Future of coagulation factor replacement therapy

Research output: Contribution to journalArticle

Abstract

Over a million patients worldwide currently suffer from hemophilia and other congenital clotting factor deficiencies. Patients affected with hemophilia A and B are treated by intravenous replacement therapy of factor VIII and factor IX, respectively. Current hemophilia treatments have favorably supported their efficacy, tolerability, and safety profiles. The onset of alloantibodies inactivating the infused coagulation factor is the main problem in hemophilia patients rendering replacement therapies ineffective; another disadvantage is the short half-life of the infused clotting factors with the need for multiple and frequent infusions to manage a bleeding episode. Now, the challenge in the management of hemophilia treatment is the prolongation of the half-life and reduction in the immunogenicity of recombinant clotting factors. The bioengineering strategies, previously applied successfully to other therapeutic proteins, encourage the current efforts to produce novel coagulation factors with more prolonged bioavailability, with increased potency and resistance to inactivation and potentially reduced immunogenicity.

Original languageEnglish
Pages (from-to)84-98
Number of pages15
JournalJournal of Thrombosis and Haemostasis
Volume11
Issue numberSUPPL.1
DOIs
Publication statusPublished - Jun 2013

Fingerprint

Blood Coagulation Factors
Hemophilia A
Half-Life
Life Support Care
Hemophilia B
Therapeutics
Isoantibodies
Bioengineering
Factor IX
Factor VIII
Biological Availability
Hemorrhage
Safety
Proteins

Keywords

  • Factor IX
  • Factor VIII
  • Fusion proteins
  • Half-life
  • Hemophilia
  • Polyethylene glycols
  • Recombinant FVIIa

ASJC Scopus subject areas

  • Hematology

Cite this

Future of coagulation factor replacement therapy. / Peyvandi, F.; Garagiola, I.; Seregni, S.

In: Journal of Thrombosis and Haemostasis, Vol. 11, No. SUPPL.1, 06.2013, p. 84-98.

Research output: Contribution to journalArticle

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