Severe hemophilia A is an X-linked bleeding disorder. Immune tolerance induction (ITI) is the best strategy of treatment when patients develop inhibitors. The objective is to illustrate the benefit of a high-purity human factor VIII/von Willebrand factor (VWF) concentrate (Octanate)
in the management of ITI. We also wanted to raise the potential interest of laboratory assays such as thrombin-generation test (TGT) and epitope mapping. Two patients were treated during ITI, first with a recombinant FVIII and then with plasma-derived factor VIII without success, and, finally,
with Octanate. Bypassing agents were used based on the results of TGT. Epitope mapping was performed during ITI therapy. These observations suggest the potential contribution of Octanate in the management of ITI in difficult cases. The use of bypassing agents can be necessary in prophylaxis
or to treat bleedings, and may be guided by TGT results. Epitope mapping is used to describe the inhibitor. This article shows a decrease of the inhibitor directed against the C2 domain after initiation of Octanate. A high-purity human factor VIII/von Willebrand factor concentrate (Octanate)
may be a valuable therapeutical option for ITI therapy. TGT and epitope mapping could be of help in the management of ITI.
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high-purity human factor VIII/von Willebrand factor concentrate;
immune tolerance induction;
Document Type: Research Article
Hematology and Oncology Pediatric Unit, CHU Saint Etienne
Hemostasis Laboratory, CHU, Saint Etienne, France
Research Unit on Hemophilia, UFR Laennec, Clinical Hemostasis Unit, Hôpital Edouard Herriot, Lyon
Hematology Laboratory, CHU Caremeau, Nimes
HZRM – Hemophilia Centre Rhein Main, Frankfurt-Mörfelden, Germany
December 1, 2015