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Hemostaseology in Pediatrics
It is a great pleasure to serve as guest editor of this “special issue pediatrics.”
“Our children are our future” and sick children need a special, individualized, and comprehensive care. The knowledge of differences between children and adults in physiological and pathophysiological pathways is necessary to guarantee the best treatment for this vulnerable group of patients.
Recent advances in diagnostics, interventional therapies, and new therapeutics licensed for the use in children need to be addressed and discussed critically.
The aim of the selection of conceptional reviews in this issue is to point out these particularities in pediatric patients and increase the awareness for new therapeutic modalities and their limitations.
Emicizumab was licensed for prophylaxis of bleeds in patients with hemophilia A (HA) with inhibitors in November 2017 by the U.S. Food and Drug Administration (FDA) and in February 2018 by the European Medicines Agency (EMA), and for HA patients without inhibitors in October 2018 by the FDA and in March 2019 by the EMA.  Despite practical guidance recommendations, clinical trials, and real-world observational studies demonstrating good efficacy and safety for bleeding prevention in HA patients with and without inhibitors, some crucial and critical questions remain still open regarding the use of emicizumab.   The first review article entitled “Emicizumab for all Pediatric Patients with Severe Hemophilia A?” by Dr. Wieland  elaborates what is already known about emicizumab and what we would like to know.
Acquired coagulation disorders are rare in children and are associated mainly with severe underlying disorders. Diagnostic and therapeutic approaches are often adapted from adults. Especially acquired von Willebrand syndrome might be an underdiagnosed disorder in childhood aggravating bleeding tendencies in critically ill children. The second review article “Acquired von Willebrand Disease (AVWD) in Children” by Dr. Sandrock-Lang and Prof. Dr. Zieger addresses diagnostic challenges regarding AVWD and underlying diseases or conditions in which AVWD should be considered and how it is managed.
Due to higher awareness, better diagnostic tests and advances in caring for critically ill children, and those with chronic diseases, the incidence of thrombotic events, especially in hospitalized children, has increased over the past decades.   New licensed drugs (direct oral anticoagulants [DOACs]) for therapy of venous thrombosis make it necessary to critically review recent clinical trials and experiences, to introduce these drugs, and to point out their interactions, limitations, and special dosing regimens in childhood. Dr. Halimeh guides us in her review “New Anticoagulants in neonates, children and adolescents” through the use of DOACs in children and their possible pitfalls.
Dr. V. Schmidt and Prof. Dr. M. Wildgruber provide in their review “Interventional Treatment Options in Children with Extracranial Vascular Malformations” a detailed overview on clinical presentation of venous, lymphatic, and arteriovenous malformations in children. They evaluated critically the principles of endovascular treatment and their similarities between adult and pediatric practice, highlighting distinct differences regarding the treatment of vascular malformations of children, including dose limitations and procedure-related side effects.
I hope that the articles of this theme issue for pediatrics may contribute to the increase in the awareness about coagulation disorders in the pediatric population and to facilitate respective therapeutic decisions.
I want to thank the authors for writing these interesting and important articles and all the reviewers for their comments to further improve the manuscripts.
Article published online:
29 April 2022
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- 15 Schmidt VF, Olivieri M, Häberle B. et al. Interventional treatment options in children with extracranial vascular malformations. Hamostaseologie 2022; 42: 133-143