Chiou Shyh-Shin, Lin Ching-Yeh, Weng Te-Fu, Wang Jiaan-Der, Chou Sheng-Chieh, Peng Ching-Tien, Lin Pei-Chin, Liao Yu-Mei, Lai Leanne, Shen Ming-Ching
Taiwan Society of Thrombosis and Hemostasis (TSTH) Study Group, Taipei, Taiwan.
Graduate Institute of Clinical Medicine, College of Medicine, Kaohsiung Medical University Hospital, Kaohsiung, Taiwan.
Drugs Real World Outcomes. 2024 Dec;11(4):711-723. doi: 10.1007/s40801-024-00452-z. Epub 2024 Nov 20.
Hemophilia A (HA) treatment strategies aim to manage bleeding episodes and improve patients' quality of life. This study investigates the effectiveness of a preventative approach using intermediate-dose prophylaxis with standard half-life FVIII products in reducing bleeding rates and enhancing the quality of life for patients with severe HA.
A 4-year prospective longitudinal study followed 35 patients with severe HA (without FVIII inhibitors) who transitioned from a reactive treatment approach to intermediate-dose prophylaxis in Taiwan from 2014 until 2018. The study tracked annual bleeding rates (ABR) and annual joint bleeding rates (AjBR) alongside associated costs and patient-reported quality-of-life measures.
Prophylaxis significantly reduced both ABR and AjBR compared with the previous treatment. After one year, ABR and AjBR decreased by 76.9% and 72.5%, respectively, with further reductions to 91.0% and 90.8% after 4 years (p < 0.001). While the average annual cost of factor VIII concentrate increased by 41.0% in the first year, the incremental cost-effectiveness ratio demonstrated ongoing benefits from ABR avoidance over the 4 years. Additionally, patients reported significant improvements in quality-of-life measures following the switch to prophylaxis (p = 0.036).
Intermediate-dose prophylaxis effectively reduced bleeding rates and improved quality of life in patients with severe HA. Despite initial cost increases, the intervention became cost effective over time. This study provides valuable data for healthcare policymakers, highlighting the long-term benefits of prophylaxis as a preventative approach for managing bleeding and improving overall well-being in patients with severe HA.
甲型血友病（HA）的治疗策略旨在控制出血发作并改善患者的生活质量。本研究调查了使用标准半衰期FVIII产品进行中等剂量预防的预防方法在降低重度HA患者出血率和提高生活质量方面的有效性。
一项为期4年的前瞻性纵向研究跟踪了2014年至2018年在台湾从反应性治疗方法转变为中等剂量预防的35例重度HA患者（无FVIII抑制剂）。该研究跟踪了年度出血率（ABR）和年度关节出血率（AjBR）以及相关成本和患者报告的生活质量指标。
与先前的治疗相比，预防显著降低了ABR和AjBR。一年后，ABR和AjBR分别下降了76.9%和72.5%，4年后进一步降至91.0%和90.8%（p < 0.001）。虽然第一年凝血因子VIII浓缩物的平均年度成本增加了41.0%，但增量成本效益比表明在4年中避免ABR带来了持续的益处。此外，患者报告在改用预防措施后生活质量指标有显著改善（p = 0.036）。
中等剂量预防有效降低了重度HA患者的出血率并改善了生活质量。尽管初期成本增加，但随着时间的推移，该干预措施变得具有成本效益。本研究为医疗保健政策制定者提供了有价值的数据，突出了预防作为管理出血和改善重度HA患者整体健康的预防方法的长期益处。
