Editorial: Modern approaches to hemophilia management in children and adolescents

Editorial on the Research Topic Modern approaches to hemophilia management in children and adolescentsHemophilia A and B are the most common severe bleeding disorders which are due to partial or total deficiency of coagulation factor VIII (FVIII) or factor IX (FIX), respectively.Depending on the severity of coagulation factor deficiency, the affected individuals may experience bleeding only with severe trauma or during major surgery (mild hemophilia) to spontaneous hemarthrosis, hematomas, and intracranial hemorrhages in the absence of any identifiable trauma (severe hemophilia) (1).Until recently, coagulation factor replacement therapy via plasma-derived factor concentrates or recombinant factor products has been the mainstay of hemophilia treatment, provided either prophylactically or on-demand to treat bleeding episodes.Despite the widespread availability of safe and effective replacement therapy, many individuals with hemophilia continue to experience significant burden from their treatment, breakthrough bleeding, and debilitating arthropathy.In addition, the relatively high rate of inhibitor development to factor products can cause major morbidity and mortality, leading to a considerable burden on patients, families, and healthcare systems (2).Over the past two decades, however, there has been remarkable progress in the management of hemophilia.Cloning of the genes encoding factor VIII and factor IX, together with advances in bioengineering have led to novel therapies that offer easier modes of administration, extended half-lives, and improved patient outcomes and quality of life.In addition, gene therapy is under investigation as the next frontier in hemophilia treatment, especially for hemophilia B (3, 4).While these advances are associated with improvement of patients' outcomes, they also result in many new observations and questions that were not previously encountered.Furthermore, access to these treatments remains limited to the minority of people with hemophilia worldwide.In this Research Topic, Urasiski et al. review the Polish national experience of switching prophylaxis factor replacement therapy from standard-dose prophylaxis with a plasma-derived FVIII to recombinant FVIII in previously-treated patients (PTP), as the result of the national tender.Authors aimed to address two important questions: safety of switching from a plasmaderived product to a recombinant product, and the effectiveness of PK-tailored dosing vs. standard-dose approach.While the national tender mandated the switching of the products, children and adolescents had the choice of standard-dose or PK-tailored approach.Following the switch, no patient developed high-titer or persistent inhibitor (two patients had low-titer but transient inhibitors).Curiously, patients who chose standard-dose prophylaxis had a lower

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