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

摘要: Many patients with haemophilia develop inhibitors to factor VIII and require bypassing agents provide haemostatic cover for limb- or life-threatening bleeding episodes. Due the reduced risk of blood-borne pathogen transmission recombinant products, on-demand VIIa (rFVIIa; NovoSeven is treatment choice children inhibitors. In haemophiliac without inhibitors, primary prophylaxis has been clinical practice several years. This paper summarises 13 case histories rFVIIa secondary was a retrospective survey adult paediatric severe treated from ten European Haemophilia Centres. There wide variation in administered dose, 200-250 microg kg(-1) per week 220 daily. many cases, this lower than recommended dose rFVIIa. 12/13 considerably number episodes compared previous treatment. Eight/nine were satisfied very treatment, cases reporting subjective quality life (QoL), all improved, much significantly improved. highly effective reducing results good patient compliance improved QoL. Randomised controlled trials are needed confirm these findings. Results recently completed trial on frequently expected late 2006.