Student Research Committee, School of Management and Medical Information Sciences, Shiraz University of Medical Sciences, Shiraz, Iran.
Hematology Research Center, Shiraz University of Medical Sciences, Shiraz, Iran.
Hematology. 2021 Dec;26(1):240-248. doi: 10.1080/16078454.2021.1885123.
This study aimed to compare the cost-effectiveness of these two regimens in hemophilia A patients, under-12-years-old in southern Iran.
A cost-effectiveness study comparing prophylaxis versus on-demand was conducted on 34 hemophilia patients (24 and 10 patients were on the prophylaxis and on-demand regimens respectively) in 2017. The Markov model was used to estimate the economic and clinical outcomes. The costs were collected from the societal perspective, and the utility criterion was the 'quality adjusted life year (QALY)' indicator. The required data were collected using a researcher-made cost checklist, the EQ5D standard questionnaire and Hemophilia Joint Health Score. The probabilistic sensitivity analysis (PSA) was performed to determine the robustness of the results.
The means of costs, joint health score and QALY in the prophylaxis regimen were $478,963.1 purchasing power parity (PPP), 96.67, and 11.98 respectively, and in the on-demand regimen were $521,797.2 PPP, 93.46 and 10.99 respectively. The PSA confirmed the robustness of the model's results. The results of the scatter plots and acceptability curves showed that the prophylaxis regimen in 97% of the simulations for the thresholds below $20950 PPP was more cost-effective than on-demand regimen.
Prophylaxis regimen showed the lower costs and higher effectiveness and utility in comparison with the on-demand regimen. It is recommended that prophylaxis should be considered as the standard care for treatment of hemophilic patients.
本研究旨在比较伊朗南部 12 岁以下甲型血友病患者中这两种方案的成本效益。
2017 年对 34 名血友病患者（分别有 24 名和 10 名患者接受预防治疗和按需治疗）进行了成本效益比较研究。使用马尔可夫模型估计经济和临床结果。从社会角度收集成本，使用效用标准“质量调整生命年（QALY）”指标。使用研究者自制的成本清单、EQ5D 标准问卷和血友病关节健康评分收集所需数据。进行概率敏感性分析（PSA）以确定结果的稳健性。
预防方案的平均成本、关节健康评分和 QALY 分别为 478963.1 美元（购买力平价）、96.67 和 11.98，按需方案分别为 521797.2 美元、93.46 和 10.99。PSA 证实了模型结果的稳健性。散点图和可接受性曲线的结果表明，在低于 20950 美元的治疗阈值下，97%的模拟场景中，预防方案比按需方案更具成本效益。
与按需方案相比，预防方案显示出较低的成本、更高的效果和效用。建议将预防方案作为治疗血友病患者的标准护理。
