Choví-Trull Maria, Megías-Vericat Juan Eduardo, Bonanad Boix Santiago, Haya Guaita Saturnino, Cid Haro Ana Rosa, Aguilar Rodriguez Marta, Poveda Andrés Jose Luis
Servicio de Farmacia, Hospital Universitari i Politècnic La Fe, Valencia, Spain.
Servicio de Farmacia, Hospital Universitari i Politècnic La Fe, Valencia, Spain.
Farm Hosp. 2025 Jul-Aug;49(4):T205-T211. doi: 10.1016/j.farma.2024.12.007. Epub 2025 Feb 14.
To analyse the differences in pharmacokinetic and clinical parameters (bleeding rates and joint health) before and after switching from standard half-life factor VIII (FVIII) to extended half-life pegylated FVIII in patients with severe/moderate haemophilia A on prophylaxis, 1 year before and after the switch in real-life.
This is a single-centre, comparative, observational, sequential, retrospective, and multidisciplinary study. Population pharmacokinetic models from the WAPPS-Hemo® application were used to calculate pharmacokinetic parameters and individualise prophylaxis. The annual rate of total and joint bleeds, joint health (Haemophilia Joint Health Score), plasma half-life and area under the curve ratios, FVIII consumption, administration frequency, and cost were analysed.
Thirty-eight adult patients with haemophilia A who switched from standard half-life FVIII to extended half-life pegylated FVIII were analysed. Significant improvements (P < .05) were observed in all pharmacokinetic parameters, with plasma half-life and area under the curve improvement ratios of 1.5 and 1.9, respectively, as well as reductions in annual total and joint bleeding rates. A higher number of patients with zero total (16.0 vs. 29.0) and joint bleeds (23.0 vs. 33.0) was also observed. The median reductions in administration frequency and dose/kg/week were 30.0% and 19.7%, respectively, avoiding 44.3 infusions/patient/year, resulting in savings of 20,843 €/patient/year. Furthermore, joint health improved (23.0 vs. 21.0; P = .017), and target joints resolved after the switch.
The pharmacokinetically guided switch from standard half-life FVIII to pegylated FVIII demonstrated significant clinical benefits with reduced bleeding rates and improvements in joint health. Additionally, improvements in pharmacokinetic parameters were observed, allowing for reduced treatment burden by decreasing administration frequency, as well as lower consumption and costs.
分析重度/中度甲型血友病患者在预防治疗中从标准半衰期因子VIII（FVIII）转换为延长半衰期聚乙二醇化FVIII前后1年的药代动力学和临床参数（出血率和关节健康状况）差异。
这是一项单中心、比较性、观察性、序贯性、回顾性多学科研究。使用WAPPS-Hemo®应用程序中的群体药代动力学模型计算药代动力学参数并进行个体化预防治疗。分析了总出血和关节出血的年发生率、关节健康状况（血友病关节健康评分）、血浆半衰期和曲线下面积比值、FVIII消耗量、给药频率和成本。
对38例从标准半衰期FVIII转换为延长半衰期聚乙二醇化FVIII的成年甲型血友病患者进行了分析。所有药代动力学参数均有显著改善（P<0.05），血浆半衰期和曲线下面积改善率分别为1.5和1.9，同时年总出血率和关节出血率降低。总出血为零（16.0对29.0）和关节出血为零（23.0对33.0）的患者数量也更多。给药频率和剂量/千克/周的中位数分别降低了30.0%和19.7%，每位患者每年避免44.3次输注，每位患者每年节省20,843欧元。此外，关节健康状况有所改善（23.0对21.0；P=0.017），转换后目标关节问题得到解决。
从标准半衰期FVIII到聚乙二醇化FVIII的药代动力学指导转换显示出显著的临床益处，出血率降低，关节健康状况改善。此外，药代动力学参数也有所改善，通过降低给药频率减轻了治疗负担，同时消耗量和成本也更低。
