Subscribe

Subscribe to our Newsletter and get informed about new publication regulary and special discounts for subscribers!

IJPPE > Volume 11 > A Look at the Future Hemophilia A Treatment
< Back to Volume

A Look at the Future Hemophilia A Treatment

Full Text PDF

Abstract:

The current treatment of patients with hemophilia A is safer and more effective than the previous one. Prophylactic substitution involves repeated intravenous administration of plasma-derived factor VIII or recombinant factor VIII products, with inconveniences and possible adverse effects. The occurrence of inhibitors requires the administration of activated prothrombin complex concentrate or activated factor VII - an expensive treatment. The immune tolerance induction is the ideal treatment for patients with high titres of inhibitors - the only potential way to eliminate inhibitors and very expensive. For these reasons, the medical world is interested in the advances that scientific research is doing in the field of new molecules without the inconveniences of current substitution therapy and which could replace it in the future. The purpose of this article is to briefly review the new therapeutic possibilities for patients with hemophilia A, which can prevent potential extraarticular bleedings, avoid the occurrence of inhibitors, and have as few adverse effects as possible.

Info:

Periodical:
International Journal of Pharmacology, Phytochemistry and Ethnomedicine (Volume 11)
Pages:
1-6
Citation:
R. G. Mihăilă, "A Look at the Future Hemophilia A Treatment", International Journal of Pharmacology, Phytochemistry and Ethnomedicine, Vol. 11, pp. 1-6, 2018
Online since:
October 2018
Export:
Distribution:
References:

[1] P.M. Mannucci, M.E. Mancuso, M. Franchini, Tailoring hemostatic therapies to lower inhibitor development in previously untreated patients with severe hemophilia A, J. Thromb. Haemost. 14 (2016)1330-1336.

DOI: https://doi.org/10.1111/jth.13356

[2] J. Hartmann J, S.E. Croteau, 2017 Clinical trials update: Innovations in hemophilia therapy, Am. J. Hematol. 91 (2016)1252-1260.

DOI: https://doi.org/10.1002/ajh.24543

[3] S.L. Meeks, G. Batsuli, Hemophilia and inhibitors: current treatment options and potential new therapeutic approaches, Hematology Am. Soc. Hematol. Educ. Program. 2016 (2016) 657-662.

DOI: https://doi.org/10.1182/asheducation-2016.1.657

[4] M. Shima, D. Lillicrap, R. Kruse-Jarres, Alternative therapies for the management of inhibitors, Haemophilia. 22(S5) (2016) 36-41.

[5] P.E. Monahan, Emerging genetic and pharmacologic therapies for controlling hemostasis: beyond recombinant clotting factors, Hematology Am. Soc. Hematol. Educ. Program. 2015 (2015) 33-40.

DOI: https://doi.org/10.1182/asheducation-2015.1.33

[6] N. Machin, M.V. Ragni, An investigational RNAi therapeutic targeting antithrombin for the treatment of hemophilia A and B, J. Blood Med. 9 (2018) 135-140.

DOI: https://doi.org/10.2147/jbm.s159297

[7] K.P. Pratt, Engineering less immunogenic and antigenic FVIII proteins, Cell Immunol. 301 (2016) 12-17.

[8] G. Young, J.N. Mahlangu, Extended half-life clotting factor concentrates: results from published clinical trials, Haemophilia. 22(S5) (2016) 25-30.

DOI: https://doi.org/10.1111/hae.13028

[9] P.M. van Helden et al., Maintenance and break of immune tolerance against human factor VIII in a new transgenic hemophilic mouse model, Blood. 118 (2011) 3698-3707.

DOI: https://doi.org/10.1182/blood-2010-11-316521

[10] S.S. Dhadwar et al., Repeated oral administration of chitosan/DNA nanoparticles delivers functional FVIII with the absence of antibodies in hemophilia A mice, J. Thromb. Haemost. 8 (2010) 2743-2750.

DOI: https://doi.org/10.1111/j.1538-7836.2010.04116.x

[11] S. Mathieu S et al., Challenges of the management of severe hemophilia A with inhibitors: two case reports emphasizing the potential interest of a high-purity human Factor VIII/von Willebrand factor concentrate and individually tailored prophylaxis guided by thrombin-generation test, Blood Coagul. Fibrinolysis. 26 (2015).

DOI: https://doi.org/10.1097/mbc.0000000000000300

[12] M.D. Tarantino et al., Recombinant porcine sequence factor VIII (rpFVIII) for acquired haemophilia A: practical clinical experience of its use in seven patients, Haemophilia. 23 (2017) 25-32.

DOI: https://doi.org/10.1111/hae.13040

[13] C. Hart, S. Schmid, Coagulation disorders in the intensive care unit - what is new?, Dtsch. Med. Wochenschr. 141 (2016) 777-780.

[14] J. Mahlangu et al., Efficacy and safety of rVIII-SingleChain: results of a phase 1/3 multicenter clinical trial in severe hemophilia A, Blood. 128 (2016) 630-637.

[15] J.A. Abrantes et al., Elucidation of Factor VIII Activity Pharmacokinetics: A Pooled Population Analysis in Patients With Hemophilia A Treated With Moroctocog Alfa, Clin. Pharmacol. Ther. 102 (2017) 977-988.

DOI: https://doi.org/10.1002/cpt.716

[16] D. Lillicrap et al., Porcine recombinant factor VIII (Obizur; OBI-1; BAX801): product characteristics and preclinical profile. Haemophilia, 2015. Information on: https://www.ncbi.nlm.nih.gov/pubmed/26278557. Accessed: 12.04.(2018).

DOI: https://doi.org/10.1111/hae.12784

[17] K.A. Shetty et al., Factor VIII associated with lipidic nanoparticles retains efficacy in the presence of anti-factor VIII antibodies in hemophilia A mice, Biopharm. Drug Dispos. 37 (2016) 409-420.

DOI: https://doi.org/10.1002/bdd.2023

[18] M. Franchini, P.M. Mannucci, Efficacy and safety of a recombinant factor VIII produced from a human cell line (simoctocog alfa), Expert Opin. Drug Saf. 16 (2017) 405-410.

DOI: https://doi.org/10.1080/14740338.2017.1285281

[19] J. Yoon et al., FVIII-specific human chimeric antigen receptor T-regulatory cells suppress T- and B-cell responses to FVIII, Blood. 129 (2017) 238-245.

DOI: https://doi.org/10.1182/blood-2016-07-727834

[20] J. Salas et al., Enhanced Pharmacokinetics of Factor VIIa as a Monomeric Fc Fusion, Thromb. Res. 135 (2015) 970-976.

[21] K.M. Hansson et al., Effects of recombinant human prothrombin on thrombin generation in plasma from patients with hemophilia A and B, J. Thromb. Haemost. 13 (2015)1293-1300.

DOI: https://doi.org/10.1111/jth.12997

[22] V. Bhat et al., Improved coagulation and haemostasis in haemophilia with inhibitors by combinations of superFactor Va and Factor VIIa, Thromb. Haemost. 115 (2016) 551-561.

DOI: https://doi.org/10.1160/th15-07-0525

[23] L.A. George et al., Correction of human hemophilia A whole blood abnormalities with a novel bypass agent: zymogen-like FXa(I16L), J. Thromb. Haemost. 13 (2015) 1694-1698.

DOI: https://doi.org/10.1111/jth.13059

[24] J.Y. Chang et al., Studies on the mechanism of action of the aptamer BAX499, an inhibitor of tissue factor pathway inhibitor, Thromb. Res. 130(3) (2012) e151-157.

[25] P. Margaritis. Gene-based continuous expression of FVIIa for the treatment of haemophilia, Front. Biosci. (Schol Ed). 4 (2012) 287-299.

DOI: https://doi.org/10.2741/s269

[26] K.E. Brummel-Ziedins et al., Activated protein C inhibitor for correction of thrombin generation in hemophilia A blood and plasma, J. Thromb. Haemost. 9 (2011) 2262-2267.

[27] C.E. McCarthy et al., Updated analysis: central venous access device infection rates in an expanded cohort of paediatric patients with severe haemophilia receiving prophylactic recombinant tissue plasminogen activator, Haemophilia. 22 (2016).

DOI: https://doi.org/10.1111/hae.12772
Show More Hide
Cited By:
This article has no citations.