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http://dx.doi.org/10.1111/hae.14959 | DOI Listing |
BMJ Open
December 2024
Department of Paediatrics, Nara Medical University, Kashihara, Japan.
Introduction: Emicizumab prophylaxis is approved for people of all ages with haemophilia A (HA) including infants and children. Although previous studies have demonstrated the efficacy and tolerability of emicizumab in infants with HA, real-world data on emicizumab use in infants are limited. The Haemophilia A in Infancy and NewbOrns: multi-instituional prospective observational study to assess the efficacy anD safety of Emicizumab (HINODE) study aims to evaluate the coagulation potential and safety of emicizumab prophylaxis in infants with congenital HA from birth to <12 months of age.
View Article and Find Full Text PDFHaemophilia
May 2024
Division of Hematology/Oncology, Department of Pediatrics, Baylor College of Medicine, Texas Children's Cancer and Hematology Center, Houston, Texas, USA.
Haemophilia
September 2021
Children's Hospital Los Angeles, Los Angeles, California, USA.
Introduction: Emicizumab has been shown to be safe and effective for prevention of bleeds in patients with severe haemophilia A (SHA), both with and without inhibitors. The subcutaneous administration and long half-life make emicizumab an attractive option for prophylaxis in infants with SHA, however data to inform treatment decisions in this younger age group are almost absent.
Aim: The aim of this report is to share real world experience to illustrate how the availability of emicizumab has shifted the prophylaxis paradigm in the management of infants with SHA.
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