| Every bleed matters in hemophilia; each event can damage the joints and muscles and contributes to considerable long-term disability and morbidity.1 But like a fingerprint, each patient’s experience is different. Their needs, their activities and their biological characteristics impact their risk of bleeding events. Unraveling the individual patient experience is central to innovation that can advance outcomes.
“We have made great progress in treating hemophilia, but we need to better understand the individual characteristics that impact how and when patients will bleed. For some, it is about their personal activity, and for others, it has more to do with their biological patterns,” said Professor James O’Donnell, Director of the Irish Centre for Vascular Biology, Royal College of Surgeons in Ireland (RCSI); Consultant Hematologist at the National Coagulation Center in St James’ Hospital, Dublin, and lead for the Irish Personalized Approach to the Treatment of Haemophilia (iPATH) study. “As research continues to uncover these markers, we will be better equipped to tailor our approach for each patient to dramatically reduce their bleeds.”
Research programs presented at the 2018 American Society of Hematology’s (ASH) Annual Meeting illustrate precisely how tailored approaches may advance care for these life-long bleeding disorders.
Tailoring regimens to pharmacokinetics (PK) may improve the treatment experience for patients with hemophilia A
People with hemophilia A have more treatment options than ever, and with prophylaxis as the standard of care, success rates in managing bleeds are high for many patients.2 Now, data indicate that adopting a highly individualized approach to treatment schedules may further improve results.3
New research from the iPATH study, an ongoing partnership between Shire, RCSI and Science Foundation Ireland (SFI), in collaboration with the Irish Haemophilia Society, investigating new personalized treatment approaches, finds important biomarkers, including blood type and age, that influence bleeding risks.3 The study also supports the use of PK to guide dosing based on individual PK profiles, maintaining proper levels of factor replacement therapy to reduce bleeding risk.3The findings validate the use of limited, 2-sample PK profiling to capture accurate PK curves that can guide individualized, PK-guided prophylaxis as part of routine clinical care.3
The four-year iPATH program is open to all Irish children and adults with moderate or severe hemophilia. The study is supported by a Science Foundation Ireland (SFI) Strategic Partnership initiative and involves scientific researchers in RCSI, Trinity College Dublin and Shire. 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.
“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. Improving medical approaches to this debilitating disease is crucial, and at Science Foundation Ireland we recognise the importance of funding scientific research that helps us to achieve that,” said Director General of Science Foundation Ireland and Chief Scientific Adviser to the Government of Ireland, Professor Mark Ferguson. “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.”
New insights on individual markers could facilitate early intervention for inhibitor development
The development of inhibitors remains one of the most challenging and unpredictable complications of hemophilia. Only some patients will develop inhibitors to their factor replacement therapy, and the reasons for the immunogenic reaction are not entirely clear.4
A new initiative evaluates potential early markers of inhibitor development in newly diagnosed, previously-untreated patients (PUPs) – the Hemophilia Inhibitor PUP Study (HIPS). Findings from this prospective multicenter observational study reveal factor VIII (FVIII)-specific antibody development and switching patterns that only occurred among patients who developed inhibitors to factor replacement therapy. This suggests that these FVIII-specific antibodies may serve as an early predictor of inhibitor development, offering an opportunity to accelerate diagnosis and prompt intervention.4
Personalization: the foundation for the next generation of care
Together with leaders across the hemophilia community, we continue to build evidence that individualized care through PK-guided prophylaxis is an important strategy to improve care, including research of novel approaches to integrate individualized strategies into routine care.2 As therapeutic options evolve, we predict that those precisely designed to meet a patient’s needs – whether young or old, an athlete or an academic – will offer the best opportunity to pursue our goal of zero bleeds.
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1 Sahu, S et. Al. Revisiting hemophilia management in acute medicine. Journal of Emergencies, Trauma and Shock. 2011 Apr-Jun; 4(2): 292-298.
2 Valentino, LA et. Al. A randomized comparison of two prophylaxis regimens and a paired comparison of on-demand and prophylaxis treatments in hemophilia A management. Journal of Thrombosis and Haemostasis. 2012 Mar; 10(3): 359-367.
3 Lavin, M et al. The Irish personalized approach to the treatment of haemophilia (iPATH) – determinants of inter-individual variation in FVIII pharmacokinetics, poster #1190. Presented at American Society of Hematology Annual Meeting 2018 on December 1st.
4 Gangadharan, B et al. Data coming out of the Human Inhibitor PUP study (HIPS) reveal 4 subgroups of patients with distinct antibody signatures, poster #3374. Presented at American Society of Hematology Annual Meeting 2018 on December 3rd.
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