An open clinical study assessing the efficacy and safety of Factor IX Grifols®, a high-purity Factor IX concentrate, in patients with severe haemophilia B

Haemophilia ◽  
2009 ◽  
Vol 16 (2) ◽  
pp. 240-246 ◽  
Author(s):  
T. LISSITCHKOV ◽  
M. MATYSIAK ◽  
K. ZAWILSKA ◽  
L. GERCHEVA ◽  
A. ANTONOV ◽  
...  
Keyword(s):  
Clinical Study ◽  
High Purity ◽  
Factor Ix ◽  
Severe Haemophilia ◽  
Efficacy And Safety ◽  
Haemophilia B

Incidence of Factor IX Inhibitor Development in Severe Haemophilia B Patients Treated with only one Brand of High Purity Plasma Derived Factor IX Concentrate

1999 ◽  
Vol 82 (10) ◽  
pp. 1247-1249 ◽  
Author(s):  
Yves Laurian ◽  
Chantal Rothschild ◽  
Robert Navarro ◽  
Claude Guérois ◽  
Valérie Gay ◽  
...  
Keyword(s):  
High Purity ◽  
Gene Deletion ◽  
Factor Ix ◽  
Cumulative Exposure ◽  
Missense Mutations ◽  
Severe Haemophilia ◽  
Inhibitor Development ◽  
Haemophilia B ◽  
Low Incidence ◽  
The Impact

SummaryFifteen previously untreated patients (Pups) with severe haemophilia B (factor IX activity ≤ 2 U/dl) only treated with one brand of plasma-derived high purity factor IX concentrate (FIX LFB) were studied. Age at first injection varied from 1 to 137 months and follow-up since this first injection from 21 to 86 months (median: 35). Cumulative exposure days (CED) were from 4 to over 100 (median: 26). Among these 15 Pups only one developed an inhibitor. Mutation analysis performed in all patients showed total gene deletion in the patient with inhibitor, partial gene deletion in another one, and missense mutations in 9 families. Mutation was not found in one patient. Actually, according to the data already published, only two patients were at high risk for inhibitor development in our population. Our study, although rather small, confirms the previously reported low incidence of inhibitors in haemophilia B. Large studies on incidence of FIX inhibitors are indeed difficult to perform, due to both the overall small number of severe haemophilia B patients and the low incidence of FIX inhibitors. Consequently, the impact of bias, such as prevalence of different types of gene defects in a given population, is major. Therefore, any study, dealing with incidence of FIX inhibitors in severe haemophilia B should report, for each patient, the type of gene defect.

Performances of an Artificial Reagent for the One-stage Factor IX Assay

1975 ◽  
Vol 33 (03) ◽  
pp. 547-552 ◽  
Author(s):  
L Meunier ◽  
J. P Allain ◽  
D Frommel
Keyword(s):  
Human Plasma ◽  
Factor Ix ◽  
Severe Haemophilia ◽  
Normal Human Plasma ◽  
Haemophilia B ◽  
One Stage ◽  
Normal Human ◽  
The One

SummaryA mixture of adsorbed normal human plasma and chicken plasma was prepared as reagent for factor IX measurement using a one-stage method. The substrate was found to be specific for factor IX. Its performances tested on samples displaying factor IX activity ranging from <l%–2,500% compared favorably with those obtained when using the plasma of severe haemophilia B patients as substrate.

Management of Haemophilia in Sweden

1976 ◽  
Vol 35 (03) ◽  
pp. 510-521 ◽  
Author(s):  
Inga Marie Nilsson
Keyword(s):  
Factor Viii ◽  
Total Population ◽  
Age Groups ◽  
Factor Ix ◽  
Haemophilia A ◽  
Severe Haemophilia ◽  
Haemophilia B ◽  
Human Fraction ◽  
Mild Haemophilia

SummaryThe incidence of living haemophiliacs in Sweden (total population 8.1 millions) is about 1:15,000 males and about 1:30,000 of the entire population. The number of haemophiliacs born in Sweden in 5-year periods between 1931-1975 (June) has remained almost unchanged. The total number of haemophilia families in Sweden is 284 (77% haemophilia A, 23% haemophilia B) with altogether 557 (436 with A and 121 with B) living haemophiliacs. Of the haemophilia A patients 40 % have severe, 18 % moderate, and 42 % mild, haemophilia. The distribution of the haemophilia B patients is about the same. Inhibitors have been demonstrated in 8% of the patients with severe haemophilia A and in 10% of those with severe haemophilia B.There are 2 main Haemophilia Centres (Stockholm, Malmo) to which haemophiliacs from the whole of Sweden are admitted for diagnosis, follow-up and treatment for severe bleedings, joint defects and surgery. Minor bleedings are treated at local hospitals in cooperation with the Haemophilia Centres. The concentrates available for treatment in haemophilia A are human fraction 1-0 (AHF-Kabi), cryoprecipitate, Antihaemophilic Factor (Hyland 4) and Kryobulin (Immuno, Wien). AHF-Kabi is the most commonly used preparation. The concentrates available for treatment in haemophilia B are Preconativ (Kabi) and Prothromplex (Immuno). Sufficient amounts of concentrates are available. In Sweden 3.2 million units of factor VIII and 1.0 million units of factor IX are given per year. Treatment is free of charge.Only 5 patients receive domiciliary treatment, but since 1958 we in Sweden have practised prophylactic treatment of boys (4–18 years old) with severe haemophilia A. At about 5-10 days interval they receive AHF in amounts sufficient to raise the AHF level to 40–50%. This regimen has reduced severe haemophilia to moderate. The joint score is identical with that found in moderate haemophilia in the same age groups. For treatment of patients with haemophilia A and haemophilia B complicated by inhibitors we have used a large dose of antigen (factor VIII or factor IX) combined with cyclophosphamide. In most cases this treatment produced satisfactory haemostasis for 5 to 30 days and prevented the secondary antibody rise.

Pharmacokinetics of a new human plasma‐derived double virus inactivated and nanofiltered factor IX concentrate in previously treated severe or moderately severe haemophilia B patients

Haemophilia ◽  
2019 ◽  
Vol 25 (6) ◽  
Author(s):  
Giancarlo Castaman ◽  
Alessandra Borchiellini ◽  
Elena Santagostino ◽  
Giuseppe Tagariello ◽  
Margit Serban ◽  
...  
Keyword(s):  
Human Plasma ◽  
Factor Ix ◽  
Severe Haemophilia ◽  
Haemophilia B ◽  
Previously Treated

Idelvion® as a modern treatment option for haemophilia B (Sponsor: CSL Behring GmbH)

2020 ◽  
Vol 12 (01) ◽  
pp. 1-20
Keyword(s):  
Fusion Protein ◽  
Safety Profile ◽  
Factor Ix ◽  
Efficacy And Safety ◽  
Haemophilia B ◽  
Previous Therapy ◽  
Injection Frequency ◽  
Recombinant Factor Ix ◽  
Modern Treatment ◽  
Long Acting

ZusammenfassungIdelvion® (albutrepenonacog alfa, rIX-FP) is a long-acting recombinant factor IX (FIX) albumin fusion protein indicated for the treatment and prophylaxis of bleeding in patients with haemophilia B. It allows prophylaxis intervals of up to 14 days.* Compared with previous therapy, this fusion protein allows for a significant reduction in injection frequency while maintaining a favourable efficacy and safety profile.

Kinetics of factor IX activity differ from that of factor IX antigen in patients with haemophilia B receiving high-purity factor IX replacement

Haemophilia ◽  
1999 ◽  
Vol 5 (3) ◽  
pp. 174-180 ◽  
Author(s):  
Liebman ◽  
Rosenwald-Zuckerman ◽  
Retzios ◽  
Yasmin ◽  
Kasper
Keyword(s):  
High Purity ◽  
Factor Ix ◽  
Haemophilia B ◽  
Kinetics Of
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