Recombinant factor VIII products and inhibitor development in previously untreated patients with severe haemophilia A: Combined analysis of three studies

Haemophilia ◽  
2019 ◽  
Vol 25 (3) ◽  
pp. 398-407 ◽  
Author(s):  
Peter Volkers ◽  
Kay‐Martin Hanschmann ◽  
Thierry Calvez ◽  
Hervé Chambost ◽  
Peter W. Collins ◽  
...  
Keyword(s):  
Factor Viii ◽  
Recombinant Factor Viii ◽  
Haemophilia A ◽  
Severe Haemophilia ◽  
Combined Analysis ◽  
Inhibitor Development

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

2008 ◽  
Vol 99 (01) ◽  
pp. 52-58 ◽  
Author(s):  
Elena Santagostino ◽  
Albert Faradji ◽  
Alfonso Iorio ◽  
Jan van der Meer ◽  
Jørgen Ingerslev ◽  
...  
Keyword(s):  
Observational Study ◽  
Factor Viii ◽  
De Novo ◽  
Observation Time ◽  
Full Length ◽  
Recombinant Factor Viii ◽  
Phase Iii ◽  
Haemophilia A ◽  
Severe Haemophilia ◽  
Safety And Efficacy

SummaryThe 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.

scholarly journals SHP656, a polysialylated recombinant factor VIII (PSA‐rFVIII): First‐in‐human study evaluating safety, tolerability and pharmacokinetics in patients with severe haemophilia A

Haemophilia ◽  
2019 ◽  
Vol 26 (1) ◽  
pp. 47-55 ◽  
Author(s):  
Andreas Tiede ◽  
Geoffrey Allen ◽  
Alexander Bauer ◽  
Pratima Chowdary ◽  
Peter Collins ◽  
...  
Keyword(s):  
Factor Viii ◽  
Human Study ◽  
Recombinant Factor Viii ◽  
Haemophilia A ◽  
Severe Haemophilia

scholarly journals Efficacy and inhibitor development in previously treated patients with haemophilia A switched to a B domain-deleted recombinant factor VIII

2004 ◽  
Vol 126 (3) ◽  
pp. 398-404 ◽  
Author(s):  
Alessandro Gringeri ◽  
Annarita Tagliaferri ◽  
Giuseppe Tagariello ◽  
Massimo Morfini ◽  
Elena Santagostino ◽  
...  
Keyword(s):  
Factor Viii ◽  
Recombinant Factor Viii ◽  
Haemophilia A ◽  
Inhibitor Development ◽  
Previously Treated Patients ◽  
Previously Treated

scholarly journals Early factor VIII exposure and subsequent inhibitor development in children with severe haemophilia A

Haemophilia ◽  
2007 ◽  
Vol 13 (2) ◽  
pp. 149-155 ◽  
Author(s):  
E. A. CHALMERS ◽  
S. A. BROWN ◽  
D. KEELING ◽  
R. LIESNER ◽  
M. RICHARDS ◽  
...  
Keyword(s):  
Factor Viii ◽  
Haemophilia A ◽  
Severe Haemophilia ◽  
Inhibitor Development
Sign in / Sign up

Export Citation Format

Share Document