Recombinant clotting factors: a review of current clinical status

In the short time span since the cloning of the human genes for factors VIII, IX and VII, recombinant factor (rF)VIII, rFIX and rFVIIa concentrates have been produced and standardised, have entered clinical trials, and have been licensed for clinical use in the US, Europe and elsewhere. Even more recently, a new generation of recombinant clotting factor concentrates has emerged, including rFVIII concentrates formulated with sucrose rather than human serum albumin and a B-domain-deleted rFVIII preparation. Additionally, rFVIII concentrates which contain no human or animal proteins in their manufacture are in development, as is a combination rFVIII plus recombinant von Willebrand factor concentrate. The clinical experience in previously treated patients with each of the existing recombinant products has documented that inhibitor development is not increased with any of them. As expected, there has been no reported instance of disease transmission with any of the recombinant clotting factor concentrates. Since their introduction, usage of each of the recombinant clotting factor concentrates has dramatically increased in countries where these products are licensed, available and affordable.