The 1990s also saw the introduction of immune tolerance INCB024360 cost induction therapy which was first discovered
in Bonn, Germany. The scientific community was understandably sceptical at first about the rather unconventional approach of administering antigen as a means of antibody eradication, but this treatment strategy has since demonstrated a high level of efficacy and is an important step forward in the treatment of haemophilia. Rounding out the ‘golden era’ was the introduction of medications (highly-active antiretroviral therapy for HIV; interferon and ribavirin for hepatitis) which brought about a dramatic change in the outcome of haemophiliac ABT-199 clinical trial patients burdened with these diseases. As a direct result of these new developments, life expectancy for patients with haemophilia in many developed countries has approached that of the general population [1–3]. Indeed, within the
context of the monogenic diseases, patients with haemophilia are much ‘better off’ in terms of life expectancy than patients with cystic fibrosis, thalassemia major or muscular dystrophy. On the basis of the progress witnessed over the past 40 years, it is not surprising that the motto of haemophilia therapy in the third millennium is ‘building on Cell press strength’. However, as considerable scope remains for further advancements in the management of this rare but complex condition, it is important to examine the main issues surrounding current treatment which can be summed up as follows: Greater and
wider factor availability Generally speaking, there are two types of products available for the treatment of haemophilia: plasma-derived and recombinant factor concentrates. Both are efficacious and both are safe as determined through ongoing monitoring via the European Haemophilia Safety Surveillance (EUHASS) system. The need for continued surveillance to prevent issues such as those encountered by the haemophilia community in the past cannot be over-emphasized and there are plans to extend this important European initiative to other countries in future. At present, however, at least two-thirds of the world’s population with haemophilia remain without access to clotting factor therapy. At the heart of the issue is cost.