Recombinant FVIII appears to be cost effective for severe haemophilia A in the USA

2021 ◽  
Vol 883 (1) ◽  
pp. 26-26
Keyword(s):  
Cost Effective ◽  
Haemophilia A ◽  
Severe Haemophilia ◽  
The Usa ◽  
Recombinant Fviii

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.

Absence of Inhibitors in Previously Untreated Patients with Severe Haemophilia A after Exposure to a Single Intermediate Purity Factor VIII Product

1997 ◽  
Vol 78 (03) ◽  
pp. 1027-1029 ◽  
Author(s):  
T T Yee ◽  
M D Williams ◽  
F G H Hill ◽  
C A Lee ◽  
K J Pasi
Keyword(s):  
Factor Viii ◽  
High Purity ◽  
Specific Activity ◽  
Haemophilia A ◽  
Severe Haemophilia ◽  
True Incidence ◽  
Conventional Fractionation ◽  
Heat Treated ◽  
Recombinant Fviii ◽  
Von Willebrand

SummaryUse of high purity and recombinant factor VIII (FVIII) concentrates has been thought to be associated with an increased incidence of FVIII inhibitors in patients with severe haemophilia A. Comparison with comparable historical control groups has suggested that the true incidence of inhibitors in patients with severe haemophilia A was ~20-25%, similar to the incidence seen with new high purity and recombinant FVIII products.We have conducted a study of inhibitor development in a cohort of 37 boys with severe haemophilia A (VIII: C <2 u/dl) exposed only to a single FVIII concentrate (BPL 8Y) with no previous blood or blood product exposure. This factor VIII concentrate is an intermediate purity product with a specific activity of ~2 IU/mg protein and contains well preserved von Willebrand factor multimers. It is manufactured by conventional fractionation technologies and terminally dry heat treated at 80° C for 72 h.

Case Report on a Female Patient with Congenital Haemophilia a and An Acquired Type-2 Inhibitor to FVIII

Blood ◽  
2008 ◽  
Vol 112 (11) ◽  
pp. 4525-4525
Author(s):  
Karin Kurnik ◽  
Christoph Bidlingmaier
Keyword(s):  
Half Life ◽  
Female Child ◽  
Haemophilia A ◽  
Severe Haemophilia ◽  
Hormonal Change ◽  
Fviii Inhibitor ◽  
Intron 22 Inversion ◽  
Depth Analysis ◽  
Recombinant Fviii

Abstract Only rare cases of female haemophilia have been reported. Moreover, no case of congenital haemophilia A associated with an acquired type-2 inhibitor to FVIII in a female child has been described in literature yet. We report the case of a 15-year old girl, diagnosed with severe haemophilia A (FVIII:C &lt;1%) at the age of 13 months. Her father suffered from severe haemophilia A (FVIII:C &lt;1%) due to an intron 22 inversion. In the girl both an intron 22 inversion and a non-random X-inactivation were found. At 3 years the girl received prophylactic treatment, and recombinant FVIII (3 × 40 IU/kg/week) from 6 years of age. Bleeding episodes were rare. Aged 13 she developed two large spontaneous haematomas. In-depth analysis confirmed decreased FVIII-recovery, –half-life and an inhibitor titre of 7.6 BU following type-2 kinetics (haemophilic autoantibodies). All of the few reported children with an aquired FVIII inhibitor presented with other autoimmune diseases or had used penicillin. Our patient showed none of this, but interestingly experienced menarche 4 weeks after inhibitor detection. This lets us speculate that the ethiology of the inhibitor formation in our patient at the time of hormonal change might be similar to that in pregnant or postpartum women. Taking into account the recommendations of Ma et al. (2006) regarding inhibitor development during pregnancy in healthy women, we initiated a modified ITI with 75 IU/kg rFVIII concentrate (4,000 IU per infusion) every other day without additional immunosuppressive therapy. After 6 weeks of ITI, inhibitor titres declined and became negative after 8 months, resulting in a normal FVIII half-life till now.

scholarly journals Experience of switching to NovoEight: views of people with haemophilia

2020 ◽  
Vol 7 (1) ◽  
pp. 70-77 ◽  
Author(s):  
Debra Pollard ◽  
Kate Khair ◽  
Mike Holland
Keyword(s):  
New Products ◽  
Cost Effective ◽  
New Product ◽  
Self Management ◽  
Haemophilia A ◽  
Severe Haemophilia ◽  
Prophylactic Regimen ◽  
Facilitators And Barriers ◽  
New Treatment ◽  
The Uk

AbstractManagement of haemophilia A requires administration of factor VIII therapy which, for those with severe haemophilia A in the UK, is predominantly self-administered at home in a prophylactic regimen to prevent or minimise bleeding. The UK undertakes a national tendering process every three years to ensure access to current and new therapies at a cost-effective price, through contracting for large volumes from individual suppliers. This means that some products may no longer be available and that new products can enter the UK market at any tendering stage.In the latest tendering round, in 2018, more than one product was withdrawn from the UK market and a new product (NovoEight®; Novo Nordisk) was added to the prescribing list. This meant people with haemophilia having to change products. The experience of 77 people with haemophilia or their carers who changed treatment products during this process was captured by questionnaires administered by haemophilia nurses from 12 treatment centres. Overall, although people with haemophilia felt that they had little influence in decision making about changing to NovoEight, they were confident with their new treatment including packaging and accessories for administration. This was seen more in those who switched from plasma-derived products where ease of infusion was rated highly. Users’ views of haemophilia treatment should be collected at times of change to identify facilitators and barriers experienced in self-management

First report on the safety and efficacy of an extended half-life glycoPEGylated recombinant FVIII for major surgery in severe haemophilia A

Haemophilia ◽  
2017 ◽  
Vol 23 (5) ◽  
pp. 689-696 ◽  
Author(s):  
K. Hampton ◽  
P. Chowdary ◽  
S. Dunkley ◽  
S. Ehrenforth ◽  
L. Jacobsen ◽  
...  
Keyword(s):  
Half Life ◽  
Major Surgery ◽  
Haemophilia A ◽  
Severe Haemophilia ◽  
Safety And Efficacy ◽  
First Report ◽  
Recombinant Fviii

scholarly journals Body Mass Index Best Predicts Recovery of Recombinant Factor VIII in Underweight to Obese Patients with Severe Haemophilia A

2019 ◽  
Vol 120 (02) ◽  
pp. 277-288
Author(s):  
Andreas Tiede ◽  
Ana Rosa Cid ◽  
Georg Goldmann ◽  
Victor Jiménez-Yuste ◽  
Michael Pluta ◽  
...  
Keyword(s):  
Body Mass Index ◽  
Body Composition ◽  
Haemophilia A ◽  
Activity Levels ◽  
Obese Patients ◽  
Severe Haemophilia ◽  
Obese Class ◽  
Fviii Activity ◽  
Recombinant Fviii ◽  
Similar Peak

Abstract Background Factor VIII (FVIII) products are usually dosed according to body weight (BW). This may lead to under- or over-dosing in underweight or obese patients, respectively. Objective This article evaluates the pharmacokinetics (PK) of recombinant FVIII concentrate, particularly recovery, in relation to body mass index (BMI) and other body composition descriptors. Materials and Methods Thirty-five previously treated adults with severe haemophilia A from five BMI categories (underweight, normal, overweight, obese class I and II/III) were included. PK was evaluated after 50 IU per kilogram of BW single-dose recombinant FVIII (turoctocog alfa). The body composition variable was based on measurements of weight, height, bioimpedance analysis, and dual-energy X-ray absorptiometry. A dosing model was derived to achieve similar peak FVIII activity levels across BMI categories. Results A statistically significant positive association between BMI and C30min, IR30min, and AUC0–inf was observed; CL and Vss showed a significant negative association with BMI; t½ was independent of BMI and other parameters. The dosing model introduced a correction factor ‘M’ for each BMI category, based on linear regression analysis of C30min against BMI, which ranged from 0.55 for underweight to 0.39 for obese class II/III. This model achieved similar peak FVIII activity levels across BMI categories, estimating an average dose adjustment of +243.3 IU (underweight) to –1,489.6 IU (obese class II/III) to achieve similar C30min. Conclusion BMI appears to be the best predictor of recombinant FVIII recovery; however, PK endpoints were also dependent on other body composition variables. The model demonstrated that dosing can be adjusted for individual BMI to achieve better FVIII predictability across BMI categories.

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