Research Centre on Public Health (CESP), University of Milan-Bicocca, 20900 Monza, Italy.
IRCCS Istituto Auxologico Italiano, 20145 Milano, Italy.
Medicina (Kaunas). 2023 Dec 24;60(1):34. doi: 10.3390/medicina60010034.
This study aimed to assess the effectiveness and costs associated with pharmacokinetics-driven (PK) prophylaxis based on the myPKFiT device in patients affected by hemophilia A (HA) in Italy. An observational retrospective study was conducted in three Italian hemophilia centers. All patients with moderate or severe HA, aged ≥ 18 years, capable of having PK estimated using the myPKFiT device, and who had had a clinical visit between 1 November 2019 and 31 March 2022 were included. Differences in clinical, treatment, health resources, and cost data were assessed comparing post-PK prophylaxis with pre-PK. The incremental cost-effectiveness ratio (ICER) was estimated as cost (EUR) per bleed avoided. The study enrolled 13 patients with HA. The mean annual bleeding rate decreased by -1.45 (-63.80%, = 0.0055) after the use of myPKFiT. Overall, the consumption of FVIII IU increased by 1.73% during follow-up compared to the period prior the use of the myPKFiT. Prophylaxis based on the myPKFiT resulted in an ICER of EUR 5099.89 per bleed avoided. The results of our study support the idea that the use of PK data in clinical practice can be associated with an improvement in the management of patients, as well as clinical outcomes, with a reasonable increase in costs.
本研究旨在评估基于 myPKFiT 设备的药代动力学指导（PK）预防在意大利血友病 A（HA）患者中的有效性和成本效益。 在意大利的三个血友病中心进行了一项观察性回顾性研究。所有年龄≥18 岁、能够使用 myPKFiT 设备估计 PK、在 2019 年 11 月 1 日至 2022 年 3 月 31 日期间有临床就诊的中重度 HA 患者均被纳入。通过比较 PK 预防前后的临床、治疗、卫生资源和成本数据，评估了差异。增量成本效益比（ICER）是根据避免出血的成本（欧元）来估计的。 该研究纳入了 13 名 HA 患者。使用 myPKFiT 后，年出血率平均下降了-1.45（-63.80%， = 0.0055）。总体而言，与使用 myPKFiT 之前的时期相比，在随访期间 FVIII IU 的消耗增加了 1.73%。基于 myPKFiT 的预防措施的 ICER 为每避免一次出血的成本为 5099.89 欧元。 我们的研究结果支持这样一种观点，即在临床实践中使用 PK 数据可能与改善患者管理以及临床结果相关，同时成本也会合理增加。
