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Presentation by Professor Frits Rosendaal -
Presentation by Professor Flora Peyvandi -
Participants of the event -
MEP Miroslav Mikolášik welcome remarks -
MEP Norica Nicolai with presenters
On March 7th 2017, the European Haemophilia Consortium (EHC) held its first Round Table of Stakeholders of the year on ‘Clinical Trials in Haemophilia’.
The event, which took place in the European Parliament, was hosted by Member of the European Parliament Mrs Norica Nicolai (Romania/ALDE), and additionally attended by MEP Dr Miroslav Mikolášik (Slovakia/EPP). Both are longtime supporters of EHCs’ efforts to improve the quality of life for the haemophilia community.
More than 50 participants were present, including patient representatives, healthcare professionals and representatives from the pharmaceutical industry. After welcoming remarks, the scientific part of the programme was kicked off with an introduction to how epidemiological data in the field of haemophilia is collected. The presentations that followed build on the topic by covering aspects such as drug and side-effects, how long-term observation of efficacy for novel therapies is carried out, the safety of these therapies, as well as the angle of previously untreated patients (PUPs) in clinical trials.
Clinical trials are of crucial importance for all patients. But one of the big dilemmas in haemophilia is what causes inhibitors – the antibodies acting against the received treatment. Those affected by inhibitors are unable to continue their regular treatment regimen and are left more vulnerable to devastating physical and psychological effects, such as disability and social isolation. It is now of great significance to observe whether new treatments will also cause inhibitors and whether they can be used to treat people with inhibitors at all.
For the full programme, speakers’ biographies, presentations and all other relevant documents from the event click here, or visit our library section.
Pictures from the day can be viewed on the EHC Facebook page.
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.
