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Infant and toddlers with Severe Hemophilia A with Inhibitor on Prophylaxis with Emicizumab
  • Jessica Garcia,
  • Ayesha Zia
Jessica Garcia
UT Southwestern
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Ayesha Zia
UT Southwestern
Author Profile

Abstract

Emicizumab is a recombinant, humanized, and bispecific monoclonal antibody that bridges activated factor (F) IX and FX in place of FVIII to restore hemostasis in persons with hemophilia A (PHA). Data on the efficacy and safety of emicizumab in young children is limited. Immunologic naivety, physiologically decreased production of vitamin K dependent proteins, specifically FIX, and enhanced clearance of emicizumab in infants may support decreased emicizumab effectiveness. We report on the facilitation of care rendered by using emicizumab in young PHA with inhibitors and extend data on the efficacy and safety in PHA < 3 years.

Peer review status:ACCEPTED

30 Nov 2020Submitted to Pediatric Blood & Cancer
30 Nov 2020Submission Checks Completed
30 Nov 2020Assigned to Editor
03 Dec 2020Reviewer(s) Assigned
16 Dec 2020Review(s) Completed, Editorial Evaluation Pending
23 Dec 2020Editorial Decision: Revise Minor
12 Jan 20211st Revision Received
12 Jan 2021Submission Checks Completed
12 Jan 2021Assigned to Editor
18 Jan 2021Reviewer(s) Assigned
19 Jan 2021Review(s) Completed, Editorial Evaluation Pending
19 Jan 2021Editorial Decision: Accept