On-demand treatment of bleeds in haemophilia patients with inhibitors: strategies for securing and maintaining predictable efficacy with recombinant activated factor VII

Authors


Benny Sørensen, Haemostasis Research Unit, Centre for Haemostasis & Thrombosis, Guy’s & St Thomas’ NHS Foundation Trust, 1st Floor North Wing, St Thomas’ Hospital, Westminster Bridge Road, London SE1 7EH, UK.
Tel.: +44 (0) 20 718 82788; fax: +44 (0) 20 740 13125; e-mail: benny.sorensen@kcl.ac.uk

Abstract

Summary.  On-demand therapy with recombinant activated factor VII (rFVIIa) can provide effective haemostasis for spontaneous bleeds in haemophilia patients with inhibitors. However, treatment approaches vary amongst physicians, positively or negatively affecting outcomes. A panel of physicians proposed recommendations for securing and maintaining predictable efficacy with rFVIIa, comparing these with ‘real-life’ patient management, using a questionnaire circulated to other expert physicians from haemophilia care centres in Europe and the United States. For rFVIIa treatment of spontaneous bleeds in inhibitor patients, early intervention with the highest appropriate dose is recommended. Home-based therapy can facilitate early intervention. If additional rFVIIa therapy is required after the initial dose, rFVIIa 90 μg kg−1 may be administered at 2–3 h intervals. Treatment should be tailored to bleed site/severity, recognizing the advantages of appropriate adjunct therapy. Questionnaire results suggested that many respondents adopted strategies in line with the recommendations. Most (36/46) recommended initial therapy within 1 h of bleed onset. rFVIIa 270 μg kg−1 was the most frequently prescribed/recommended initial dose for paediatric (aged ≤15 years; 22/44 respondents) and adult (aged >15 years; 23/44 respondents) patients. However, there may be opportunity for improved bleed management on occasion, with regard, for instance, to dosing and dose interval. To secure and maintain predictable efficacy with rFVIIa, judicious dose selection and treatment timing are important, together with adjunct therapy where necessary. As inhibitor patients present with different bleeding scenarios, a tailored treatment approach should be adopted.

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