| Autor: |
Antonazzo IC; Research Centre on Public Health (CESP), University of Milan-Bicocca, 20900 Monza, Italy.; IRCCS Istituto Auxologico Italiano, 20145 Milano, Italy., Cortesi PA; Research Centre on Public Health (CESP), University of Milan-Bicocca, 20900 Monza, Italy.; IRCCS Istituto Auxologico Italiano, 20145 Milano, Italy., Zanon E; Hemophilia Center, University Hospital of Padua, 35128 Padua, Italy., Pasca S; Laboratory Medicine, Department of Biomedical Sciences, Padua University Hospital, 35128 Padua, Italy., Morfini M; Italian Association of Haemophilia Centres-AICE, 50100 Firenze, Italy., Santoro C; Hematology, Umberto I University Hospital, 00161 Rome, Italy., De Cristofaro R; Center for Haemorrhagic and Thrombotic Diseases, Department of Medical Sciences, Catholic University School of Medicine, 'A. Gemelli' Hospital, 00168 Rome, Italy., Di Minno G; Department of Clinical Medicine and Surgery, Regional Service Centre of Coagulation Disorders, 'Federico II' University, 80138 Naples, Italy., Cozzolino P; Research Centre on Public Health (CESP), University of Milan-Bicocca, 20900 Monza, Italy., Mantovani LG; Research Centre on Public Health (CESP), University of Milan-Bicocca, 20900 Monza, Italy.; IRCCS Istituto Auxologico Italiano, 20145 Milano, Italy. |
| Abstrakt: |
Background and Objectives: 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. Materials and Methods: 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. Results: The study enrolled 13 patients with HA. The mean annual bleeding rate decreased by -1.45 (-63.80%, p = 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. Conclusions: 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. |
