Characterisation of recombinant factor IX before and after GlycoPEGylation

2020 ◽  
Vol 588 ◽  
pp. 119654
Author(s):  
Finn Stausholm Nielsen ◽  
Anette Skammelsen Schmidt ◽  
Anne Kroll Kristensen ◽  
Anders Dybdal Nielsen ◽  
Brian Kåre Kristensen ◽  
...  
1997 ◽  
Vol 78 (01) ◽  
pp. 261-265 ◽  
Author(s):  
Gilbert C White ◽  
Aime Beebe ◽  
Brenda Nielsen

2018 ◽  
Vol 69 (7) ◽  
pp. 1911-1914
Author(s):  
Oana Viola Badulescu ◽  
Razvan Tudor ◽  
Wilhelm Friedl ◽  
Manuela Ciocoiu ◽  
Paul Dan Sirbu

Hemophilia is a hereditary coagulopathy that is largely in the attention of developing countries, not because of its low incidence, but because of the high costs involved in the treatment of the disease and its disabling consequences of the disease, if treated inappropriately. The concentrates of coagulation factors currently available for the substitution treatment of hemophilic patients have undergone additional viral purification and inactivation techniques, in order to achieve a higher infectious safety, an aspect that also implies an increase in treatment costs for these patients. Currently, the major morbidity of patients with hemophilia is represented by the disabling articular pathology, secondary to repetitive bleeding episodes developed in the articular space. Although it has been proved that the prophylactic administration of coagulation factors helps to prevent joint disease in the case of patients that were not subject to prophylaxis, the repeated bleeding in the joints induces synovitis, which is irreversible and may progress despite subsequent prophylaxis. Under these conditions, total joint arthroplasty remains the only solution to reduce both, pain and subsequent bleeding episodes of hemophilic arthropathy. Effective hemostasis is a basic condition for successful interventions in hemophilic patients. In this regard, this paper aims to highlight the effectiveness of Nonacog Alpha, a product that contains recombinant factor IX, in the management of hemostasis, in the case of a patient with type B hemophilia, with indication of total endoprosthesis of the left hip.


Blood ◽  
2001 ◽  
Vol 98 (8) ◽  
pp. 2571-2573 ◽  
Author(s):  
Alberta Azzi ◽  
Riccardo De Santis ◽  
Massimo Morfini ◽  
Krystyna Zakrzewska ◽  
Roberto Musso ◽  
...  

Abstract Recombinant factor VIII and factor IX concentrates, human-plasma–derived albumin, and samples from previously untreated patients with hemophilia were examined for the presence of TT virus (TTV) by using polymerase chain reaction testing. Blood samples from the patients were obtained prospectively before and every 3 to 6 months after therapy was begun. TTV was detected in 23.5% of the recombinant-product lots and 55.5% of the albumin lots tested. Only first-generation factor VIII recombinant concentrates stabilized with human albumin were positive for TTV, whereas all second-generation (human protein–free) concentrates were negative for the virus. In 59% of patients treated with either first- or second-generation recombinant factor concentrates, TTV infection developed at some point after the initial infusion. Infection with TTV in these patients before and after treatment did not appear to be clinically important. Thus, first-generation recombinant factor VIII concentrates may contain TTV and the source of the viral contamination may be human albumin.


2013 ◽  
pp. 33 ◽  
Author(s):  
Massimo Franchini ◽  
Frattini ◽  
Crestani ◽  
Sissa ◽  
Bonfanti

2017 ◽  
Vol 117 (03) ◽  
pp. 508-518 ◽  
Author(s):  
K.John Pasi ◽  
Kathelijn Fischer ◽  
Margaret Ragni ◽  
Beatrice Nolan ◽  
David J. Perry ◽  
...  

SummaryThe safety, efficacy, and prolonged half-life of recombinant factor IX Fc fusion protein (rFIXFc) were demonstrated in the Phase 3 B-LONG (adults/adolescents ≥12 years) and Kids B-LONG (children <12 years) studies of subjects with haemophilia B (≤2 IU/dl). Here, we report interim, long-term safety and efficacy data from B-YOND, the rFIXFc extension study. Eligible subjects who completed B-LONG or Kids B-LONG could enrol in B-YOND. There were four treatment groups: weekly prophylaxis (20–100 IU/kg every 7 days), individualised prophylaxis (100 IU/kg every 8–16 days), modified prophylaxis (further dosing personalisation to optimise prophylaxis), and episodic (ondemand) treatment. Subjects could change treatment groups at any point. Primary endpoint was inhibitor development. One hundred sixteen subjects enrolled in B-YOND. From the start of the parent studies to the B-YOND interim data cut, median duration of rFIXFc treatment was 39.5 months and 21.9 months among adults/adolescents and children, respectively; 68/93 (73.1 %) adults/adolescents and 9/23 (39.1 %) children had ≥100 cumulative rFIXFc exposure days. No inhibitors were observed. Median annualised bleeding rates (ABRs) were low in all prophylaxis regimens: weekly (≥12 years: 2.3; <6 years: 0.0; 6 to <12 years: 2.7), individualised (≥12 years: 2.3; 6 to <12 years: 2.4), and modified (≥12 years: 2.4). One or two infusions were sufficient to control 97 % (adults/adolescents) and 95 % (children) of bleeding episodes. Interim data from B-YOND are consistent with data from B-LONG and Kids B-LONG, and confirm the longterm safety of rFIXFc, absence of inhibitors, and maintenance of low ABRs with prophylactic dosing every 1 to 2 weeks.Supplementary Material to this article is available online at www.thrombosis-online.com.


2014 ◽  
Vol 168 (1) ◽  
pp. 113-123 ◽  
Author(s):  
Jerry Powell ◽  
Amy Shapiro ◽  
Margaret Ragni ◽  
Claude Negrier ◽  
Jerzy Windyga ◽  
...  

Haemophilia ◽  
2014 ◽  
Vol 20 (6) ◽  
pp. 891-897 ◽  
Author(s):  
H. V. Wilmot ◽  
J. Hogwood ◽  
E. Gray

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