Here you can find:
Presentations:
- Welcome and introduction, Prof Paul Giangrande
- Raising awareness for VWD, Dr Antoine Rauch
- Living with VWD, Ms Baiba Ziemele
- Prophylaxis in VWD, Dr Susan Halimeh
- Combined FVIII/ vWF versus RvWF, Prof Pier Mannuccio Mannucci
- Role of vWF in preventing inhibitor development and facilitating immune tolerance: Focus on clinical trials evidence, Dr Carmen Escuriola-Ettingshausen
- Role of vWF in preventing inhibitor development and facilitating immune tolerance: Focus on immunology, Dr Srini Kaveri Part 1 – Part 2
- Acquired VWD, Dr Augusto B. Federici
On 16 June 2014, the European Haemophilia Consortium (EHC) organised its second Round Table of Stakeholders in Brussels, Belgium, to bring the EHC membership up to date on the current clinical developments in von Willebrand Disease (VWD) and factor (VWF). The event brought together around 40 participants representing the EHC membership, physicians, industry and other interest groups such as hospitals and hospital pharmacists.
Prof Paul Giangrande, chairman of the EHC Medical Advisory Group (MAG), chaired the event and opened the session by indicating that although VWD is one of the most common bleeding disorders, it is still largely under-diagnosed due to the fact that its symptoms are often treated individually and not seen as part of another condition.
The event then proceeded with two main topics: clinical developments in the diagnosis and treatment of VWD and the role of VWF in the prevention of inhibitor development. A final talk looked at the latest findings in the diagnosis and treatment of acquired von Willebrand syndrome (AVWS).
A few findings emerged from the presentations and discussions. It appears that VWD is still a difficult disease to diagnose, as demonstrated by Ms Baiba Ziemele, who currently still has no certainty about which type of VWD she is affected with. Diagnosis should be based on VWF levels but also on family history and the bleeding score. As a result of the difficulty in diagnosis, we note both over-diagnosis and under diagnosis of the disease currently taking place in developed and developing countries respectively.
VWD affects both primary and secondary haemostasis. This is caused by the dysfunction of platelet adhesion and the reduction of factor VIII activity in plasma. The therapeutic options should correct both defects. In terms of treatment regimen, this will vary greatly depending on the type of VWD affecting an individual but also depending on the bleeding profile. For patients with severe VWD and suffering from a greater number of bleeds, prophylaxis should be considered. However, it is important to note that this treatment course is quite expensive and may not be available in all European countries. Ultimately, the treatment regimen should be designed in accordance between patients and physicians. From the discussions it emerged that at the moment there are enough satisfactory treatments for this condition, however there are challenges in accessing these treatments, performing the right diagnosis and determining the correct treatment regimen for each patient.
With regard to the role of VWF in preventing inhibitor development, it appears that there is still great uncertainty regarding whether VWF does in fact play a role. Both presenters on this topic concluded that further research was needed.
Finally, AVWS is an acute syndrome, which takes place later in life and often following surgery or cancer treatment. It was noted that most patients affected by this syndrome go into complete remission, however timely diagnosis and treatment greatly improve morbidity rates.
The presentations were followed by lively discussions in which several people affected by VWD were able to share their experiences on living with the disease and accessing correct diagnosis and treatment.
Speakers at the Round Table included:
• Dr Antoine Rauch, University of Lille, France
• Ms Baiba Ziemele, President of the Latvian Haemophilia Society, Latvia
• Dr Susan Halimeh, Gerinnungszentrum Rhein-Ruhr, Germany,
• Prof Pier Mannuccio Mannucci, University of Milan, Italy
• Dr Carmen Escuriola-Ettingshausen, Haemophilia Centre Rhein Main, Germany
• Dr Srini Kaveri, Centre de Recherche des Cordoliers/ INSRM, France
• Dr Augusto Federici, University of Milan, Italy
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.
