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

Kyumin Lee; Julia Q Chau; Yani B Suber; Anna R Sternberg; Allyson M Pishko; Lindsey A George; Vijay G Bhoj; Bhavya S Doshi; Benjamin J Samelson-Jones

doi:10.1182/blood.2023021944

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.

Authors

Kyumin Lee¹, Julia Q Chau¹, Yani B Suber¹, Anna R Sternberg¹, Allyson M Pishko², Lindsey A George^{1

3

4}, Vijay G Bhoj⁵, Bhavya S Doshi^{1

3

4}, Benjamin J Samelson-Jones^{1

3

4}

Affiliations

¹ Division of Hematology, Department of Pediatrics, Children's Hospital of Philadelphia, Philadelphia, PA.
² Division of Hematology, Department of Medicine, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA.
³ Division of Hematology, Department of Pediatrics, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA.
⁴ Raymond G. Perelman Center for Cellular and Molecular Therapeutics, Children's Hospital of Philadelphia, Philadelphia, PA.
⁵ Department of Pathology, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA.

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.

© 2024 American Society of Hematology. Published by Elsevier Inc. Licensed under Creative Commons Attribution-NonCommercial-NoDerivatives 4.0 International (CC BY-NC-ND 4.0), permitting only noncommercial, nonderivative use with attribution. All other rights reserved.

Publication types

Letter

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