Interesting News from the EHC Congress in Bucharest

All participants from the developed part of Europe, however, were pleased that the Romanian association used this opportunity to put pressure on its ministry and other government institutions, arranged a meeting with their representatives and with the EHC leadership, resulting in a joint memorandum on improving at least the basic care conditions in the Balkan country. This is the most important outcome of the conference; otherwise, the programme itself brought only few significant novelties.

First and foremost, the EHC leadership announced that it had raised the minimum requirement for concentrate consumption per capita per year for member countries to 3 IU. The requirement might seem absurd for most countries, but few people realise that four EHC member countries still rely exclusively on cryoprecipitate, meaning they have no concentrates available, and in another six countries consumption does not even reach one unit per capita. Almost unimaginable for us today.

The biggest topic was information about the development of concentrates with extended half-life. It turned out that the situation is somewhat less optimistic than it had seemed not so long ago. Especially for Factor VIII preparations, where achieving the right target is somewhat more complex. For haemophilia B, certain successes have already been achieved, so that for instance in prophylaxis it is possible to reduce the infusion frequency from twice a week to once every 14 days. Even that is a huge achievement. However, there is a danger that manufacturers will charge several times higher prices for their products. EHC President Brian O'Mahony pointed out that in 1996, pharmaceutical companies promised a flood of cheap recombinant products, but it remained just promises. Now price increases may also occur with the new extended-acting preparations. The EHC will pull out all the stops to prevent a dangerous increase in prices.

A few years ago, information about promising gene therapy also emerged. However, the situation here is even far less realistic than with longer-acting drugs. The British and Americans are indeed working jointly on developing such a possibility, but again it looks more promising for haemophilia B than A, and still only remotely so. Several people have already undergone gene therapy, which introduces genes without the defect causing insufficient Factor IX production into liver cells, but a goal usable in practice is still far away. And for haemophilia A, even further away.

Extending the half-life and efficacy of clotting factors can be achieved in two ways, and global manufacturers are testing both. One of them currently seems more promising, but the final results still require patient waiting. The only effective path to eliminating and curing haemophilia therefore remains liver transplantation, where coagulation factor production takes place. A path usable in practice only remotely.

What is important is that methods of improved therapy continue to be tested and have not ceased. The confirmation that haemophilia treatment has undergone the greatest advance of all genetically determined diseases in the last 25 years thus still holds true.