Continuous infusion of recombinant factor VIII formulated with sucrose in surgery: Non-interventional, observational study in patients with severe haemophilia A

Haemophilia ◽  
2014 ◽  
Vol 21 (1) ◽  
pp. e19-e25 ◽  
Author(s):  
K. Meijer ◽  
S. Rauchensteiner ◽  
E. Santagostino ◽  
H. Platokouki ◽  
R. E. G. Schutgens ◽  
...  
Keyword(s):  
Observational Study ◽  
Factor Viii ◽  
Continuous Infusion ◽  
Recombinant Factor Viii ◽  
Haemophilia A ◽  
Severe Haemophilia

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.

Continuous infusion of extended half-life factor VIII (efmoroctocog alpha) for surgery in severe haemophilia A

Haemophilia ◽  
2018 ◽  
Vol 24 (4) ◽  
pp. e280-e283
Author(s):  
I. C. L. Kremer Hovinga ◽  
R. E. G. Schutgens ◽  
P. R. van der Valk ◽  
L. F. D. van Vulpen ◽  
E. P. Mauser-Bunschoten ◽  
...  
Keyword(s):  
Factor Viii ◽  
Continuous Infusion ◽  
Half Life ◽  
Haemophilia A ◽  
Severe Haemophilia ◽  
Life Factor

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

An observational study of sucrose-formulated recombinant factor VIII for Japanese patients with haemophilia A

2008 ◽  
Vol 100 (07) ◽  
pp. 32-37 ◽  
Author(s):  
Chiho Ikegawa ◽  
ChisatoYo koyama ◽  
Verena Haupt ◽  
Jean-Christophe Delumeau
Keyword(s):  
Observational Study ◽  
Factor Viii ◽  
De Novo ◽  
Severe Disease ◽  
Japanese Patients ◽  
Recombinant Factor Viii ◽  
Phase Iii ◽  
Haemophilia A ◽  
Postmarketing Surveillance Study ◽  
Usual Clinical Practice

SummaryThe safety and efficacy of sucrose-formulated recombinant factor VIII (rFVIII-FS; Kogenate® FS) under usual clinical practice were evaluated for 12 months in an observational, postmarketing surveillance study conducted at 214 treatment centres throughout Japan. The study included 631 patients with haemophilia A, 80% of whom had severe or moderately-severe disease (≤2% FVIII:C).Most patients (n=477;75.6%) had >100 prior exposure days (EDs), but the study also included 62 (9.8%) patients with <20 EDs who were at high risk for inhibitor development. A total of 71,240 infusions were administered during the observation (mean, 113 ± 108 per patient). Physicians rated efficacy and tolerability of rFVIII-FS as “very good” or “good” in >99% of patients. FVIII inhibitors were observed in seven patients (5 de novo; 1 persistent/fluctuating; 1 recurrent).The overall de novo inhibitor incidence was 0.8% (5/631; or 5/599 among the subgroup of patients with negative baseline titre and no known inhibitor history).De novo cases represented 3.2% (2/62) of patients with <20 EDs at enrollment (2/57 in the no inhibitor subgroup) and 0.2 % (1/477) of patients pretreated with >100 EDs (1/452 in the no inhibitor subgroup) at enrollment. The results of this large observational study demonstrate that rFVIII-FS is both safe and efficacious as used in the usual clinical setting for the treatment of Japanese patients with mild to severe haemophiliaA. This study supports the efficacy of rFVIII-FS with an incidence of inhibitor formation no greater than in a comparable European study or previous phase III clinical studies.

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