The More Recent History of Hemophilia Treatment

Semin Thromb Hemost. 2022 Nov;48(8):904-910. doi: 10.1055/s-0042-1756188. Epub 2022 Sep 15.

Abstract

The availability first in the 1970s of plasma-derived and then in the 1990s of recombinant clotting factor concentrates represented a milestone in hemophilia care, enabling not only treatment of episodic bleeding events but also implementation of prophylactic regimens. The treatment of hemophilia has recently reached new landmarks. The traditional clotting factor replacement therapy for hemophilia has been substituted over the last 10 years by novel treatments such as bioengineered factor VIII and IX molecules with extended half-life and non-factor treatments including the bispecific antibody emicizumab. This narrative review is dedicated to these newer therapies, which are contributing significantly to improving the long-term management of prophylaxis in hemophilia patients. Another section is focused on the current state of gene therapy, which is a promising definitive cure for severe hemophilia A and B.

Publication types

  • Review

MeSH terms

  • Blood Coagulation Factors / therapeutic use
  • Factor IX / therapeutic use
  • Factor VIII / therapeutic use
  • Genetic Therapy
  • Half-Life
  • Hemophilia A* / drug therapy
  • Hemophilia B* / drug therapy
  • Hemostatics* / therapeutic use
  • Humans

Substances

  • Factor VIII
  • Blood Coagulation Factors
  • Hemostatics
  • Factor IX