A survey on clinical practice in monitoring and management of bleeding in children with haemophilia A on emicizumab prophylaxis in the PedNet centres
Susanna Ranta a), m); Ester Zapotocka b); Nadine G. Andersson c), n); Kathelijn Fischer d), f); Gili Kenet e); Marloes de Kovel f); Christoph Königs g); Veerle Labarque h), i); Christoph Male j); Martin Olivieri k); Jayashree Motwani l)
a) Astrid Lindgren Children’s Hospital, Karolinska University Hospital, Stockholm, Sweden
b) Department of Pediatric Hematology and Oncology, University Hospital Motol and Second Faculty of Medicine, Charles University, Prague, Czech Republic
c) Center for Thrombosis and Haemostasis, Skåne University Hospital, Malmö, Sweden
d) Center for Benign Haematology, Thrombosis and Haemostasis Van Creveldkliniek, University Medical Center Utrecht, Utrecht, the Netherlands
e) National Hemophilia Center, Sheba, Tel Hashomer & the Amalia Biron Thrombosis Research Institute, Tel Aviv University, Israel
f) PedNet Haemophilia Research Foundation, Baarn, the Netherlands
g) Goethe University, University Hospital Frankfurt, Department of Paediatrics and Adolescent Medicine, Frankfurt, Germany
h) Department of Pediatric Hemato-Oncology, University Hospitals Leuven, Leuven, Belgium
i) Centre of Molecular and Vascular Biology, KU Leuven, Leuven, Belgium
j) Department of Paediatrics, Medical University of Vienna, Vienna, Austria
k) Pediatric Thrombosis and Hemostasis Unit, Pediatric Hemophilia Center, Dr. von Hauner Children’s Hospital, LMU Munich, Munich, Germany
l) Department of Haematology, Centre Director for Haemophilia Comprehensive Care Centre, Birmingham Children’s Hospital, UK
m) Childhood Cancer Research Unit, Department of Women’s and Children’s Health, Karolinska Institutet, Stockholm, Sweden
n) Department of Clinical Sciences and Paediatrics, Lund University, Lund, Sweden
Highlights
• Practices regarding emicizumab vary among pediatric haemophilia centres.
• Optimal approach to achieve/maintain FVIII tolerance under emicizumab is unclear.
• ITI under emicizumab is often offered to patients with high-titer FVIII inhibitors.