Clinical observation on safety and efficacy of a plasma- and albumin-free recombinant factor VIII for on-demand treatment of Chinese patients with haemophilia A

Summary. Recombinant FVIII (rFVIII) has become the best choice for treating bleeding of haemophilia A patients. A plasma- and albumin-free recombinant FVIII (rAHF-PFM, ADVATE®), as the third generation rFVIII, virtually eliminates the risk of blood-borne disease transmission by excluding all human blood derived additives throughout cell culture, purification and formulation. In this multicentre prospective clinical study we evaluated the efficacy, safety and immunogenicity of ADVATE® in Chinese patients with haemophilia A. Fifty-eight patients enrolled and received ADVATE® treatment. Of the patients enrolled, eight (13.79%) had severe haemophilia, 45 (77.59%) had moderate haemophilia and five (8.62%) had mild haemophilia. Fifty-four patients completed 6 months of observation. A total of 781 bleeds occurred in these 58 subjects, all evaluable per-protocol. A total of 984 infusions were administered with a mean of 17.0 ± 11.1 infusions per patient. On average, each patient received a mean of 15030.2 ± 7972.7 IU ADVATE® (median 13 625 IU, range 9500–19 750 IU) during 6 months. The majority of bleeding episodes (95.9%) were successfully treated with one or two infusions of ADVATE®. Overall, response to the first ADVATE® treatment was rated as either ‘excellent’ (82.8%) or ‘improved’ (17.2%) in all subjects. All patients tolerated ADVATE® infusions well. One patient (1/58, 1.7%) developed an inhibitor of 4 Betheseda units at day 180 visit. The results of this clinical observational study support that ADVATE® is efficacious, safe and well tolerated in the treatment of Chinese patients with haemophilia A.