title: Clinical efficacy of a novel VWF-containing FVIII concentrate, Wilate(®), in the prophylaxis and treatment of bleeding episodes in previously treated haemophilia A patients.
abstract: The new-generation coagulation factor VIII (FVIII)/von Willebrand factor (VWF) concentrate, Wilate(®), is effective in the therapy of von Willebrand disease for which it was originally developed. The ratio of haemostatic components (FVIII to VWF) of this high-purity plasma-derived concentrate is 1:1, with VWF naturally stabilising FVIII. Bleeding episode control in patients with haemophilia A (HA) is generally managed by replacement therapy with recombinant or plasma-derived FVIII. When complexed with VWF as in the physiological situation, the product may also show a lower incidence of inhibitor development in patients with HA. The clinical efficacy, safety and tolerability of Wilate(®) were therefore evaluated in 81 previously treated individual patients with severe HA. Data from 5 good clinical practice (GCP) prospective clinical studies were pooled and assessed with regards to the prevention and treatment of bleeding and during surgical procedures. Haemostatic efficacy was excellent or good in 96.7% of 1495 rated treatments of bleeding episodes. The average dose per treatment was approximately 30 IU FVIII/kg. In surgical settings, the global haemostatic efficacy of Wilate(®) was generally rated excellent or good for use during and after operations with a mean total dose of FVIII of 848.6 IU/kg±578.5 IU/kg administered peri-operatively. Overall, tolerability of Wilate(®) was rated very good or good by patients and physicians. Rarely occurring adverse events were mild in intensity and no anti-FVIII inhibitors were detected. Wilate(®) also displayed a good viral safety profile with no seroconversions occurring in response to treatment. These data show that Wilate(®) is an efficacious treatment option in the management of patients with severe HA.
authors.affiliation: Department of Pediatrics, Hematology and Oncology, Warsaw Medical University, Warszawa, Poland.
authors.forename: Anna
authors.initials: A
authors.lastname: Klukowska
authors.forename: Jerzy
authors.initials: J
authors.lastname: Windyga
authors.forename: Angelika
authors.initials: A
authors.lastname: Batorova
mesh_terms: D000293:Adolescent; D000328:Adult; D000368:Aged; D002648:Child; D002986:Clinical Trials as Topic; D005169:Factor VIII / Q000009:adverse effects / Q000493:pharmacokinetics / Q000627:therapeutic use*; D006467:Hemophilia A / Q000188:drug therapy / Q000601:surgery*; D006470:Hemorrhage / Q000188:drug therapy* / Q000517:prevention & control; D006490:Hemostatics / Q000009:adverse effects / Q000493:pharmacokinetics / Q000627:therapeutic use*; D006801:Humans; D008875:Middle Aged; D055815:Young Adult; D014841:von Willebrand Factor / Q000009:adverse effects / Q000493:pharmacokinetics / Q000627:therapeutic use*
chemical_list: D006490:Hemostatics; D014841:von Willebrand Factor; D005169:Factor VIII