Yesterday at the World Federation of Hemophilia in Argentina, I attended a session on the future of hemophilia care. Researchers from China to Germany presented many of the advances that have been made in the care of those afflicted with hemophilia well as the challenges that still exist.
It is hard to believe that in the 1960s many hemophiliacs who did not survive beyond age 20, could live a normal life span today. With treatment, complications of chronic joint disease can be avoided. However, there are still challenges in treatment throughout the world. Affordability and access to the products still make it difficult to receive treatment in many countries.
Here in Buenos Aires, one of the most exciting presentations was the introduction of a long acting product which will allow patients to be treated once weekly instead of the often 3 day per week regime. Can you imagine having to perform an intravenous infusion multiple times a week in your child or yourself? While these patients and their families have no choice now, at some point that may be decreased to once a week!
Even more exciting is the elimination of the disorder completely. One day with gene therapy, patients may no longer have to suffer the pain and complications of internal bleeding. There is still much to be done in the world of hemophilia, and I am extremely grateful to have met so many people from around the world with the same goal of improving care and ultimately the lives of those living with this disorder!
¡Hasta Pronto!
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