The nonactivated
prothrombin complex concentrate (PCC)
Octaplex (Octapharma PPGmbH, Vienna, Austria) has been used successfully for the treatment of congenital and acquired
coagulation factor deficiencies and associated
bleeding. The aims of this study were to assess retrospectively whether
Octaplex is an effective treatment option for
haemophilia A patients with high-titre inhibitors of
factor VIII (FVIII) and to investigate the impact of
Octaplex on
thrombin generation in vitro and ex vivo. Retrospective data were collected from 15
haemophilia A patients with FVIII inhibitors who had been treated with
Octaplex. Mild bleeds were treated for a median of 1 day with a median dose of 77 IU/kg and moderate bleeds for 3 days with 57 IU/kg. The physician's overall satisfaction with
Octaplex, taking into account efficacy, safety and cost in comparison with other treatment options, was assessed for each bleed. The overall rating was good, very good or excellent for 29 of 41 (71%) bleeds. No
adverse drug reactions were reported. In in-vitro studies of
thrombin generation with normal plasma samples, experimental inhibition of FVIII activity prolonged the lag phase, diminished the peak
thrombin concentration and decreased the area under the concentration-time curve, as expected. Marked improvement in
thrombin generation parameters was achieved by adding 0.5-3 IU
factor IX/ml PCC into the samples. The same held true when using plasma samples from
haemophilia A patients with FVIII inhibitors. These results demonstrate that
Octaplex overcomes inhibition of FVIII in in-vitro and ex-vivo assays of
thrombin generation, and that
Octaplex is an effective treatment option for
haemophilia A patients with FVIII inhibitors.