The European Haemophilia Consortium’s (EHC) periodic review of novel treatments in haemophilia and other bleeding disorders is a review entirely developed by the EHC to help educate EHC National Member Organisations (NMOs) about the rapidly changing landscape of medicinal treatment in rare bleeding disorders. The EHC encourages its NMOs to use and adapt this document to their national needs but takes no responsibility for any changes.
The review covers recent major developments and is divided by different types of disorders for which there is an update to report. The information provided in this publication is compiled from multiple sources, including presentations at recent scientific meetings, websites, financial information and writing directly to pharmaceutical companies. It is then redrafted and presented in easy-to-understand language. This review is updated periodically.
The EHC is grateful for the work and support of the Novel Treatment Review Committee, which has overseen the content and production of this document. The EHC also wishes to give Miguel Crato, EHC President, special thanks for his work on these publications.
The EHC greatly welcomes all treatment developments that may benefit patients in the future. The EHC takes no position on any product type or class reported in this review.
We hope that the information provided herein is useful and we are available for any questions.
Consult all the issues of the EHC Novel Treatment Review here:
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.
