An inhibitor is when someone’s immune response to clotting factor products prevents it from working properly. Someone with an inhibitor can face many of the same challenges as someone without adequate treatment.
Inhibitors continue to be the major problem associated with treatment. Researchers are continuing to look at the role a wide range of factors play in the development of an inhibitor. There is particular interest in any non-genetic factors which might play a role as it may be possible to change some of these to reduce the risk to patients. For example there is evidence that when more product is used the first time someone is treated then their risk of developing an inhibitor is higher. This puts a premium on early diagnosis as younger children are typically treated with smaller amounts of product. Those who are diagnosed later often present with a bleed and therefore need more product the first time they receive treatment.
We were also informed about some of the potential therapies for inhibitors which are being explored; how can the immune system be taught to recognise Factor and tolerate it? A wide range of techniques from using viral vectors or nano-partials as delivery systems through to working with T-cells and Tregs to selectively switch off parts of the immune response are being explored.
Haemophilia Scotland are very grateful to Baxter Healthcare UK for there generous support which enabled us to attend the WFH Congress in Melbourne.