:: Volume 9, Issue 1 (Int J Mol Cell Med 2020) ::
Int J Mol Cell Med 2020, 9(1): 33-49 Back to browse issues page
Factor VIII: Perspectives on Immunogenicity and Tolerogenic Strategies for Hemophilia A Patients
Sheyda Khalilian1 , Majid Motovali-Bashi 2, Halimeh Rezaei1
1- Genetics Division, Department of Cell and Molecular Biology and Microbiology, Faculty of Biological Science and Technology, University of Isfahan, Iran.
2- Genetics Division, Department of Cell and Molecular Biology and Microbiology, Faculty of Biological Science and Technology, University of Isfahan, Iran. , mbashi@sci.ui.ac.ir
Abstract:   (1128 Views)
A major complication in treating hemophilia A is the development of neutralizing antibodies (inhibitors) against therapeutic administered factor VIII (FVIII), which occurs in approximately 20-30% of patients with severe disease. These inhibitors render FVIII replacement therapy ineffective and increase the morbidity and mortality risk. The currently accepted method to eradicate inhibitors is immune tolerance induction (ITI), and frequent intensive administration of FVIII until inhibitor titers drop. Current ITI protocols are extremely costly and not effective in all patients. During the last decade, many types of research have been accomplished to clarify the mechanisms that mediate immune tolerance induction. Novel experimental therapies including monoclonal antibodies, viral vector-mediated gene therapy, regulatory T cell induction using immunosuppressive drugs, and nanoparticle-based immune modulation show promising results in hemophilia A clinical trials. This review focuses on treatment options towards the anti-FVIII immune responses and current novel therapies in clinical trials.
Keywords: Factor VIII, hemophilia A, inhibitors, immune tolerance
Full-Text [PDF 802 kb]   (209 Downloads)    
Type of Study: Review | Subject: Molecular & Cellular Immunology
Received: 2020/02/22 | Accepted: 2020/05/1 | Published: 2020/04/29



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Volume 9, Issue 1 (Int J Mol Cell Med 2020) Back to browse issues page