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.
For more information about new technologies, please consult our latest New Products Newsletters and videos on gene therapy.
Jim is aged 66 and has Haemophilia A with a high titer inhibitor which he developed at age 14.
Maria Elisa Mancuso (MD, PhD) is a Haematologist and works as a Senior Haematology Consultant at the Center for Thrombosis and Haemorrhagic Diseases of IRCCS Humanitas Research Hospital in Rozzano, Milan, Italy. She is Adjunct Clinical Professor at Humanitas University. She obtained a post-degree in Clinical and Experimental Haematology and a PhD in Clinical Methodology. She is involved in clinical research and has published several original articles in peer-reviewed journals a The Lancet, Blood, Journal of Thrombosis and Haemostasis, Haematologica, Thrombosis and Haemostasis, British Journal of Haematology and Haemophilia. She is reviewer for several peer-reviewed journals and member of the Editorial Board of JTH. She is a member of several scientific societies (ISTH, WFH, ASH, EAHAD, SISET, AICE) and was a medical member of the Inhibitor Working Group of the European Hemophilia Consortium. She is co-chair of the ADVANCE Study Group. She has acted also as co-chair of the Scientific and Standardization Subcommittee of ISTH on FVIII, FIX and rare bleeding disorders. She has been involved as principal and co-investigator in several clinical trials, and she takes care of both children and adults with hemophilia and other congenital bleeding disorders with a specific scientific interest in novel therapies, prophylaxis, inhibitors, and chronic hepatitis C.
As a patient with factor II deficiency, the diagnostic and treatment of rare bleeding disorders is a matter dear to my heart. My motivation to participate in the work of the ERIN committee is to improve both diagnostic and treatment for patients with rare bleeding disorders across Europe.
Economist and financial expert by profession, executive coach and trainer by passion and haemophilia advocate by every drop of my blood through my son (who has severe haemophilia A with inhibitors). Bringing a good decade of practical experience from the corporate insurance world, laser focus, growth mindset and resilience from my own experience, offering you anything I can just do, in hope that together we can make life more fulfilled for those impacted by bleeding disorders.
Amy Owen-Wyard is a Registered Mental Health Nurse. With experience working with children, young people, their families and adults with severe and enduring mental health conditions. Amy was also involved in a service improvement to provide a holistic care approach for those in general hospitals to support both their mental and physical health, whilst sharing her expertise and knowledge in mental health with the wider multidisciplinary team.
