Weekly recombinant FIX prophylaxis for severe haemophilia B in normal clinical practice: data from UKHCDO and Finland

Haemophilia ◽  
2017 ◽  
Vol 23 (3) ◽  
pp. e240-e243 ◽  
Author(s):  
M. Scott ◽  
V. Nummi ◽  
R. Lassila ◽  
H. Xiang ◽  
C. R. M. Hay
Keyword(s):  
Clinical Practice ◽  
Severe Haemophilia ◽  
Haemophilia B ◽  
Normal Clinical Practice

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.

Ipsilateral total shoulder and elbow prosthetic replacement in a patient with severe haemophilia B

Haemophilia ◽  
1995 ◽  
Vol 1 (4) ◽  
pp. 270-273 ◽  
Author(s):  
A. M. PHILLIPS ◽  
W. J. RIBBANS ◽  
N. J. GODDARD
Keyword(s):  
Severe Haemophilia ◽  
Prosthetic Replacement ◽  
Haemophilia B

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

Inhibitor development in haemophilia according to concentrate

2015 ◽  
Vol 113 (05) ◽  
pp. 968-975 ◽  
Author(s):  
Riita Lassila ◽  
Flora Peyvandi ◽  
Gabriele Calizzani ◽  
Alex Gatt ◽  
Thierry Lambert ◽  
...  
Keyword(s):  
Calculated Data ◽  
Incidence Rates ◽  
Clotting Factor ◽  
Haemophilia A ◽  
Severe Haemophilia ◽  
Inhibitor Development ◽  
Haemophilia B ◽  
Number Of Patients ◽  
Recombinant Fviii ◽  
Set Up

SummaryInhibitor development represents the most serious side effect of haemophilia treatment. Any difference in risk of inhibitor formation depending on the product used might be of clinical relevance. It was this study’s objective to assess inhibitor development according to clotting factor concentrate in severe haemophilia A and B. The European Haemophilia Safety Surveillance (EUHASS) was set up as a study monitoring adverse events overall and according to concentrate. Since October 2008, inhibitors were reported at least quarterly. Number of treated patients was reported annually, specifying the number of patients completing 50 exposure days (Previously Untreated Patients, PUPs) without inhibitor development. Cumulative incidence, incidence rates and 95 % confidence intervals (CI) were calculated. Data from October 1, 2008 to December 31, 2012 were analysed for 68 centres that validated their data. Inhibitors developed in 108/417 (26 %; CI 22–30 %) PUPs with severe haemophilia A and 5/72 (7 %; CI 2–16%) PUPs with severe haemophilia B. For Previously Treated Patients (PTPs), 26 inhibitors developed in 17,667 treatment years [0.15/100 treatment years; CI 0.10–0.22) for severe haemophilia A and 1/2836 (0.04/100; (CI 0.00–0.20) for severe haemophilia B. Differences between plasma-derived and recombinant concentrates, or among the different recombinant FVIII concentrates were investigated. In conclusion, while confirming the expected rates of inhibitors in PUPs and PTPs, no class or brand related differences were observed.

scholarly journals Once‐weekly prophylactic treatment vs. on‐demand treatment with nonacog alfa in patients with moderately severe to severe haemophilia B

Haemophilia ◽  
2016 ◽  
Vol 22 (3) ◽  
pp. 381-388 ◽  
Author(s):  
K. Kavakli ◽  
L. Smith ◽  
K. Kuliczkowski ◽  
J. Korth‐Bradley ◽  
C. W. You ◽  
...  
Keyword(s):  
Prophylactic Treatment ◽  
Severe Haemophilia ◽  
Haemophilia B ◽  
On Demand

P107 Prevalence of haemarthrosis and clinical impact on the musculoskeletal system in people with haemophilia in the United Kingdom: evaluation of UKHCDO and haemtrack patient reported data

Rheumatology ◽  
2020 ◽  
Vol 59 (Supplement_2) ◽  
Author(s):  
Richard A Wilkins ◽  
Heidi J Siddle ◽  
Graham Chapman ◽  
Anthony J Redmond ◽  
Hau Xiang ◽  
...  
Keyword(s):  
United Kingdom ◽  
Ankle Joint ◽  
Health Research ◽  
Clotting Factor ◽  
Haemophilia A ◽  
Severe Haemophilia ◽  
Research Support ◽  
Haemophilia B ◽  
The United Kingdom ◽  
Joint Health

Abstract Background Haemarthrosis whereby bleeding occurs within a joint is a significant feature of haemophilia. Despite the availability of prophylaxis clotting factor concentrates in the United Kingdom haemarthrosis is associated with worsening joint health and haemarthropathy in people with severe haemophilia. The ankle joint has been identified as the most affected joint followed by the knee and elbow. Whilst annual joint bleed rates (AJBR) are commonly reported in haemophilia research, bleed rates in individual affected joints and joint health status in paediatric and adult patients is yet to be established. Methods In 2018, paediatric (&lt;18 years) and adult (≥18 years) patients with severe non-inhibitor haemophilia A and B registered with the United Kingdom Haemophilia Centres Doctors Organisation (UKHCDO) National haemophilia Database (NHD) were evaluated for haemarthrosis prevalence and associated joint health. Fully itemised Haemophilia Joint Health Scores (HJHS) were obtained from NHD records in combination with AJBR from Haemtrack (HT) compliant patients. Haemarthrosis prevalence and AJBR were reported as a total and for individual joints (ankles, knees and elbows). Results During 2018, 2233 individuals were identified; 273 reported ≥75% simultaneous HT compliance and electronic fully itemised HJHS data. The median (range) age of children is 10 (6-14) years and adults 40 (25-51) years. The joint bleed prevalence of haemophilia A and B in children is 33% and 47% respectively, and in adults 42% and 60% respectively. In children with haemophilia A (n = 80) the knee (data) was the most common site of bleeding. In haemophilia A adults (n = 157) the ankle and elbow were equally affected. In haemophilia B children (n = 17) and adults (n = 19) the elbow was the most prevalent site. Total HJHS scores in children with haemophilia A and B were 0.00 (1.00 SD) and 0.40 (0.90 SD) respectively. Total HJHS scores in adults with haemophilia A and B were 21.20 (16.80 SD) and 15.40 (15.10 SD) respectively. Mean HJHS scores itemised by joint were higher in adults compared with children. In children with haemophilia A and haemophilia B, mean (3.80) and median (4.00) scores for the ankle joint were higher than for the knee (2.90 and 1.00) and elbow joint (3.30 and 1.00). Conclusion Whilst there are limitations to this self-select subset of individuals with severe haemophilia, the prevalence of haemarthrosis is evenly distributed in all adult joints with a trend towards the knee in paediatrics. Despite prophylaxis 30% of children and 60% adults still reported bleeding over a 12-month period. Irrespective of the prevalence of joint bleeds in children with haemophilia the HJHS does not appear to be clinically sensitive enough to detect changes in joint health. Adults deemed adherent with prophylaxis and haemtrack still demonstrate worsening HJHS scores despite a low AJBR. Disclosures R.A. Wilkins Grants/research support; Funded by the National Institute for Health Research. H.J. Siddle Grants/research support; Funded by the National Institute for Health Research. G. Chapman None. A.J. Redmond None. H. Xiang None. M. Scott None. M. Richards None. L. Horn None. B. Palmer None. D. Stephensen None.

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