The new albumin-free recombinant factor VIII concentrates for treatment of hemophilia: do they represent an actual incremental improvement?
The goal of eliminating the low levels of infectious disease risk from hemophilia treatment has resulted in the development of multiple generations of recombinant factor VIII (rFVIII) products. The ideal product should be devoid of human and animal proteins, which may transmit infectious agents. These products should also maintain molecular integrity, hemostatic efficacy, similar immunogenicity, and acceptable side effect profiles as compared to plasma-derived factor VIII. Currently available first-, second-, and third-generation rFVIII products include Recombinate; Kogenate FS/Helixate FS and ReFacto; and Advate, respectively. During the evolution of rFVIII products, either full-length or B-domain-deleted factor VIII were transfected into immortalized cell lines. The B-domain-deleted product, ReFacto, has resulted in an additional method to monitor factor VIII levels. The third-generation products offer the theoretical advantage of being produced without human and/or animal proteins. Upon initial introduction into the marketplace, the newer products have a higher cost. However, when analyzing historical trends, the prices of these products are almost equivalent to first-generation products within 3 years of licensure. Thus, the initial cost of the product may be a minimal issue in the medical decision process when selecting rFVIII replacement therapy.
Josephson
Cassandra D
CD
Children's Healthcare of Atlanta Blood Banks, Atlanta, GA, USA. cjoseph@emory.edu
Abshire
Thomas
T
D000293:Adolescent; D000328:Adult; D000368:Aged; D000418:Albumins; D000818:Animals; D001780:Blood Coagulation Tests; D017848:Blood-Borne Pathogens; D016466:CHO Cells; D002460:Cell Line; D002461:Cell Line, Transformed; D002648:Child; D002675:Child, Preschool; D002986:Clinical Trials as Topic; D006224:Cricetinae; D003412:Cricetulus; D004340:Drug Contamination; D005169:Factor VIII / Q000191:economics / Q000235:genetics / Q000302:isolation & purification / Q000592:standards / Q000627:therapeutic use*; D006467:Hemophilia A / Q000188:drug therapy*; D006801:Humans; D007223:Infant; D017053:Infection Control / Q000379:methods; D008297:Male; D008647:Mesocricetus; D008875:Middle Aged; D015337:Multicenter Studies as Topic; D011994:Recombinant Proteins / Q000737:chemistry / Q000191:economics / Q000302:isolation & purification / Q000592:standards / Q000627:therapeutic use; D014162:Transfection; D016896:Treatment Outcome
D000418:Albumins; D011994:Recombinant Proteins; C078147:F8 protein, human; D005169:Factor VIII