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Initial Data from Shire Collaboration Identifies Key Biomarkers That Affect Bleeding Patterns, Response to Treatment for Hemophilia A #ASH18

Shire plc, RCSI (The Royal College of Surgeons in Ireland), and Science Foundation Ireland (SFI), in collaboration with the Irish Haemophilia Society, today presented new data, from the novel clinical study called The Irish Personalised Approach to the Treatment of Haemophilia (iPATH), illustrating key drivers to implementing truly personalized care for people with hemophilia A during the 60 th American Society of Hematology (ASH) Annual Meeting in San Diego, California.

Standard of care treatment for hemophilia A currently is prophylaxis with recombinant factor VIII (rFVIII) replacement therapy, an approach designed to minimize bleeding episodes using a weight-based dosing strategy. 1 However, the half-life of factor VIII may vary markedly between individual patients, meaning some patients experience FVIII levels too low for sufficient protection from bleeds much earlier than others. 

As a result, even with prophylactic treatment, some people continue to experience bleeding events. 2 This approach can be improved by integrating FVIII pharmacokinetics (PK) to produce an individualized treatment regimen. 2 In a poster (Poster #1190) presented today at the ASH meeting, captured as part of the ongoing iPATH study, researchers found important biomarkers that influence bleeding risk in people with hemophilia, including a link between type O blood group and shorter half-life, and a link between age and longer half-life. 2 The study also supports the feasibility of using limited, 2-sample PK profiling to capture accurate PK curves that can then be utilized to guide individualized, PK- guided prophylaxis. 2 The 2-sample PK profiling was done through the use of myPKFiT TM for ADVATE ® [Antihemophilic Factor (Recombinant)], a free web-based, Rx software for use with hemophilia A patients 16 and older weighing at least 45 kilograms and treated with ADVATE. 3 The four-year iPATH program is open to all Irish children and adults with moderate or severe hemophilia. 4 The study is supported by a Science Foundation Ireland (SFI) Strategic Partnership initiative and involves scientific researchers in RCSI, Trinity College Dublin (TCD) and Shire.4

The iPATH partnership includes the Irish Haemophilia Society, and clinical researchers based in St James’s Hospital Dublin, Our Lady’s Children’s Hospital Crumlin, Cork University Hospital and University Hospital Galway. 4 “This research offers important insights about how we can apply personalized treatment planning for hemophilia as part of routine clinical care. By understanding the link between clinical parameters such as age or blood type, we can appreciate differences in PK and thus response to prophylactic care,” said study lead Professor James O’Donnell, Director of the Irish Centre for Vascular Biology, RCSI, and a Consultant Hematologist at the National Coagulation Center in St James’ Hospital, Dublin. “Importantly, this also supports the accuracy of simplified, 2-sample PK profiling, giving clinicians confidence that they can generate a reliable PK curve to guide individualized PK-guided dosing and minimize bleeds for their patients.” 2 “Every single bleed matters for people with hemophilia. 

Particularly if a joint or muscle bleed remains untreated, each one can lead to other long term consequences. So a core objective of this research is to better understand the specific characteristics of individuals that affect how and when they are likely to bleed,” said Peter Turecek, Senior Director, Global Medical Affairs, Shire. “If we can individualize treatment schedules by estimating PK curves through myPKFiT for ADVATE, we can further personalize treatment with the goal of getting as close as possible to zero bleeds.” 

“I am very pleased to see such promising research results arising from the iPATH collaboration, a partnership that will positively impact treatment for patients of hemophilia around the world,” said Professor Mark Ferguson, Director General of Science Foundation Ireland and Chief Scientific Adviser to the Government of Ireland. “Improving medical approaches to this debilitating disease is crucial, and at Science Foundation Ireland we recognize the importance of funding scientific research that helps us to achieve that. I am confident that this research will provide further insights which will inform best practice for the future of hemophilia treatment, and I look forward to our continued work with Shire and RCSI.”

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