Thromb Haemost 2008; 99(01): 52-58
DOI: 10.1160/TH07-06-0409
Blood Coagulation, Fibrinolysis and Cellular Haemostasis
Schattauer GmbH

Safety and efficacy of sucrose-formulated full-length recombinant factor VIII: Experience in the standard clinical setting

Roberto Musso
1   Azienda Ospedale Vittorio Emanuele, Ospedale Ferrarotto, Catania, Italy
,
Elena Santagostino
2   A. Bianchi Bonomi Haemophilia and Thrombosis Centre, IRCCS Maggiore Hospital Foundation, Milan, Italy
,
Albert Faradji
3   Haemophilia Regional Centre, Hôpital de Hautepierre, Strasbourg, France
,
Alfonso Iorio
4   Division of Internal and Cardiovascular Medicine and Stroke Unit, University of Perugia, Italy
,
Jan van der Meer
5   Division of Haemostasis, Thrombosis and Rheology, University Medical Centre Groningen (UMCG), Groningen, the Netherlands
,
Jørgen Ingerslev
6   Centre for Haemophilia and Thrombosis, Department of Clinical Biochemistry, Aarhus University Hospital, Skejby, Denmark
,
Thierry Lambert
7   APHP Bicêtre University Hospital, Le Kremlin-Bicêtre, France
,
Monika Maas-Enriquez
8   Bayer Health- Care, Leverkusen, Germany
,
Eduard Gorina
9   Bayer HealthCare, Berkeley, California, USA
,
for the KOGENATE® Bayer European PMS Study Group › Author Affiliations
Further Information

Publication History

Received: 13 June 2007

Accepted after major revision: 28 October 2007

Publication Date:
24 November 2017 (online)

Summary

The safety of full-length sucrose-formulated recombinant factor VIII (rFVIII-FS; KOGENATE® FS) for up to 24 months of use was evaluated in a postmarketing observational study in Europe. Long-term safety and efficacy data were available for 212 patients with severe haemophilia A, including 13 previously untreated patients (PUPs) and 12 patients with 1–19 exposure days (EDs). Patients accumulated a mean (± SD) of 187 (121) EDs to rFVIII-FS and received a total of 39,627 infusions, mainly for prophylaxis and for the treatment of 4,283 spontaneous or trauma-related bleeds during an average observation time of 710 (136) days. Of these bleeding episodes, 85.4% were successfully treated with one or two infusions of rFVIII-FS. Haemostasis was also evaluated during 46 minor to major surgical pro- cedures, and the response to infusion was “excellent” or “good” in all cases. FVIII inhibitor formation was observed in six patients (two de novo; four persistent or recurrent). The de novo cases represent 8.0% (2 of 25) of patients who reported 0–19 previous EDs at study entry. Four of the five patients who reported possible drug-related adverse effects developed inhibitors. The results of this observational study demonstrate the efficacy and safety of rFVIII-FS during normal clinical use in the treatment of patients with severe haemophilia A. Furthermore, these findings are consistent with those of previous phase III clinical studies with rFVIII-FS, particularly with regard to its efficacy and low incidence of inhibitor formation.

 
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