Past, Present, and Future Options in the Treatment of Hemophilia A

Patients who present with hemophilia A require treatment to replace low levels of Factor VIII (FVIII). Intervention can either be on demand or prophylactically. The goal of therapy is to prevent joint bleeds that lead to permanent damage. The progression of therapy in hemophilia A has provided patients with options resulting in a greatly improved quality of life. The newest development of gene therapy, to replace FVIII, may be the answer to cure this disease. This review provides a historical account of the treatment for hemophilia A as well as current available treatment options.

[1]  J. Dumont,et al.  Recombinant factor VIII Fc fusion protein for immune tolerance induction in patients with severe haemophilia A with inhibitors—A retrospective analysis , 2018, Haemophilia : the official journal of the World Federation of Hemophilia.

[2]  D. Perry,et al.  AAV5–Factor VIII Gene Transfer in Severe Hemophilia A , 2017, The New England journal of medicine.

[3]  O. Christophe,et al.  Emicizumab, a bispecific antibody recognizing coagulation factors IX and X: how does it actually compare to factor VIII? , 2017, Blood.

[4]  J. Schved,et al.  Recombinant human factor VIIa (rFVIIa) in hemophilia: mode of action and evidence to date , 2017, Therapeutic advances in hematology.

[5]  A. Akinc,et al.  Targeting of Antithrombin in Hemophilia A or B with RNAi Therapy , 2017, The New England journal of medicine.

[6]  K. Lieuw.  Many factor VIII products available in the treatment of hemophilia A: an embarrassment of riches? , 2017, Journal of blood medicine.

[7]  Flora Peyvandi,et al.  The past and future of haemophilia: diagnosis, treatments, and its complications , 2016, The Lancet.

[8]  V. Ramanan,et al.  A Randomized Trial of Factor VIII and Neutralizing Antibodies in Hemophilia A. , 2016, The New England journal of medicine.

[9]  M. Carr,et al.  Emerging and future therapies for hemophilia , 2015, Journal of blood medicine.

[10]  A. Cannavò,et al.  Immune tolerance induction in hemophilia , 2015 .

[11]  A. Shapiro.  Long-lasting recombinant factor VIII proteins for hemophilia A. , 2013, Hematology. American Society of Hematology. Education Program.

[12]  A. I. Vorobiev,et al.  Blood Clotting Factor VIII: From Evolution to Therapy , 2013, Acta naturae.

[13]  Z. McQuilten,et al.  Off-Label Use of Recombinant Factor VIIa in Pediatric Patients , 2012, Pediatrics.

[14]  C. Hay,et al.  The principal results of the International Immune Tolerance Study: a randomized dose comparison. , 2012, Blood.

[15]  F. Rosendaal,et al.  Analysis of low frequency bleeding data: the association of joint bleeds according to baseline FVIII activity levels , 2011, Haemophilia : the official journal of the World Federation of Hemophilia.

[16]  V. Blanchette,et al.  Prophylaxis in the haemophilia population , 2010, Haemophilia.

[17]  K. Fischer,et al.  Factor VIII requirement to maintain a target plasma level in the prophylactic treatment of severe hemophilia A: influences of variance in pharmacokinetics and treatment regimens , 2010, Journal of thrombosis and haemostasis : JTH.

[18]  P. Mannucci,et al.  Back to the future: a recent history of haemophilia treatment , 2008, Haemophilia : the official journal of the World Federation of Hemophilia.

[19]  G. Castaman.  Desmopressin for the treatment of haemophilia , 2007, Haemophilia : the official journal of the World Federation of Hemophilia.

[20]  J. Ingerslev,et al.  Tranexamic acid combined with recombinant factor VIII increases clot resistance to accelerated fibrinolysis in severe hemophilia A , 2007, Journal of thrombosis and haemostasis : JTH.

[21]  M. Coetzee.  The use of topical crushed tranexamic acid tablets to control bleeding after dental surgery and from skin ulcers in haemophilia , 2007, Haemophilia : the official journal of the World Federation of Hemophilia.

[22]  G. Lippi,et al.  Inhibitors in mild/moderate haemophilia A: An update , 2006, Thrombosis and Haemostasis.

[23]  M. Diamond,et al.  Clinical perspectives of emerging pathogens in bleeding disorders , 2006, The Lancet.

[24]  W. Hoots.  Pathogenesis of hemophilic arthropathy. , 2006, Seminars in hematology.

[25]  S. Pipe,et al.  1 Bioengineering of Coagulation Factor VIII for Improved Secretion , 2004, Pediatric Research.

[26]  P. Mannucci.  Hemophilia: treatment options in the twenty‐first century , 2003, Journal of thrombosis and haemostasis : JTH.

[27]  R. Ljung,et al.  Inhibitors in the Swedish population with severe haemophilia A and B: a 20‐year survey , 2002, Acta paediatrica.

[28]  P. Garzone,et al.  Clinical evaluation of recombinant factor IX. , 1998, Seminars in hematology.

[29]  P. Mannucci.  Desmopressin (DDAVP) in the treatment of bleeding disorders: the first 20 years. , 1997, Blood.

[30]  A. Gatherer,et al.  Sarcoma of the Larynx , 1958, The Journal of Laryngology & Otology.