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November 16th, 2004

WFH Joint Symposium Review – Natural Proteins in the Treatment of Blood Disorders: Return to Reason


Invited discussant, Dr Simon Brown ( London, UK . High Titre FVIII Inhibitors in Children Treated Solely with BPL 8Y).

Dr Brown gave a brief update of inhibitor development in severe haemophilia A patients treated with the VWF-containing pdFVIII concentrate, BPL 8Y. Retrospective data are now available for 74 children thus treated from 5 UK haemophilia centres, Royal Free Hospital and Great Ormond Street Hospital, London, Oxford, Cardiff, and Birmingham Children’s Hospital. This update is confined to high titre (> 5 BU) inhibitor cases for reasons of better documentation.

In total, there were 2 (2.7 %) high titre inhibitors and 1 low titre inhibitor (reported previously) in the 74 children. 96.8 % of the children were free of an inhibitor 48 months after the first exposure to the preparation. The median age at first exposure was 12 (0.5 – 70) months and the treatment period overlooked a median duration of 78 (15 – 149) months.

This low figure for inhibitor incidence with pdFVIII-VWF compares very favourably with similar figures in the literature and contrasts with inhibitor incidence with rFVIII. It calls for a prospective study. This is now unfortunately unlikely.


© Octapharma AG, 2004