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The use of a single von Willebrand factor‐containing, plasma‐derived FVIII product in hemophilia A immune tolerance induction: the US experience
Author(s) -
KURTH M.,
PUETZ J.,
KOUIDES P.,
SANDERS J.,
SEXAUER C.,
BERNSTEIN J.,
GRUPPO R.,
MANCOJOHNSON M.,
NEUFELD E. J.,
RODRIGUEZ N.,
WICKLUND B.,
QUON D.,
ALEDORT L.
Publication year - 2011
Publication title -
journal of thrombosis and haemostasis
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.947
H-Index - 178
eISSN - 1538-7836
pISSN - 1538-7933
DOI - 10.1111/j.1538-7836.2011.04493.x
Subject(s) - von willebrand factor , medicine , retrospective cohort study , von willebrand disease , titer , complication , immune tolerance , coagulopathy , gastroenterology , immunology , immune system , antibody , platelet
Summary. Background : Inhibitors are a serious complication for patients with severe hemophilia A. Immune tolerance induction (ITI) is the primary method for eradicating these inhibitors. The role of type of concentrate and in particular the use of von Willebrand factor‐containing, plasma‐derived factor VIII (VWF/pd‐FVIII) concentrate in primary or rescue ITI remains unclear. Objectives: To report retrospective collection of data on the use of a single VWF/pd‐FVIII concentrate in primary and rescue ITI. Methods: Retrospective chart review of hemophilia A inhibitor patients at 11 US institutions who received VWF/pd‐FVIII concentrate in primary or rescue ITI. Results: Primary ITI was carried out in eight inhibitor patients with a 75% complete and partial success. Secondary ITI was carried out in 25 inhibitor patients, with 52% attaining complete or partial success . Conclusions : This report represents the largest group of primarily pediatric, high‐titer inhibitor patients treated with a single VWF/pd‐FVIII concentrate. It adds retrospective data to the use of VWF‐containing plasma‐derived factor VIII concentrate in primary and rescue ITI, particularly in those patients with characteristics of poor response to ITI.