Although in recent years ITI (immune tolerance induction) therapy has proved to be a highly effective approach, factor VIII inhibitors are still the most severe complication that can be encountered in the treatment of haemophilia A. For this reason, the scientific community has decided to set itself an ambitious new goal: to prevent the appearance of inhibitors.
We already know of some genetic and environmental risk factors that play a decisive role in inducing the immune system to react against infused factor VIII; however, the mechanisms that lead to the production of inhibitors are so complex and involved that the information currently in our possession is nothing more than the tip of the iceberg.
Finding out exactly why and how the body produces inhibitors could lead to the development of markers, or “molecular signals” making it possible to quickly and precisely identify those subjects most at risk. This would make it possible to take action against inhibitors even before they appear.
It was precisely with this aim that the NHLBI (National Heart, Lung and Blood Institute) SOS (State of the Science) Workshop was established in the United States. This workshop, which was held in May 2018, brought together the most authoritative experts in haemophilia, immunology, genomics, proteomics and other important disciplines, to devise a coordinated strategy in order to conduct, in the years to come, research on the development of inhibitors that is more productive than ever before.
The work by the members of the SOS Workshop panel, which was recently published, consists in a detailed summary listing the main aspects to be explored and the strategies to be adopted in order to fully understand the mechanism underlying the formation of inhibitors, in order to effectively prevent their appearance
The workshop represented a valuable opportunity to focus attention on a topic of great current interest in the world of haemophilia, and a source of hope for all patients with this illness.
- Sabatino DE et al. Origins and organization of the NHLBI State of the Science Workshop: Generating a national blueprint for future research on factor VIII inhibitors. Haemophilia. 2019(25), 575–580