Enhanced procoagulant activity of select hemophilia B causing factor IX variants with emicizumab-Reference-Cited by-同舟云学术

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

Published:2024-09-12 Issue:11 Volume:144 Page:1230-1235
ISSN:0006-4971
Container-title:Blood
language:en
Short-container-title:

Author:

Lee Kyumin¹,Chau Julia Q.¹,Suber Yani B.¹^ORCID,Sternberg Anna R.¹^ORCID,Pishko Allyson M.²,George Lindsey A.¹³⁴^ORCID,Bhoj Vijay G.⁵,Doshi Bhavya S.¹³⁴^ORCID,Samelson-Jones Benjamin J.¹³⁴^ORCID

Affiliation:

1. 1Division of Hematology, Department of Pediatrics, Children’s Hospital of Philadelphia, Philadelphia, PA

2. 2Division of Hematology, Department of Medicine, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA

3. 3Division of Hematology, Department of Pediatrics, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA

4. 4Raymond G. Perelman Center for Cellular and Molecular Therapeutics, Children's Hospital of Philadelphia, Philadelphia, PA

5. 5Department of Pathology, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA

Abstract

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.

Publisher

American Society of Hematology

Link

https://ashpublications.org/blood/article-pdf/144/11/1230/2241564/blood_bld-2023-021944-main.pdf

Reference25 articles.

1. Activation of factor VIII and mechanisms of cofactor action;Fay;Blood Rev,2004

2. A bispecific antibody to factors IXa and X restores factor VIII hemostatic activity in a hemophilia A model;Kitazawa;Nat Med,2012

3. Emicizumab prophylaxis in patients who have hemophilia A without inhibitors;Mahlangu;N Engl J Med,2018