PB1245 - Efficacy, safety and dosing of pasteurized plasma-derived von Willebrand factor/Factor VIII concentrate for the treatment of von Willebrand Disease: a systematic review

Tuesday, June 27, 2023

18:30 – 19:30 ET

Room: Exhibition

Presenting Author(s)

Carmen Escuriola-Ettingshausen, MD

Physician
Haemophilia Centre Rhine-Main, Mörfelden-Walldorf, Germany
Mörfelden-Walldorf, Hessen, Germany

Co-Author(s)

Riitta Lassila, MD, PhD (she/her/hers)

Professor of Coagulation Medicine
Helsinki University Hospital, Coagulation Disorders Unit, Department of Haematology, Comprehensive Cancer Centre, and Research Program Unit in System Oncology, University of Helsinki, Helsinki, Finland
Helsinki, Uusimaa, Finland
GE

Gines Escolar

MD PhD Senior Consultant
Hemostasis and Erythropathology Laboratory, Hematopathology, Department of Pathology, Centre de Diagnòstic Biomèdic (CDB), Hospital Clínic de Barcelona, Institut d'Investigacions Biomèdiques August Pi i Sunyer (IDIBAPS), Universitat de Barcelona, Barcelona, Spain.
Barcelona, Catalonia, Spain
Christoph Male, MD, MSc

Head of the Pediatric Hemostasis & Thrombosis Unit
Department of Paediatrics, Medical University of Vienna, Vienna, Austria.
Vienna, Wien, Austria
KS

Kathrin Schirner

Global Safety Lead
CSL Behring Innovation GmbH, Marburg, Germany
Marburg, Hessen, Germany
LH

Lisa Heyder

Senior Clinical Safety Scientist
CSL Behring Innovation GmbH, Marburg, Germany
Marburg, Hessen, Germany
EB

Erik Berntorp

Professor at Clinical Coagulation
Clinical Coagulation Research, Department of Translational Medicine, Lund University, Malmö, Sweden
Malmo, Skane Lan, Sweden

Background: Von Willebrand disease (VWD) is an inherited bleeding disorder caused by deficient or dysfunctional von Willebrand factor (VWF). VWF replacement therapy is indicated in VWD management.

Aims: To evaluate available evidence on efficacy, safety, dosing, and consumption of pasteurized plasma-derived human coagulation factor VIII/human VWF (pdVWF/FVIII) when used for on-demand treatment, long-term and surgical prophylaxis in patients with inherited VWD.

Methods: A systematic search was performed according to predefined selection criteria in MEDLINE® and Cochrane Library databases (up to June 30, 2022) to identify studies reporting use of pasteurized pdVWF/FVIII in VWD. Pharmacovigilance data were also retrieved across the same period.

Results: Fifteen individual studies were identified, including 12 observational and 3 interventional studies. Treatment protocols, efficacy and safety assessments varied across the studies, which hindered direct comparisons. Nevertheless, hemostatic efficacy of pasteurized pdVWF/FVIII was rated excellent/good for on-demand treatment in 95–98% of bleeds, in 93–100% of clinical responses to long-term prophylactic treatment, and in 94–100% of surgical procedures. Analysis of pharmacovigilance safety reports showed that pasteurized pdVWF/FVIII was associated with low incidence rates of adverse events.

Conclusion(s): This systematic literature review summarizes evidence published over four decades of clinical use of pasteurized pdVWF/FVIII, demonstrating the long-term efficacy and safety of pasteurized pdVWF/FVIII when used for on-demand treatment, long-term prophylaxis, and surgical prophylaxis in patients of all ages with all types of inherited VWD. Analysis of pharmacovigilance data collected over 40 years of surveillance reaffirms the positive safety profile of pasteurized pdVWF/FVIII.