Global Forum on Treatment Safety and Availability

ČSH

The World Federation of Hemophilia (WFH) invites experts every other year - in odd-numbered years - to present the latest developments to the entire community. The topics always concern treatment products, each time from a slightly different angle. This time, their safety and availability were on the agenda. It was confirmed that the high safety standards of recent decades leave no doubt that all products manufactured today are, in terms of current knowledge, completely safe and free from the threat of infection by known viruses. The terrible era of HIV and HCV infections is definitively over.

Much discussion therefore focused especially on plasma-derived products, which still account for a high percentage of global production. In our country too, they cover 60% of consumption, with only 40% accounted for by recombinant products. From this perspective, it is interesting that the Czech Republic ranks third in the world for the production of donated blood plasma per capita. The United States collects the most (30 liters of plasma per 1,000 inhabitants), and the Czech Republic is third with 30 liters. Only Austria is ahead of us. In America, plasma donation is often a source of income, hence the high number. However, paid donation can potentially be the greatest threat to safety.

Today, global concerns about virus transmission through plasma no longer prevail; the greatest worry is the development of inhibitors. This is clearly the biggest risk of frequent infusions of blood derivatives, whether plasma-derived or recombinant.

Only about 25% of the world's population has access to treatment with coagulation products. Higher costs stand in the way of wider distribution, but thanks to the efforts of the WFH over recent decades, this situation is slowly improving. But really only very, very slowly.

The main focus today is on products with extended half-life and gene therapy. With both, it is like a seesaw - sometimes they are discussed with great hope, other times expectations need to be tempered. It is a fact, however, that products with extended half-life are already in use somewhere in the world, especially those with Factor IX, which is incomparably simpler and more manageable than Factor VIII. We have already written about this matter several times on our website, where a number of articles can be found. Opinions vary greatly, particularly regarding what consequences the modified molecules carrying the active factor may bring in the future.

The biggest "hit" of the present, however, is gene therapy. It is discussed at every international expert meeting, but again with fluctuating results. Once more, it is simpler to eliminate the Factor IX deficit, i.e., the cause of hemophilia B. At the moment, it appears that effective gene therapy cannot be expected within the next ten years.

A clear positive remains that research and development in the field of hemophilia treatment continues unabated, with work proceeding on several fronts in various countries in Europe and the USA. It is not without reason that no hereditary disease has seen such progress and such rapid research development in the last quarter century as hemophilia.

The extensive development of treatment is now being replaced by intensive development, primarily in the developed part of the world - that is, the considerably smaller part compared to the rest. The materials from the 9th WFH Global Forum can reassure us with the finding that the Czech Republic is doing very well in all respects and belongs among the most successful countries in the world in hemophilia treatment.