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A single centre experience of prophylactic rFVIIA in paediatric patients with severe haemophilia and inhibitors

Abstract number: PP-WE-615

Breen¹ K.A., Kavanagh¹ M., Kelly¹ I., Nolan¹ B., Regan¹ I., McMahon¹ C.

¹¹Haematology, Our Lady's Children's Hospital, Dublin, Ireland

How-to-cite Breen KA, Kavanagh M, Kelly I, Nolan B, Regan I, McMahon C. A single centre experience of prophylactic rFVIIA in paediatric patients with severe haemophilia and inhibitors. Journal of Thrombosis and Haemostasis 2009; Volume 7, Supplement 2: Abstract PP-WE-615

Prophylactic use of factor concentrates has been demonstrated to reduce the incidence of bleeds and joint damage in those with severe Haemophilia. Development of neutralising antibodies (inhibitors) to factor VIII and IX has become an increasingly significant problem in the management of Haemophilia and occurs in 15–30% of those with FVIII and 3–5% with FIX deficiency. Patients with high titre inhibitors experience bleeding episodes that are difficult to treat and frequently lead to the development of chronic arthropathy. rFVIIa is an effective treatment for such bleeds. It may also prove useful for bleed prevention in these patients but there is little supporting evidence, especially in Paediatric populations. Six paediatric patients aged 2 months–11 years, (median 4.5 years) with severe FVIII deficiency (5) and severe FIX deficiency (1), and inhibitors, were treated with rFVIIa prophylaxis from 2002 to 2007 in our centre. Four had high responding inhibitors with peak levels of 14–712 BU and the other two had levels of 1.3 and 3.5 BU. Two had int-22 h mutation. Five children were on concurrent immune tolerance therapy. All patients experienced significant bleeding episodes (joint, intracranial and gastrointestinal) prior to commencement of prophylaxis. The dose of rFVIIa prophylaxis administered was 90–180 μg/kg/day. Prophylaxis was continued until the inhibitor was no longer detectable. Four children received prophylaxis for a period of 4–16 months. Two continue on prophylaxis. The average number of bleeding episodes prior to commencing rFVIIa prophylaxis was 3.5 per month compared to 1.6 per month during prophylaxis. There were no rFVIIa associated thrombotic complications during prophylaxis. We conclude that prophylaxis with rFVIIa reduces the number and severity of bleeding episodes in a paediatric population with severe Haemophilia and inhibitors.

Disclosure of interest: none declared.

To cite this abstract use the following format:

Journal of Thrombosis and Haemostasis 2007; Volume 5, Supplement 2: abstract number

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