Over the past weekend some 70 delegates from across Europe gathered in sunny Athens to hear all about the latest advances in new treatments in haemophilia. This was the seventh edition of the workshop and the most attended, which underscores the growing interest and educational need.
This workshop is one of several activities developed by the EHC to try to bring more knowledge to its community about innovative treatments. The main difference between this and other EHC events, however, is the workshop’s ‘community-only’ tradition. This is to ensure complete freedom for patients and healthcare professionals to ask any question or raise any point they wish to, under Chatham House Rules. This means that participants are free to use the information received, but neither the identity nor the affiliation of participants, may be revealed.
This year’s workshop was divided into three parts: licensed therapies, therapies under development and gene therapy.
In the licensed therapies part, participants heard presentations on the clinical use of emicizumab in patients with inhibitors, considerations on when and how to switch patients without inhibitors to emicizumab, presentations on longer-acting replacement therapies, and safety and economic considerations on novel therapies. Participants also heard an update on therapies for von Willebrand Disease. In the ‘therapies under-development’ section, participants were given an update on anti-TFPI and SerpinPC. In the session on ‘gene therapy,’ besides receiving an update on the status of the clinical trials, speakers covered potential re-treatment with vector and explored thus-far unanswered questions regarding adeno-associated viruses. Ethical considerations regarding gene therapy in children and how we will be able to afford this novel therapy were also discussed. Both days featured panel discussions in which patients and clinicians were able to discuss treatment strategies using novel therapies and any other unanswered questions for the day.
Besides offering an opportunity for learning and keeping abreast with the latest developments in treatments, the workshop also shed light on the ‘hot topics’ within the community. This year, participants focused on the current lack of long-term data on treatment use with novel therapies. It seems that clinicians, just like patients, are wrapping their head around all of the treatment options that will available and how to assess which treatment will better be suited to which patient. This leads to the conclusion that there will be an even greater need for personalization of care depending on the patient’s family history, life-style and treatment goals. Another discussion item regarded how to maintain links with patients who might feel that they no longer need their treatment centre or NMO as much, and the responsibility of that NMO or Centre to manage good education and communication around these issues.
All in all, the weekend was packed with lots of very interesting and thought-provoking discussions and we can only but recommend to all of our members to keep an eye on the 2020 edition, which will take place from 20 to 22 November.