W&R) in previously treated patients. The primary endpoint assessed was severe bleeding, derived from all identified phase III registered clinical trials. Health outcomes were compared via indirect treatment comparison. Costs incorporated in the model included drug and administration, routine care, rescue medications, bleeding-related adverse events, other adverse events, and mortality costs. A trial (26-week) time horizon was used, as certain endpoints used in the model were bound to within-trial results.
In the intent-to-treat (ITT) population, the overall estimated cost per patient for EPAG was US$66,560 compared to US$91,039 for ROMI and US$30,099 for W&R. Compared to the ITT population, the difference in cost between EPAG and ROMI was slightly greater in splenectomized patients (US$65,998 for EPAG compared to US$91,485 for ROMI) and slightly less in non-splenectomized patients (US$67,151 for EPAG compared to US$91,455 for ROMI), though the overall trend remained the same. When assessing cost per severe bleeding event avoided in the ITT population, EPAG dominated (less expensive, more effective) ROMI. Sensitivity analyses confirmed these results.
EPAG was preferred over ROMI in the treatment of cITP, largely driven by the reduction in severe bleeding events associated with its use.
血小板生成素受体激动剂艾曲泊帕（EPAG）和罗米司亭（ROMI）是对皮质类固醇或免疫球蛋白反应不足的慢性免疫性血小板减少症（cITP）成人患者的治疗选择。
建立了一个成本-后果模型，以评估EPAG、ROMI以及观察与救援（W&R）在既往治疗患者中相对于治疗成功的成本。评估的主要终点是严重出血，源自所有已识别的III期注册临床试验。通过间接治疗比较来比较健康结局。模型中纳入的成本包括药物及给药、常规护理、救援药物、出血相关不良事件、其他不良事件以及死亡成本。采用了一个试验（26周）的时间范围，因为模型中使用的某些终点受试验内结果的限制。
在意向性治疗（ITT）人群中，EPAG每位患者的总体估计成本为66,560美元，而ROMI为91,039美元，W&R为30,099美元。与ITT人群相比，脾切除患者中EPAG和ROMI之间的成本差异略大（EPAG为65,998美元，ROMI为91,485美元），非脾切除患者中略小（EPAG为67,151美元，ROMI为91,455美元），不过总体趋势保持不变。在评估ITT人群中避免每例严重出血事件的成本时，EPAG优于（成本更低、效果更好）ROMI。敏感性分析证实了这些结果。
在cITP的治疗中，EPAG优于ROMI，这主要是由于使用EPAG可减少严重出血事件。
