Literature DB >> 23610614

Factor VIII inhibitors in hemophilia A: rationale and latest evidence.

Char Witmer1, Guy Young.   

Abstract

Factor VIII (FVIII) replacement therapy is the foundation of treatment in hemophilia A and is effective unless a patient develops an alloantibody (inhibitor) against exogenous FVIII. Inhibitor development is currently the most significant treatment complication seen in patients with hemophilia and is associated with considerable morbidity and a decreased quality of life. The development of an inhibitor is the result of a complex interaction between a patient's immune system and genetic and environmental risk factors. The mainstay of treatment is the eradication of the inhibitor through immune tolerance. This review summarizes the current evidence regarding inhibitor risk factors, eradication, and hemostatic bypassing agents.

Entities:  

Keywords:  FVIII; FVIII alloantibodies; hemophilia A; immune tolerance; inhibitors; risk factors

Year:  2013        PMID： 23610614      PMCID： PMC3629762          DOI： 10.1177/2040620712464509

Source DB:  PubMed          Journal:  Ther Adv Hematol        ISSN： 2040-6207


  117 in total

Review 1.  Immune tolerance induction for patients with severe hemophilia A: a critical literature review.

Authors:  Massimo Franchini; Giuseppe Lippi
Journal:  J Thromb Thrombolysis       Date:  2011-11       Impact factor: 2.300

2.  Successful treatment of high titer inhibitors in mild hemophilia A with avoidance of factor VIII and immunosuppressive therapy.

Authors:  D Robbins; R Kulkarni; R Gera; A B Scott-Emuakpor; K Bosma; J A Penner
Journal:  Am J Hematol       Date:  2001-11       Impact factor: 10.047

3.  Factor VIII inhibitors in mild and moderate-severity haemophilia A. UK Haemophilia Centre Directors Organisation.

Authors:  C R Hay; C A Ludlam; B T Colvin; F G Hill; F E Preston; N Wasseem; R Bagnall; I R Peake; E Berntorp; E P Mauser Bunschoten; K Fijnvandraat; C K Kasper; G White; E Santagostino
Journal:  Thromb Haemost       Date:  1998-04       Impact factor: 5.249

4.  Activated prothrombin concentrate for patients with factor VIII inhibitors.

Authors:  E M Kurczynski; J A Penner
Journal:  N Engl J Med       Date:  1974-07-25       Impact factor: 91.245

5.  A randomized comparison of bypassing agents in hemophilia complicated by an inhibitor: the FEIBA NovoSeven Comparative (FENOC) Study.

Authors:  Jan Astermark; Sharyne M Donfield; Donna M DiMichele; Alessandro Gringeri; Steven A Gilbert; Jennifer Waters; Erik Berntorp
Journal:  Blood       Date:  2006-09-21       Impact factor: 22.113

Review 6.  Incidence of inhibitors in haemophilia A patients--a review of recent studies of recombinant and plasma-derived factor VIII concentrates.

Authors:  I Scharrer; G L Bray; O Neutzling
Journal:  Haemophilia       Date:  1999-05       Impact factor: 4.287

7.  Anti-inhibitor coagulant complex prophylaxis in hemophilia with inhibitors.

Authors:  Cindy Leissinger; Alessandro Gringeri; Bülent Antmen; Erik Berntorp; Chiara Biasoli; Shannon Carpenter; Paolo Cortesi; Hyejin Jo; Kaan Kavakli; Riitta Lassila; Massimo Morfini; Claude Négrier; Angiola Rocino; Wolfgang Schramm; Margit Serban; Marusia Valentina Uscatescu; Jerzy Windyga; Bülent Zülfikar; Lorenzo Mantovani
Journal:  N Engl J Med       Date:  2011-11-03       Impact factor: 91.245

8.  HLA genotype of patients with severe haemophilia A due to intron 22 inversion with and without inhibitors of factor VIII.

Authors:  J Oldenburg; J K Picard; R Schwaab; H H Brackmann; E G Tuddenham; E Simpson
Journal:  Thromb Haemost       Date:  1997-02       Impact factor: 5.249

9.  Polymorphisms in the IL10 but not in the IL1beta and IL4 genes are associated with inhibitor development in patients with hemophilia A.

Authors:  Jan Astermark; Johannes Oldenburg; Anna Pavlova; Erik Berntorp; Ann-Kari Lefvert
Journal:  Blood       Date:  2005-12-27       Impact factor: 22.113

10.  Induction of immune tolerance in patients with hemophilia and antibodies to factor VIII by combined treatment with intravenous IgG, cyclophosphamide, and factor VIII.

Authors:  I M Nilsson; E Berntorp; O Zettervall
Journal:  N Engl J Med       Date:  1988-04-14       Impact factor: 91.245
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  50 in total

1.  von Willebrand factor binds to the surface of dendritic cells and modulates peptide presentation of factor VIII.

Authors:  Nicoletta Sorvillo; Robin B Hartholt; Esther Bloem; Magdalena Sedek; Anja ten Brinke; Carmen van der Zwaan; Floris P van Alphen; Alexander B Meijer; Jan Voorberg
Journal:  Haematologica       Date:  2015-12-03       Impact factor: 9.941

Review 2.  Approaches to Mitigate the Unwanted Immunogenicity of Therapeutic Proteins during Drug Development.

Authors:  Laura I Salazar-Fontana; Dharmesh D Desai; Tarik A Khan; Renuka C Pillutla; Sandra Prior; Radha Ramakrishnan; Jennifer Schneider; Alexandra Joseph
Journal:  AAPS J       Date:  2017-01-12       Impact factor: 4.009

Review 3.  Alloantibodies to therapeutic factor VIII in hemophilia A: the role of von Willebrand factor in regulating factor VIII immunogenicity.

Authors:  Johannes Oldenburg; Sébastien Lacroix-Desmazes; David Lillicrap
Journal:  Haematologica       Date:  2015-02       Impact factor: 9.941

4.  Implementing emicizumab in hemophilia inhibitor management: emicizumab should be prescribed after tolerance.

Authors:  Guy Young
Journal:  Blood Adv       Date:  2018-10-23

Review 5.  Experience of Immune Tolerance Induction Therapy for Hemophilia A Patients with Inhibitors from a Single Center in India.

Authors:  Tulika Seth
Journal:  Indian J Hematol Blood Transfus       Date:  2019-11-04       Impact factor: 0.900

Review 6.  Low Dose Prophylaxis in Hemophilia Care.

Authors:  Neeraj Sidharthan; Remya Sudevan
Journal:  Indian J Hematol Blood Transfus       Date:  2019-06-15       Impact factor: 0.900

Review 7.  A Molecular Revolution in the Treatment of Hemophilia.

Authors:  John S S Butterfield; Kerry M Hege; Roland W Herzog; Radoslaw Kaczmarek
Journal:  Mol Ther       Date:  2019-11-13       Impact factor: 11.454

Review 8.  Next generation antibody drugs: pursuit of the 'high-hanging fruit'.

Authors:  Paul J Carter; Greg A Lazar
Journal:  Nat Rev Drug Discov       Date:  2017-12-01       Impact factor: 84.694

9.  Long-term safety and efficacy of emicizumab in a phase 1/2 study in patients with hemophilia A with or without inhibitors.

Authors:  Midori Shima; Hideji Hanabusa; Masashi Taki; Tadashi Matsushita; Tetsuji Sato; Katsuyuki Fukutake; Ryu Kasai; Koichiro Yoneyama; Hiroki Yoshida; Keiji Nogami
Journal:  Blood Adv       Date:  2017-09-27

10.  FVIII activity following FVIII protein infusion or FVIII gene transfer predicts the bleeding risk in hemophilia A rats.

Authors:  Karin M Lövgren; Malte S Larsen; Shannon M Zintner; Juliana C Small; Mads Kjelgaard-Hansen; Mattias Häger; Maj Petersen; Bo Wiinberg; Paris Margaritis
Journal:  J Thromb Haemost       Date:  2020-04-16       Impact factor: 5.824
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