Pharmacokinetics, efficacy and safety of IMMUNATE (R) Solvent/Detergent (IMMUNATE (R) S/D) in previously treated patients with severe hemophilia A: Results of a prospective, multicenter, open-label phase III study
Abstract
Background: IMMUNATE (R) Solvent/Detergent (S/D) is a plasma-derived, human factor VIII (FVIII)/von Willebrand factor (VWF) complex subjected to S/D and vapor heat treatment. Methods: This prospective clinical study evaluated the pharmacokinetics (PK) (compared to IMMUNATE (R)), efficacy and safety of IMMUNATE S/D in 56 previously treated patients with severe hemophilia A.
Subjects received IMMUNATE S/D either on-demand (47/56), as a prophylactic regimen (49/56), or both (40/56). Results: IMMUNATE and IMMUNATE S/D were equivalent with respect to the FVIII and VWF PK parameters assessed.
Bleeding episodes (623) were reported in 47/56 subjects. For 89% of episodes, subjects required only 1 infusion with a mean dose of 29.6 IU/kg and 96% of episodes had an excellent or good response.
The duration of prophylaxis ranged from 0.1 to 5.2 months. The median number of bleeds per month in subjects on prophylaxis was 0 (range 0-10).
No FVIII inhibitory antibodies were observed in 56 subjects after 2,646 treatment exposure days. No related serious adverse events were reported.
Conclusion: The introduction of S/D treatment did not alter the PK characteristics and function of VWF and FVIII molecules in IMMUNATE S/D which is effective and safe for treatment of bleeding episodes, management of surgical procedures and prophylaxis.