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A new major €4 million, patient-orientated study entitled: “The Irish Personalised Approach to the Treatment of Haemophilia (iPATH)” was announced on Monday (4th December) by the RCSI (Royal College of Surgeons Ireland), Science Foundation Ireland (SFI) and Shire, in partnership with the Irish Haemophilia Society.

This study will develop new and innovative approaches to improve the clinical care of patients with the inherited bleeding disorder haemophilia, a genetic disorder that affects approximately 1 in 3000 males in Ireland.

The aim of the iPATH study is to replace the current ‘one size fits all’ global treatment standard with a new personalised treatment approach, where tailored treatments will be based on individual patient needs.

It has been claimed that this study, which will be led by Professor James O’ Donnell, Director of the Irish Centre for Vascular Biology, RCSI, and a Consultant Haematologist in the National Coagulation Centre in St James’ Hospital, Dublin, will establish Ireland as a world leader in the development of personalised medicine for patients with bleeding disorders.

Professor O’Donnell explained that the time had come to move beyond the principal focus of physicians caring for patients with haemophilia; ensuring that their treatments were effective, safe, and free from viral contamination.

He stated that the next step would be to enhance the quality of life of people with haemophilia in the future, by developing innovative treatment strategies tailored to specific patient needs.

He continued: “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 haemophilia.

“By explaining these mechanisms, the iPATH study will pave the way for the introduction of personalised medicine for patients with haemophilia”.

The four-year iPATH programme is open to all Irish children and adults with moderate or severe haemophilia. The study is supported by an SFI Strategic Partnership initiative and involves scientific researchers in RCSI, Trinity College Dublin (TCD) and Shire, in addition to partnership with 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.

“To improve standards of care for people with haemophilia we need to combine innovation with personalisation to optimise treatment outcomes,” said Peter Turecek, Senior Director, Global Medical Affairs, Shire.

Commenting on the announcement, Director General of SFI and Chief Scientific Adviser to the Government of Ireland, Professor Mark Ferguson, expressed his delight at tyhis collaboration, stating; “The iPATH partnership combines the best expertise from industry and academia to improve treatment options and outcomes for patients with haemophilia worldwide”.