Enhanced procoagulant activity of select hemophilia B causing factor IX variants with emicizumab

Blood. 2024 Sep 12;144(11):1230-1235. doi: 10.1182/blood.2023021944.

Abstract

Emicizumab improves the procoagulant activity of select loss-of-function factor IX (FIX) variants with likely dysfunctional assembly of the intrinsic Xase complex, resulting in hemophilia B (HB). FVIII mimetics may represent an alternative nonfactor therapy for select patients with HB.

Publication types

  • Letter
  • Research Support, N.I.H., Extramural

MeSH terms

  • Antibodies, Bispecific* / pharmacology
  • Antibodies, Bispecific* / therapeutic use
  • Antibodies, Monoclonal, Humanized* / pharmacology
  • Antibodies, Monoclonal, Humanized* / therapeutic use
  • Blood Coagulation / drug effects
  • Factor IX* / genetics
  • Hemophilia B* / blood
  • Hemophilia B* / drug therapy
  • Hemophilia B* / genetics
  • Humans

Substances

  • Factor IX
  • emicizumab
  • Antibodies, Bispecific
  • Antibodies, Monoclonal, Humanized