Takeda International Trading Co. Ltd, Beijing, China.
Sun Yat-Sen University, No. 132 Waihuan East Road, Guangzhou, 510006, China.
Adv Ther. 2022 Aug;39(8):3777-3788. doi: 10.1007/s12325-022-02220-3. Epub 2022 Jun 30.
Multiple studies have demonstrated the effectiveness of pharmacokinetic (PK)-guided individualized prophylaxis with human coagulation factor VIII (FVIII) compared with standard prophylaxis, but no studies have evaluated the economics of PK-guided prophylaxis in China. Hence, we conducted this study to assess the cost-effectiveness of PK-guided prophylaxis with recombinant FVIII (rFVIII) versus standard prophylaxis in Chinese adult patients with severe hemophilia A.
A discrete event simulation model was developed to simulate 10,000 patients with hemophilia A who received rFVIII treatment over a 1-year time horizon. The standard prophylaxis rFVIII dose was 30 IU/kg by intravenous injection. The PK-guided prophylaxis dosage was adjusted for each patient to maintain FVIII trough level at 1-5 IU/dL. Dosing interval for both approaches was kept fixed at 48 h. The health outcomes included annual joint bleed rate (AJBR) and quality-adjusted life years (QALYs). The model considered the costs of drug. Incremental cost-effectiveness ratio (ICER) was estimated and scenario analysis was performed.
A total of 94.3% of patients receiving PK-guided individualized prophylaxis achieved the goal of maintaining the trough concentration at 1-5 IU/dL compared with 62.7% on standard prophylaxis. AJBR and QALYs gained in PK-guided and standard prophylaxis were 1.527 vs 1.601, and 0.8384 vs 0.8383, respectively. Costs of drug prophylaxis and costs of treatment for bleeding events in PK-guided prophylaxis (148,641.47 USD; 4546.43 USD) were lower than those in standard prophylaxis (159,620.93 USD; 4753.39 USD). An average saving of USD 11,186.47 was obtained by the PK-guided approach. The prophylaxis treatment scenarios were the most influential factors.
PK-guided individualized prophylaxis appeared to be a dominant treatment compared with standard prophylaxis, with slightly higher QALYs but lower total costs.
多项研究表明，与标准预防相比，基于药代动力学（PK）的个体化预防用人类凝血因子 VIII（FVIII）更有效，但尚无研究评估 PK 指导预防在中国的经济学价值。因此，我们开展此项研究，旨在评估 PK 指导预防用重组 FVIII（rFVIII）与中国重度 A 型血友病成人患者的标准预防相比的成本效果。
建立离散事件仿真模型，模拟 10000 例接受 rFVIII 治疗的 A 型血友病患者，时间范围为 1 年。标准预防 rFVIII 剂量为 30IU/kg 静脉注射。PK 指导预防剂量根据每位患者的情况进行调整，以维持 FVIII 谷浓度在 1-5IU/dL。两种方法的给药间隔均固定为 48h。健康结果包括年关节出血率（AJBR）和质量调整生命年（QALYs）。模型考虑了药物成本。估计增量成本效果比（ICER）并进行情景分析。
与标准预防相比，接受 PK 指导个体化预防的患者中，有 94.3%的患者达到维持谷浓度在 1-5IU/dL 的目标，而标准预防的这一比例为 62.7%。PK 指导预防和标准预防的 AJBR 和 QALYs 获益分别为 1.527 和 1.601，0.8384 和 0.8383。PK 指导预防的药物预防成本和出血事件治疗成本（148641.47 美元；4546.43 美元）低于标准预防（159620.93 美元；4753.39 美元）。PK 指导方法可节省平均 11186.47 美元。预防治疗方案是最具影响力的因素。
与标准预防相比，PK 指导个体化预防似乎是一种优势治疗方法，具有略高的 QALYs，但总费用较低。
