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“Summary.  Current treatment for haemophilia provides excellent efficacy and safety albeit with a number of unresolved issues. The development of inhibitors following treatment with factor VIII (FVIII) is the most challenging complication of haemophilia and bears the see more highest economic burden for a chronic disease. Moreover, prophylactic therapy for haemophilia requires repeated infusions of FVIII, frequently as often as two or three times weekly, which can impact greatly on patients’ daily lives. As considerable scope remains for further advancements in the management of this condition, the primary

focus of this paper relates to issues regarding current treatment and strategies in place to resolve the various issues. For countries approaching access to replacement therapy, it is important to know whether or not plasma-derived and recombinant products are associated

with different risks of inhibitor development in previously untreated patients with severe haemophilia. The ongoing international SIPPET study is expected to provide an answer to this clinical dilemma. Methods under investigation to prolong the half-life of factor concentrates offer new hope to reduce the burden of prophylaxis for patients with haemophilia, with early results suggesting greater benefits with FIX. Current treatment for haemophilia already provides excellent efficacy and safety, and this Y 27632 must be borne in mind in the development of new products. It is imperative that there will be no risk to the patient

and genuine improvement over the range of available treatments. The first major wave of change in haemophilia treatment took place in the 1970s with the introduction of lyophilized coagulation factors. This permitted the activity of comprehensive treatment centres and enabled home treatment programmes. This decade also saw the initiation in Sweden of prophylaxis regimes as well as the discovery in Italy of desmopressin (DDAVP) for mild haemophilia A and von Willebrand disease (VWD). The 1980s were characterized by many shadows but also some lights. These were the years of AIDS and hepatitis, but the ensuing dramas encountered in the haemophiliac population fostered research MCE that led to the rapid cloning of factor VIII (FVIII) and factor IX (FIX) genes which were the basis for production of FVIII and FIX by recombinant technology. Progress in viral inactivation methods also made plasma factors pathogen-free and much safer and, indeed, since the late 1980s/early 1990s no pathogens have been transmitted by factor concentrates. The 1990s heralded a new ‘golden era’ in haemophilia treatment which continues into the third millennium. It was during this decade that recombinant FVIII (rFVIII) and recombinant FIX (rFIX) became widely available.