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J. Clin. Med. 2017, 6(4), 46; doi:10.3390/jcm6040046

Treatment and Prevention of Bleeds in Haemophilia Patients with Inhibitors to Factor VIII/IX

1
Haemophilia and Thrombosis Centre, San Giovanni Bosco Hospital, Napoli 80144, Italy
2
Department of Transfusion Medicine and Haematology, Carlo Poma Hospital, Mantova 46100, Italy
3
Regional Reference Centre for Coagulation Disorders, Federico II University Hospital, Napoli 80131, Italy
*
Author to whom correspondence should be addressed.
Academic Editors: Massimo Morfini and Giovanni Di Minno
Received: 28 January 2017 / Revised: 6 April 2017 / Accepted: 10 April 2017 / Published: 17 April 2017
(This article belongs to the Special Issue Outstanding Advances in Hemophilia Therapies)
View Full-Text   |   Download PDF [833 KB, uploaded 17 April 2017]   |  

Abstract

The development of alloantibodies neutralising therapeutically administered factor (F) VIII/IX (inhibitors) is currently the most severe complication of the treatment of haemophilia. When persistent and at a high titre, inhibitors preclude the standard replacement treatment with FVIII/FIX concentrates, making patients’ management challenging. Indeed, the efficacy of bypassing agents, i.e., activated prothrombin complex concentrates (aPCC) and recombinant activated factor VII (rFVIIa), needed to overcome the haemostatic interference of the inhibitor, is not comparable to that of factor concentrates. In addition, the therapeutical response is unpredictable, with a relevant inter-individual and even intra-individual variability, and no laboratory assay is validated to monitor the efficacy and safety of the treatment. As a result, inhibitor patients have a worse joint status and quality of life compared to inhibitor-free subjects and the eradication of the inhibitor by immune tolerance induction is the preeminent therapeutic goal, particularly in children. However, over the last decades, treatment with bypassing agents has been optimised, allowing home treatment and the individualisation of regimens aimed at improving clinical outcomes. In this respect, a growing body of evidence supports the efficacy of prophylaxis with both bypassing agents in reducing bleeding rates and improving the quality of life, although the impact on long-term outcomes (in particular on preventing/reducing joint deterioration) is still unknown. This review offers an update on the current knowledge and practice of the use of bypassing agents in haemophiliacs with inhibitors, as well as on debated issues and unmet needs in this challenging setting. View Full-Text
Keywords: bleeding; haemophilia; inhibitors; bypassing therapy; prophylaxis bleeding; haemophilia; inhibitors; bypassing therapy; prophylaxis
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This is an open access article distributed under the Creative Commons Attribution License which permits unrestricted use, distribution, and reproduction in any medium, provided the original work is properly cited. (CC BY 4.0).

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MDPI and ACS Style

Rocino, A.; Franchini, M.; Coppola, A. Treatment and Prevention of Bleeds in Haemophilia Patients with Inhibitors to Factor VIII/IX. J. Clin. Med. 2017, 6, 46.

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