In the last two decades, great advances in our understanding of the genetic and molecular bases of haemophilia, along with the results of extensive clinical research, have led to dramatic improvements in the management of people with this inherited bleeding disorder. The period from the 1990s onward has been characterised by an increase in the availability and quality of recombinant coagulation factors and the broad implementation of prophylactic treatment regimens1. In parallel, progressive improvement of virus-inactivation methods in plasma products, as well as of methods used to screen viruses in blood donations and plasma pools (i.e., polymerase chain reaction testing) led to an impressive increase in the safety of plasma-derived factor concentrates, as clearly documented by the fact that no blood-borne transmission of hepatitis viruses or human immunodeficiency virus has occurred in the last 20 years2. As a consequence, the quality of life of haemophiliacs has dramatically improved and their life expectancy has progressively approached that of their non-haemophilic peers3. In this context, the most challenging complication of current therapy has become the development of inhibitory alloantibodies, which renders replacement therapies ineffective, limit the access of patients to a safe and effective standard of care and predispose them to an unacceptably high risk of morbidity and mortality4.
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Pier Mannuccio Mannucci (2010) studied this question.
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