Clinical Study in Ireland Aims to Improve Care for Hemophilia Patients by Using Personalized Approaches

A novel clinical study called “The Irish Personalized Approach to the Treatment of Hemophilia (iPATH)” will seek to investigate the potential of new personalized treatment approaches in hemophilia by tailoring care based on the needs of individual patients.

The collaborative study will be led by the Irish Hemophilia Society, in partnership with Shire, a global biotech company that specializes in rare diseases, the Royal College of Surgeons Ireland (RCSI) and Science Foundation Ireland (SFI).

iPATH will divert from standardized treatments and study hemophilia patients in Ireland who are registered at a single National Coagulation Centre, where data on the use of factor concentrates and bleeding rates have been collected, to conduct a study aimed at better understand the underlying causes and mechanisms of the blood disease. Ultimately, the parties involved have the goal of developing personalized approaches to care that eventually can be extended to the global community.

“Today, in developed countries, most patients receive prophylactic treatment, which is recognized as the standard of care. For those patients on prophylaxis, treatment should be optimized by combining innovation with personalization,” Peter Turecek, senior director of global medical affairs at Shire, said in a press release. “Through the iPATH study, we hope to uncover new solutions that build on and maximize the role of factor therapy and further personalize care for hemophilia patients.”

“Hoping to enhance quality of life for people with hemophilia … we need to begin developing innovative treatment strategies that can be tailored specifically according to the needs of each individual patient. To achieve this objective, we first need to understand the biological mechanisms that underpin the marked differences in bleeding risks and long-term complications that exist between individual patients with hemophilia,” said James O’Donnell, study leader and director of the Irish Centre for Vascular Biology at RCSI. “By understanding these mechanisms, the iPATH study could potentially pave the way for the introduction of personalized medicine for patients with hemophilia,” he said.

“The future of hemophilia care should be based on a personalized approach to treatment. We anticipate that this exciting, innovative and collaborative research program may provide us with answers to potentially optimize future treatments for individuals with hemophilia in Ireland and to hopefully further apply this research to hemophilia patients globally,” added Brian O’Mahony, chief executive of the Irish Hemophilia Society.

iPATH will be conducted over the course of four years, with the support of a strategic partnership initiative between SFI, RCSI, Trinity College Dublin (TCD) and Shire. The partnership also includes clinical researchers based in several Dublin hospitals and is designed to be potentially adapted for other diseases.