scholarly journals PROTECT VIII Kids: BAY 94‐9027 (PEGylated Recombinant Factor VIII) safety and efficacy in previously treated children with severe haemophilia A

Haemophilia ◽  
2020 ◽  
Vol 26 (3) ◽  
Author(s):  
Elena Santagostino ◽  
Gili Kenet ◽  
Kathelijn Fischer ◽  
Tina Biss ◽  
Sanjay Ahuja ◽  
...  
Keyword(s):  
Factor Viii ◽  
Recombinant Factor Viii ◽  
Haemophilia A ◽  
Severe Haemophilia ◽  
Safety And Efficacy ◽  
Previously Treated

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.

Full-length sucrose-formulated recombinant factor VIII for treatment of previously untreated or minimally treated young children with severe haemophilia A

2005 ◽  
Vol 93 (03) ◽  
pp. 457-467 ◽  
Author(s):  
Joan Gill ◽  
Chantal Rothschild ◽  
Marilyn Manco-Johnson ◽  
Jeanne Lusher ◽  
Elke Kellermann ◽  
...  
Keyword(s):  
Young Children ◽  
Factor Viii ◽  
Full Length ◽  
Recombinant Factor Viii ◽  
Bleeding Complications ◽  
Adverse Event Rate ◽  
Haemophilia A ◽  
Severe Haemophilia ◽  
Safety And Efficacy ◽  
Mean Values

SummaryThe safety and efficacy of a full-length sucrose-formulated recombinant factor VIII product (rFVIII-FS; KogenateFS; Kogenate Bayer) was evaluated in previously untreated (PUPs) and minimally treated (MTP) patients with severe haemophilia A (FVIII < 2%). Patients (37 PUPs; 24 MTPs) aged 0.1–25.7 months were treated with rFVIII-FS for a cumulative of 9,141 exposure days (EDs), median 114 EDs (range 4–478), on prophylactic or on-demand therapy. Eighty-nine percent of all treated bleeding episodes were successfully treated with 1 (74%) or 2 (15%) infusions. Clinical response to first infusion for each bleeding episode was rated as ‘excellent’ in 58%, or ‘good’ in 33%, of all cases. Recombinant FVIII-FS was used in 27 surgical procedures, mainly catheter implantations, which were all conducted without bleeding complications. FVIII recovery mean values (~2%/kg/ IU) were as expected for any licensed FVIII concentrate. FVIII neutralizing antibody formation was 15% (9/60). Aside from inhibitor formation, three adverse events were rated as ‘at least possibly drug-related’ for a total drug-related adverse event rate of 0.14%. No viral seroconversions were observed. Overall, excellent safety and efficacy were demonstrated with rFVIII-FS for therapy of young children with severe haemophilia A.

scholarly journals Long‐term safety and efficacy of recombinant factor VIII Fc fusion protein (rFVIIIFc) in subjects with haemophilia A

Haemophilia ◽  
2015 ◽  
Vol 22 (1) ◽  
pp. 72-80 ◽  
Author(s):  
B. Nolan ◽  
J. Mahlangu ◽  
D. Perry ◽  
G. Young ◽  
R. Liesner ◽  
...  
Keyword(s):  
Fusion Protein ◽  
Factor Viii ◽  
Recombinant Factor Viii ◽  
Haemophilia A ◽  
Safety And Efficacy ◽  
Fc Fusion Protein
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