Some of the mechanisms behind the success of ITI (immune tolerance induction) therapy could soon be revealed. An on-going study is monitoring the way in which the body of 20 patients with severe haemophilia A and inhibitors reacts to the administration of ITI, evaluating both the response by the immune system of people undergoing this treatment and the changes that take place in their gene expression. The study, which involves a number of Haemophilia Centres in Italy and Canada, and was recently presented at the Joint Meeting between the 10th edition of the BIC (Bari International Conference) and the 3rd International Conference on Inhibitors in Haemophilia, held in Genoa between 6 and 8 September this year, has revealed important differences in the production of certain molecules between people who respond well to therapy, and achieve immune tolerance, and those in whom ITI is not efficacious. The study has two main objectives:
- to identify specific molecules that act as markers, i.e. that make it possible to understand whether the body is reacting adequately to the on-going ITI therapy;
- to fully understand the mechanisms that govern the immune system’s response to the therapy, especially in patients in whom ITI is not effective.
ITI is the only treatment able to eradicate inhibitors, which constitute the greatest threat that a patient with haemophilia can encounter. ITI currently has a success rate of approximately 60-70%; however, fully understanding the mechanisms underlying its success may considerably improve its efficacy, making the prospect of an inhibitor-free future an increasingly realistic idea.
- Rossini S. et al, Prospective Study Of The Immunological Response To Factor VIII In Severe Hemophilia A Patients During Immune Tolerance Induction Treatment. Poster presented at the Joint Meeting between the 10th edition of the BIC (Bari International Conference) and the 3rd International Conference on Inhibitors in Haemophilia. Genoa, 6-8 September 2019. Link: https://bic2019.org/posters/2019/07/31/prospective-study-of-the-immunological-response-to-factor-viii-in-severe-hemophilia-a-patients-during-immune-tolerance-induction-treatment/