Coagulation factor concentrates: past, present, and future.
Clotting factor transfusions are vital for people with diseases such as haemophilia. In the 1970s and 1980s, transfusions with pooled plasma led to a devastatingly high number of recipients becoming infected with blood-borne pathogens such as HIV and hepatitis C. This epidemic triggered the development of virus-free factor concentrates through a combination of improved donor selection and screening, effective virucidal technologies, and recombinant protein expression biotechnology. There is now a wide range of recombinant factor concentrates, and an impressive safety record with respect to pathogen transmission. However, remaining therapeutic challenges include the potential threat of transmission of prions and other pathogens, the formation of inhibitory alloantibodies, and the international disparity that exists in product availability due to differences in licensure status as well as prohibitively high costs. In the future, it is likely that bioengineered recombinant proteins that have been modified to enhance pharmacokinetic properties or reduce immunogenicity, or both, will be used increasingly in clinical practice.
Key
Nigel S
NS
University of North Carolina School of Medicine and Harold R Roberts Comprehensive Hemophilia Diagnostic and Treatment Center, Chapel Hill, NC, USA. Nigel_Key@med.unc.edu
Negrier
Claude
C
D025861:Blood Coagulation Disorders, Inherited / Q000628:therapy*; D001779:Blood Coagulation Factors / Q000502:physiology / Q000627:therapeutic use*; D006801:Humans; D016032:Randomized Controlled Trials as Topic
D001779:Blood Coagulation Factors