The results of the SIPPET (Survey of Inhibitors in Plasma-Products Exposed Toddlers) study were published today in the New England Journal of Medicine. The paper suggests that, in previously untreated patients ( PUPs), the risk of developing an inhibitor when using recombinant factor VIII products is higher than when using plasma-derived factor VIII concentrates. This is a significant study and the results may prove to be important. There is no evidence of a higher risk of inhibitors with recombinant FVIII in previously treated patients (PTPs).
The study was conducted between 2010 and 2014 and included PUPs with severe hemophilia A who were treated at 42 participating sites in 14 countries from Africa, Asia, the Americas, and Europe. In the study, 251 PUPs were randomized so that 125 patients received plasma-derived FVIII (which contained von Willebrand factor) and 126 received recombinant FVIII. The study used a variety of plasma-derived and recombinant FVIII clotting factor concentrates. The study compared two classes of FVIII concentrates – plasma-derived and recombinant – so no conclusions can be made about the risk associated with specific products.
EHC will make further comments in the coming week.
The paper can be viewed 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.
