Federici Augusto B, Santoro Rita Carlotta, Santoro Cristina, Pieri Lisa, Santi Roberto Mario, Barillari Giovanni, Borchiellini Alessandra, Tosetto Alberto, Zanon Ezio, De Cristofaro Raimondo, Mairal Esther, Mir Roser
Department of Haematology and Haemotherapy, Luigi Sacco Hospital, Milano, Italy.
Haemophilia Centre, Haemostasis and Thrombosis Unit, Department of Haemato-Oncology, Azienda Ospedaliero-Universitaria Dulbecco, Catanzaro, Italy.
Clin Appl Thromb Hemost. 2024 Jan-Dec;30:10760296241264541. doi: 10.1177/10760296241264541. Epub 2024 Jul 21.
Plasma-derived von Willebrand factor-containing factor VIII concentrates (pd-VWF/FVIII-C) are the mainstay of treatment in von Willebrand disease (VWD). Real-world data on efficacy and safety of these pd-VWF/FVIII-C are required. To retrospectively evaluate the efficacy and safety of pd-VWF/FVIII-C (Fanhdi® and Alphanate®, Grifols) in clinical practice in Italy. A multicentric, observational, retrospective study at 10 Italian centers was conducted. Eligible patients diagnosed with inherited VWD (ISTH criteria) were treated with either Fanhdi® or Alphanate® for bleeding episodes, prevention of surgical bleeding and secondary long-term prophylaxis (SLTP) according to clinical practice with medical records collected from January 2007 to December 2019. Efficacy/safety of pd-VWF/FVIII-C was assessed according to FDA-agreed objective criteria following regulatory procedures. Fifty-seven patients (M/F: 21/36) were enrolled in the study with the following VWD types: VWD1 (n = 29, 52%), VWD2A (n = 10, 18%), VWD2B (n = 7, 12%), VWD2M (n = 2, 4%), VWD2N (n = 1, 2%), VWD2 unclassified (n = 1, 2%), and VWD3 (n = 7, 12%). These pd-VWF/FVIII-C were used to manage 58 bleeding episodes (n = 24 patients), 100 surgeries (n = 47 patients), and 7 SLTP (n = 6 patients). Global clinical efficacy with these pd-VWF/FVIII-C was reported to be excellent/good in 85% of bleeding episodes, 98% of surgeries, and 100% of SLTP. As far as safety, no adverse-drug-related episodes, immunogenic or thrombotic events were reported. This study confirmed that Fanhdi® and Alphanate® were effective and safe in the management of bleeding episodes, the prevention of bleeding during surgeries and for SLTP in Italian patients with inherited VWD.
血浆源性含血管性血友病因子的凝血因子VIII浓缩物（pd-VWF/FVIII-C）是血管性血友病（VWD）治疗的主要手段。需要关于这些pd-VWF/FVIII-C疗效和安全性的真实世界数据。为了回顾性评估pd-VWF/FVIII-C（Fanhdi®和Alphanate®，基立福公司）在意大利临床实践中的疗效和安全性。在意大利的10个中心进行了一项多中心、观察性、回顾性研究。符合遗传性VWD诊断标准（国际血栓与止血学会标准）的合格患者，根据临床实践，针对出血发作、预防手术出血和二级长期预防（SLTP），使用Fanhdi®或Alphanate®进行治疗，并收集了2007年1月至2019年12月的医疗记录。按照美国食品药品监督管理局认可的客观标准，遵循监管程序评估pd-VWF/FVIII-C的疗效/安全性。57名患者（男/女：21/36）纳入研究，VWD类型如下：VWD1（n = 29，52%）、VWD2A（n = 10，18%）、VWD2B（n = 7，12%）、VWD2M（n = 2，4%）、VWD2N（n = 1，2%）、VWD2未分类（n = 1，2%）和VWD3（n = 7，12%）。这些pd-VWF/FVIII-C用于处理58次出血发作（n = 24例患者）、100次手术（n = 47例患者）和7次SLTP（n = 6例患者）。据报告，这些pd-VWF/FVIII-C在85%的出血发作、98%的手术和100%的SLTP中总体临床疗效为优/良。在安全性方面，未报告与药物相关的不良事件、免疫原性或血栓形成事件。这项研究证实，Fanhdi®和Alphanate®在处理意大利遗传性VWD患者的出血发作、预防手术期间出血以及SLTP方面有效且安全。
