title: Pharmacokinetics, efficacy, and safety of a plasma-derived VWF/FVIII concentrate (VONCENTO) for on-demand and prophylactic treatment in patients with von Willebrand disease (SWIFT-VWD study).
abstract: VONCENTO (CSL Behring Gmbh, Marburg, Germany) is a plasma-derived, high concentration, lower volume [relative to HAEMATE P (CSL Behring)], high-purity von Willebrand factor (VWF)/factor VIII (FVIII) concentrate with a VWF/FVIII ratio similar to HAEMATE P. This open-label, multicentre study investigated the pharmacokinetic, haemostatic efficacy, and safety profiles of VONCENTO in study participants at least 12 years of age with von Willebrand disease (VWD) who required treatment of nonsurgical bleeding (NSB) events or underwent surgery or prophylaxis. The first 12-month on-demand treatment period comprised a pharmacokinetic investigation and an efficacy analysis. After 12 months, qualifying study participants were switched to prophylactic therapy and included in a further 12-month efficacy analysis. In total, 21 study participants (including three adolescents, and 13 study participants with VWD type 3) received VONCENTO as on-demand treatment for 12 months. 'Excellent'/'good' haemostatic efficacy was achieved in 98.3% of the 407 NSB events assessed by investigators. Following the switch to prophylactic treatment, the total number of NSBs in eight patients markedly decreased from 304 to 10 (with haemostatic efficacy judged to be 'excellent' for all). The annualised bleeding rate also significantly decreased from a median of 26.5 events to one event. Safety assessments showed no inhibitory antibodies to either FVIII or VWF, no transmission of infectious agents, no thromboembolic events and no treatment-related serious adverse events. VONCENTO was shown to be well tolerated and provided excellent haemostatic efficacy in the treatment of bleeds or during prophylaxis in study participants with VWD, including also those with type 3, the severest form of VWD.
authors.affiliation: aSpecialized Hospital for Active Treatment (SHAT), Sofia, Bulgaria bGOUVPO Altaysky State Medical University of Roszdrav, Barnaul, Russian Federation cIndependent Public Clinical Hospital No. 1, Wroclaw, Poland dInstitute of Pathology and Transfusion Medicine AMN, Lviv, Ukraine eInstitute of Hematology, Rio de Janeiro, Brazil fDepartment of Pediatrics, Hematology and Oncology of Warsaw Medical University, Warsaw, Poland gClinical Research and Development, CSL Behring, Marburg, Germany.
authors.forename: Toshko J
authors.initials: TJ
authors.lastname: Lissitchkov
authors.forename: Evgeny
authors.initials: E
authors.lastname: Buevich
authors.forename: Kazimierz
authors.initials: K
authors.lastname: Kuliczkowski
authors.forename: Oleksandra
authors.initials: O
authors.lastname: Stasyshyn
authors.forename: Monica Hermida
authors.initials: MH
authors.lastname: Cerqueira
authors.forename: Anna
authors.initials: A
authors.lastname: Klukowska
authors.forename: Christine
authors.initials: C
authors.lastname: Joch
authors.forename: Wilfried
authors.initials: W
authors.lastname: Seifert
mesh_terms: D000328:Adult; D005169:Factor VIII / Q000493:pharmacokinetics / Q000627:therapeutic use*; D005260:Female; D006487:Hemostasis; D006490:Hemostatics / Q000008:administration & dosage / Q000627:therapeutic use*; D006801:Humans; D008297:Male; D014842:von Willebrand Diseases / Q000188:drug therapy*; D014841:von Willebrand Factor / Q000493:pharmacokinetics / Q000627:therapeutic use*
chemical_list: D006490:Hemostatics; D014841:von Willebrand Factor; D005169:Factor VIII