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Human von Willebrand factor/factor VIII concentrates in the management of pediatric patients with von Willebrand disease/hemophilia A

Authors Castaman G, Linari S

Received 22 December 2015

Accepted for publication 2 May 2016

Published 30 June 2016 Volume 2016:12 Pages 1029—1037

DOI https://doi.org/10.2147/TCRM.S87543

Checked for plagiarism Yes

Review by Single-blind

Peer reviewers approved by Dr Colin Mak

Peer reviewer comments 2

Editor who approved publication: Professor Garry Walsh

Giancarlo Castaman, Silvia Linari

Department of Oncology, Center for Bleeding Disorders, Careggi University Hospital, Florence, Italy

Abstract: Several plasma-derived intermediate and high-purity concentrates containing von Willebrand factor (VWF) and factor VIII (FVIII) are currently available. The main role of these products in the management of pediatric the population is represented by the replacement therapy in patients with severe or intermediate forms of von Willebrand disease, in whom other treatments are ineffective or contraindicated. Another important role of VWF/FVIII concentrates in children may be their use in immune tolerance induction (ITI) protocols. ITI is particularly recommended for hemophilia A children who have developed an inhibitor against FVIII, currently the most serious complication of substitutive treatment in hemophilia. Although recombinant concentrates may represent the preferred option in children with hemophilia A, VWF/FVIII concentrates may offer an advantage in rescuing patients who failed previous ITI.

Keywords: von Willebrand factor, factor VIII, plasma-derived concentrates, children, von Willebrand disease, hemophilia A

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