The role of previously untreated patient studies in understanding the development of FVIII inhibitors

Haemophilia : the Official Journal of the World Federation of Hemophilia
M D CarcaoB Ewenstein

Abstract

Development of inhibitors against factor VIII (FVIII), the major complication of haemophilia A treatment today, is influenced by multiple factors. Genetic (F8 mutation, family history, ethnicity, polymorphisms in immune modulating genes) and non-genetic (intensive exposure to FVIII, presence of pro-inflammatory signals as might occur with large bleeds, infections, surgery, or other immune stimulants [e.g. vaccines]) risk factors as well as their complex inter-relationships contribute to the inhibitor risk profile of haemophilia patients, particularly in the previously untreated patient (PUP) population. Studies in PUPs have been fundamental to furthering the understanding of FVIII inhibitor development, as well as discovering previously unappreciated risk factors. The multi-factorial nature of inhibitor development makes it difficult to ascertain the contribution of FVIII products in inhibitor development through individual PUP studies. Sufficiently powered studies of large cohorts may overcome these limitations but interpretations should be conducted cautiously. Proper design and implementation of PUP safety studies will become even more important with the introduction of new molecules, such as extended half-life or human cell...Continue Reading

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Aug 17, 2016·Thrombosis and Haemostasis·Jenny GoudemandSébastien Lacroix-Desmazes
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