Treating haemophilia A with recombinant blood factors: a comparison.

Expert Opin Pharmacother

Department of Biochemistry, J. Holland Laboratory for Biomedical Sciences, American Red Cross, Rockville, MD 20855, USA.

Published: May 2004

The mainstay in the treatment of haemophilia A is replacement therapy with repeated infusions of plasma-derived Factor VIII (FVIII) concentrates or recombinant FVIII products. While modern plasma-derived FVIII concentrates have an excellent safety profile, there is an inexorable shift towards the use of recombinant products, especially in affluent countries. Recombinant FVIII products have demonstrated excellent haemostatic efficacy and higher safety with regard to the transmission of blood-borne pathogens. The experience in haemophilia A treatment with five currently available recombinant FVIII products, including the first third-generation product, Advate, which is completely free from human or animal proteins, is summarised. Some unresolved problems concerning the efficacy and assaying of recombinant factors and future perspectives of both recombinant and plasma-derived FVIII products in global haemophilia care, are also discussed.

Download full-text PDF

Source
http://dx.doi.org/10.1517/14656566.5.5.1061DOI Listing

Publication Analysis

Top Keywords

fviii products
16
recombinant fviii
12
fviii concentrates
8
plasma-derived fviii
8
recombinant
7
fviii
6
products
5
treating haemophilia
4
haemophilia recombinant
4
recombinant blood
4

Similar Publications

Efficient production of recombinant human FVII in CHO cells using the piggyBac transposon system.

Protein Expr Purif

January 2025

Paul C. Lauterbur Research Center for Biomedical Imaging, Institute of Biomedical and Health Engineering, Shenzhen Institutes of Advanced Technology, Chinese Academy of Sciences, Shenzhen, P.R. China, 518055; Shenzhen Weiguang Biological Products Co., Ltd., Shenzhen, P.R. China, 518107. Electronic address:

As an important coagulation factor, activated coagulation factor VII (FVIIa) is mainly used to treat the bleeding of hemophilia patients who have developed inhibitory antibodies against FVIII and FIX conventional treatment. Recombinant human factor VII (rhFVII) produced in mammalian cell lines have been developed as the most important resource of FVIIa. However, cell lines express rhFVII protein derived from an exogenous expression vector at a lower level than most other proteins.

View Article and Find Full Text PDF

Purpose: Prophylaxis with recombinant factor VIII (rFVIII) products is the gold-standard treatment for hemophilia A. However, interindividual differences affect the half-life and clearance of rFVIII products. The myPKFiT is a web-based medical-device software program for population pharmacokinetic (PK) simulation of FVIII products to guide accurate FVIII doses and dosing intervals.

View Article and Find Full Text PDF

Congenital hemophilia A (HA) disease severity has traditionally been categorized according to intrinsic factor (F)VIII levels, with <1% of normal indicating severe HA, 1% to 5% moderate HA, and 6% to 40% mild HA. However, mounting evidence illustrates considerable variability in bleeding phenotype regardless of FVIII level. Despite treatment advances, people with moderate or mild HA may be neglected, as treatment guidelines and established norms focus on FVIII levels, and many clinical trials do not include people with FVIII > 1%.

View Article and Find Full Text PDF

Hemophilia A (HA) is an inherited condition that is characterized by a lack of coagulation factor VIII (FVIII), which is needed for blood clotting. To produce recombinant factor VIII (rFVIII) for treatment, innovative methods are required. This study presents a thorough examination of the genetic engineering and biotechnological methods that are essential for the production of this complex process.

View Article and Find Full Text PDF

Background: Hemophilia A is caused by coagulation factor VIII (FVIII) deficiency and increases bleeding risk during invasive procedures.

Objectives: To investigate FVIII concentrate use and bleeding outcomes for invasive procedures after valoctocogene roxaparvovec gene transfer.

Design: This manuscript presents post hoc analysis of the phase III GENEr8-1 trial.

View Article and Find Full Text PDF

Want AI Summaries of new PubMed Abstracts delivered to your In-box?

Enter search terms and have AI summaries delivered each week - change queries or unsubscribe any time!