Real-world case series and summary of current literature of infants and toddlers with severe hemophilia A with inhibitor on prophylaxis with emicizumab

Jessica Garcia; Ayesha Zia

doi:10.1002/pbc.28942

Real-world case series and summary of current literature of infants and toddlers with severe hemophilia A with inhibitor on prophylaxis with emicizumab

Pediatr Blood Cancer. 2021 May;68(5):e28942. doi: 10.1002/pbc.28942. Epub 2021 Feb 8.

Authors

Jessica Garcia^{1

2}, Ayesha Zia^{1

2}

Affiliations

¹ UT Southwestern, Dallas, Texas, USA.
² Children's Medical Center, Dallas, Texas, USA.

PMID: 33559300
DOI: 10.1002/pbc.28942

Abstract

Emicizumab is a recombinant, humanized, and a 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.

Keywords: emicizumab; hemophilia A; infant; inhibitor; toddler.

Publication types

Case Reports
Review

MeSH terms

Antibodies, Bispecific / therapeutic use*
Antibodies, Monoclonal, Humanized / therapeutic use*
Hemophilia A / drug therapy*
Humans
Infant
Infant, Newborn
Male

Substances

Antibodies, Bispecific
Antibodies, Monoclonal, Humanized
emicizumab