More Questions Than Answers

The standing theme of the WFH remains the motto "Treatment For All." Fortunately, countries in the northern Euro-Atlantic area no longer need to address this, but globally, treatment remains available to only about 75-80 percent of those who need it. This naturally influences congress proceedings and will likely continue to do so for a long time. For us, however, other topics are more interesting.

It is well known that the research, development, and therapy of no other genetically determined disease has undergone such progress in the last quarter century. The population is ageing, people with haemophilia are living to the same age as the rest of the world's population, and new problems are emerging that nobody anticipated just a few years ago. For example, until recently, myocardial infarction was rare among haemophilia patients. That is no longer the case -- in advanced European, North American, and some Asian countries, complications are appearing. This is related to the fact that problems associated with the underlying disease have been largely eliminated, and other conditions are emerging that were previously reserved only for those who are -- as far as blood clotting is concerned -- healthier.

Medical experts in Melbourne therefore discussed whether to also administer medications that work in the opposite direction, i.e. against thrombosis, when needed. This particularly concerns post-operative states; orthopaedic surgeries are increasingly common. So should medications such as warfarins or heparins be administered or not? Numerous experts spoke on this topic, but no definitive conclusion was reached. Most, however, tend towards the opinion that they should. Bleeding can always be dealt with somehow, but thrombosis can have absolutely fatal and irreversible consequences.

After many years, warnings about the negative consequences of prophylaxis also emerged. Until now, only its benefits had been discussed, and there is still no doubt about them. However, someone calculated that after sixty years of prophylaxis, the recipient receives at least 9,360 injections. And that can be a problem. Today, in the most advanced countries with high concentrate consumption, many have indeed reached this milestone -- and a problem has arisen. Venous access points are so damaged that there is practically nowhere left to insert a needle. Central access then comes into play, but there is a considerable risk of infection.

Medicine will have to come to terms with these and other difficulties. A major topic today is concentrates with extended half-life, but not everything is rosy there either. The main method is so-called PEGylation, i.e. binding the active substance to other molecules. However, nobody yet knows exactly how the body -- specifically the liver -- will handle PEGylated molecules during their elimination. For haemophilia B, the effectiveness of products has already been extended approximately six to seven times, but for haemophilia A, only 1.6 times. That is not much, but on the other hand, with long-term prophylaxis, approximately fifty injections per year can be saved. And that -- in light of the new circumstances -- is not to be dismissed either.

Add to this the evergreen of debates about the development and treatment of inhibitors, and we truly have more topics to address than enough. Nothing is free, nothing is purely rosy -- in short, there are more topics to ponder than enough.

More and more companies are developing programmes for electronic reporting of concentrate consumption and thus easier statistics. Today, of course, primarily for smartphones and tablets, making everything more mobile and practical. In the near future, we will bring specific tips on some applications for devices using Android or Apple and will try to promote their use in treatment centres. They will help not only patients but also the centres themselves. And it will be up to Society members whether they push for their adoption at their centres.

Vladimir Dolejs