05/10/2015
Unconventional Topics at the European Assembly
ČSH
The annual conference of the European Haemophilia Consortium was hosted this time by Belgrade, Serbia. It addressed certain organisational details of the EHC, while the professional part discussed topics that are not often covered in such circles. Subjects of ageing, the female population, new inhibitor management, and also interesting technical innovations were debated. It became apparent how significantly civilisational processes affect the lives of people with hemophilia. And not always positively. Life expectancy is rising sharply — while in 1938 one could expect to live about 13 years, in 1964 it was 30 years, and now it is multiples of that. People with hemophilia are living to exactly the same age as the general population.
However, this brings with it a greater influence of the majority lifestyle and thus an increase in, for example, cardiovascular diseases. It is known that ischaemic heart disease and similar conditions were almost nonexistent among people with hemophilia — or rather, they used to be almost nonexistent. They are beginning to appear in increasing numbers, with the USA leading this unhappy trend. Besides living to an age where heart attacks become possible, unhealthy and predominantly sedentary lifestyles play a role, and so on. Specialists will therefore have to focus in the future on prevention and previously unprecedented treatment of such conditions. However, no binding recommendations yet exist, such as those that apply to prophylaxis or the treatment of acute bleeding episodes, and this will need to be rectified quickly.
There was much discussion in Belgrade about the problem known as inhibitors. Fortunately, there are only few cases in our country — about 16 — but if someone is affected by this problem, it is a nightmare. For supplementing clotting factor levels, there are essentially only two products: NovoSeven by Novo Nordisk and FEIBA by Baxter. It is impossible to determine definitively which is better or more effective, but for the first time it was openly stated that study results always come out better for the product whose manufacturer funded the study.
The EHC is just launching a major project that begins with the establishment of a specialised group for inhibitors. In 2015 it is being formed, next year it is to develop principles for treatment needs, and in 2017 they are to come into effect. Just as the EHC advocated for the adoption of binding standards for hemophilia treatment in the EU, it now wants to proceed similarly for cases of inhibitor development. Only the Netherlands has specialised centres for patients with inhibitors, but it is encouraging that the Czech Republic is among seven rare European countries that have been running specialised programmes for them for years.
An interesting initiative came from the University Hospital in Oslo. Their hemophilia centre uses joint ultrasound diagnostics on a wide scale to determine whether a joint is actually bleeding or whether the pain is caused only by arthritic changes. Magnetic resonance imaging can also reveal this, but it is very expensive and there are long waiting times. Not just in our country, but anywhere in the world. The Norwegians demonstrated the process right on the spot.
There was even talk of using simpler home self-diagnostic devices connected to smartphones. The benefit lies in a rapid response and more effective treatment at acceptable costs.
The use of extended half-life concentrates is considered a done deal in the most developed European countries. In the case of haemophilia A, the half-life of Factor VIII — that is, the duration of effectiveness — is extended 1.5 to 1.6 times; for haemophilia B, Factor IX, 2.4 to 2.6 times. This results in extended application intervals during prophylaxis and a reduction in the number of injections by about fifty per year, or increased protection through higher levels. Several institutions are conducting studies, but it is not yet possible to evaluate the impact of prolonged products on the potential development of inhibitors or certain negative effects that may only emerge after longer periods of use. In particular, the United Kingdom and Italy, which are at the forefront of research and development of such treatment products, speak of their future as a clear prospect. The question remains the price — so far, it seems it will increase proportionally to the increase in efficacy.
A number of European countries face very similar ethical problems in this context as we do. It concerns the quantification of treatment costs, especially in isolated cases of patients with inhibitors. For example, a British newspaper published an article stating that a particular boy cost 5.2 million pounds in one year. In our country, we protested to the General Health Insurance Company (VZP) some time ago against the publication of a ranking of almost unambiguously identifiable clients; Director Zdeněk Kabátek promised a remedy, but never kept his promise. This is just a side note to illustrate that we are not alone in Europe in fighting windmills.
In the selection of the host city for the EHC annual conference, Vilnius emerged victorious for 2017, significantly defeating Baku. In evaluating the organisational level of the congress, it once again became clear how well the Czech Hemophilia Society had performed in preparing the 2012 event. It is still fondly remembered among European federations, also because this year's event by the Serbian colleagues did not turn out particularly well.