Prophylactic treatment of haemophilia patients with inhibitors: clinical experience with recombinant factor VIIa in European Haemophilia Centres

Prophylactic treatment of haemophilia patients with inhibitors: clinical experience with recombinant factor VIIa in European Haemophilia Centres

Many patients with haemophilia develop inhibitors to factor VIII and require bypassing agents to provide haemostatic cover for limb‐ or life‐threatening bleeding episodes. Due to the reduced risk of blood‐borne pathogen transmission with recombinant products, on‐demand recombinant factor VIIa (rFVII...

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Bibliographic Details
Published in:Haemophilia : the official journal of the World Federation of Hemophilia Vol. 13; no. 5; pp. 502 - 507
Main Authors: MORFINI, M., AUERSWALD, G., KOBELT, R. A., RIVOLTA, G. F., RODRIGUEZ-MARTORELL, J., SCARAGGI, F. A., ALTISENT, C., BLATNY, J., BOREL-DERLON, A., ROSSI, V.
Format: Journal Article
Language:English
Published: Oxford, UK Blackwell Publishing Ltd 01-09-2007
Subjects:
Adolescent
Adult
Child
Child, Preschool
Coagulants - administration & dosage
Europe
Factor VII - administration & dosage
Factor VIIa
Female
haemophilia
Hemarthrosis - prevention & control
Hemophilia A - prevention & control
Hemorrhage - prevention & control
Humans
inhibitors
Male
Patient Compliance
prophylaxis
recombinant
Recombinant Proteins - administration & dosage
Retrospective Studies
rFVIIa
Treatment Outcome
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Summary:Many patients with haemophilia develop inhibitors to factor VIII and require bypassing agents to provide haemostatic cover for limb‐ or life‐threatening bleeding episodes. Due to the reduced risk of blood‐borne pathogen transmission with recombinant products, on‐demand recombinant factor VIIa (rFVIIa; NovoSeven®) is the treatment of choice for children with inhibitors. In haemophiliac patients without inhibitors, primary prophylaxis has been clinical practice for several years. This paper summarises 13 case histories of rFVIIa secondary prophylaxis for haemophilia patients with inhibitors. This was a retrospective survey of adult and paediatric severe haemophilia patients with inhibitors treated with rFVIIa from ten European Haemophilia Centres. There was a wide variation in administered rFVIIa dose, from 200–250 μg kg−1 per week to 220 μg kg−1 daily. In many cases, this was lower than the recommended on‐demand dose of rFVIIa. In 12/13 cases, prophylaxis with rFVIIa considerably reduced the number of bleeding episodes compared with previous treatment. Eight/nine patients were satisfied or very satisfied with rFVIIa treatment, and in cases reporting subjective quality of life (QoL), all were improved, much improved, or significantly improved. In haemophilia patients with inhibitors, prophylaxis with rFVIIa is highly effective in reducing the number of bleeding episodes and results in good patient compliance and improved QoL. Randomised controlled trials are needed to confirm these findings. Results of a recently completed clinical trial on secondary prophylaxis with rFVIIa in frequently bleeding haemophilia patients with inhibitors are expected in late 2006.
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ISSN:1351-8216
1365-2516
DOI:10.1111/j.1365-2516.2007.01455.x