Treatment of Hemophilia in the Near Future

Flora Peyvandi; Isabella Garagiola

doi:10.1055/s-0034-1543998

Seminars in Thrombosis and Hemostasis, Table of Contents

Semin Thromb Hemost 2015; 41(08): 838-848
DOI: 10.1055/s-0034-1543998

Thieme Medical Publishers 333 Seventh Avenue, New York, NY 10001, USA.

Treatment of Hemophilia in the Near Future

Flora Peyvandi

¹Angelo Bianchi Bonomi Haemophilia and Thrombosis Centre, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Department of Pathophysiology and Transplantation, University of Milan, Luigi Villa Foundation, Milan, Italy

,

Isabella Garagiola

¹Angelo Bianchi Bonomi Haemophilia and Thrombosis Centre, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Department of Pathophysiology and Transplantation, University of Milan, Luigi Villa Foundation, Milan, Italy

› Author Affiliations

Abstract

Advancements and debacles have characterized hemophilia treatment over the past 50 years. The 1970s saw the availability of plasma-derived concentrates making prophylaxis and home therapy possible. This optimistic perception changed extremely in the early 1980s, when most people with hemophilia were infected with HIV and hepatitis viruses. Then, also in the 1980s, the rapid progress in molecular biology led to the development of recombinant therapeutic products. This important advancement was a huge technological leap fresh off from the earlier 1980s disaster. Now in the 21st century, the newer bioengineering drugs open a new hopeful phase for the management of hemophilia. The current efforts are concentrated on producing novel coagulation factors with prolonged bioavailability, increased potency, and resistance to inactivation and potentially reduced immunogenicity; this phase of evolution is improving very quickly. 2014 is the year of marketing approval by the Food and Drug Administration of the first bioengineered FVIII and FIX long-acting drugs, using Fc-fusion strategy. This represents the first significant advance in the hemophilia therapy that dramatically transforms patient management by substantially reducing the frequency of injections, improving compliance, and simplifying prophylaxis and, in turn, refining the quality of life of hemophilia patients, offering them a nearly normal life expectancy, particularly to newborns with hemophilia B.

Keywords

PEGylation - Fc- and albumin-fusion - aptamers - therapeutic antibody - RNA interference

Full Text

References

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