Results from a large multinational clinical trial (guardian™1) using prophylactic treatment with turoctocog alfa in adolescent and adult patients with severe haemophilia A: safety and efficacy

Haemophilia ◽  
2013 ◽  
Vol 19 (5) ◽  
pp. 691-697 ◽  
Author(s):  
S. R. Lentz ◽  
M. Misgav ◽  
M. Ozelo ◽  
S. Z. Šalek ◽  
D. Veljkovic ◽  
...  
Keyword(s):  
Clinical Trial ◽  
Prophylactic Treatment ◽  
Adult Patients ◽  
Haemophilia A ◽  
Severe Haemophilia ◽  
Safety And Efficacy ◽  
Multinational Clinical Trial

Ultrasound evaluation of joint damage and disease activity in adult patients with severe haemophilia A using the HEAD‐US system

Haemophilia ◽  
2021 ◽  
Author(s):  
Víctor Jiménez‐Yuste ◽  
Hortensia de la Corte‐Rodríguez ◽  
María Teresa Álvarez‐Román ◽  
Mónica Martín‐Salces ◽  
Felipe Querol ◽  
...  
Keyword(s):  
Disease Activity ◽  
Joint Damage ◽  
Adult Patients ◽  
Haemophilia A ◽  
Severe Haemophilia ◽  
Ultrasound Evaluation

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.

Sign in / Sign up

Export Citation Format

Share Document