As this is my first column, I would like to introduce myself. At the start of my career I worked in adult hematology for three years before I switched to pediatrics to become a pediatric hematologist. After working many years as a pediatric hemato-oncologist, I decided to focus on hemostasis; and so from 1992 to 2008 I was the director of the Van Creveld Kliniek, the Dutch National Hemophilia Centre.
After switching my career focus for four years working on the board of directors of a large teaching hospital I realized I missed hemophilia research dearly. Presently, I work as a senior investigator at the University Hospital in Utrecht and as the Director of the PedNet Registry, a collaboration of 31 hemophilia centres from 16 countries.
The Journey Begins
International collaborations are very important to improve the knowledge of rare diseases such as hemophilia. And if you attended the WFH 2014 World Congress in Melbourne, you’ll understand what I mean.
My journey as VP-medical started, officially, at Congress. And it was a memorable one, as both the venue and the city added to the overall experience. Our local hosts and organizers, together with the WFH staff, did their utmost to have all this combined with a very interesting program. There was ample time to meet and interact with colleagues and friends. This is crucial for a conference such as ours, where the benefits are not only the dissemination of scientific knowledge but also the interaction between all disciplines involved in hemophilia care. Most importantly, of course, the WFH is a patient organization and so many people with hemophilia were involved in all aspects of the program.
The farewell event was a good example of what the venue was capable of: a wonderful dinner and a great party. For me and many others, to see so many young people dancing and having a great time, was a highlight of the evening: this is what modern hemophilia care has made possible.
Importance of Prophylaxis
In the Netherlands, we adapted prophylaxis very early and during my career as a hemophilia doctor I was able to observe the benefits of early prophylaxis.
I have seen many young children receive their diagnosis and the distress this gave to their parents. After the start of prophylaxis and the acceptance of frequent infusions, they feel that their life has come back. Bleeding is reduced to normal and children can be allowed to participate in regular activities without concern that they will develop abnormal joints.
In the Netherlands, like in many European countries, all treatment costs, both in the clinic and during home treatment, are completely covered by health insurance. Obviously, this helps parents and physicians to make the choice to start treatment regardless of the cost of clotting products.
The benefits of prophylaxis are many, and after finding their way with venous access, families and children experience a normal life, participating in the same activities as their peers. When venous access is indeed feasible, home treatment is given by the parents or by local health care personnel. This has made a huge difference, not only for the families but also for the supportive care provided by the hemophilia treatment centres. Treatment centres in the developed world have changed from providing mainly emergency care to becoming support and knowledge centres that focus on defining care for treatment complications, such as inhibitors and musculoskeletal problems.
Decades ago, WFH and WHO acknowledged that prophylaxis is the standard of care for all patients with severe hemophilia. However, it is still debated when to start and how to dose. Recent studies have demonstrated that 55 per cent of the children with severe hemophilia are born in families unaware of hemophilia. In these cases, the diagnosis is often made only after several bleeds, so that by the time of diagnosis patients have already had joint bleeds as well. Starting prophylaxis is a large step, even more so for families without members with long-standing arthropathy. Patient organizations can increase awareness and help families cope with the need for frequent infusions. The biggest gain for patients is to prevent their joints from bleeding; and this must begin at the earliest age possible.
It is with great enthusiasm that I support the initiative of the WFH and its partners in the collection of plasma and the development of products, so that we may have a sustainable donation program for countries that still have no treatment at all. Of course this can only succeed when governments are involved as well.
We all have embraced the main goal of the WFH in closing the gap. This means that we work hard not only to increase the number of patients that get a diagnosis, but also to make sure they will have access to clotting factor products and, eventually, prophylaxis to prevent bleeding and to improve their long-term outcome.
Are we able to deliver care and clotting products to many more patients? Can we introduce prophylaxis in children that presently have not received regular treatment? These are the most important questions for me in the coming years and I hope with all our efforts together we will be able to answer them with a resounding yes!