Hemophilia A and B are the most common severe bleeding disorders which are due to partial or total deficiency of coagulation factor VIII or factor IX, 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).
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. Also, 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.
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.
The goal of this Research Topic is to highlight the progress made in the past decade in the field of hemophilia in children. We aim to cover a wide range of topics, including the latest advancements in understanding hemostasis mechanisms, novel and emerging therapeutic options, and state-of-the-art approaches for monitoring children with hemophilia.
We encourage authors to submit their high-quality original basic, translational, or clinical research on this topic. Authoritative reviews are welcomed. Interesting case reports or case series covering novel methodologies, therapeutic advances, or any significant clinical observations will also be considered.
The following topics would be of special interest:
• Modern laboratory assessment of hemophilia in children in an era of extended half-life products and non-factor products
• Applications of pharmacokinetics to tailor treatment regimens in children and adolescents with hemophilia
• Novel and emerging replacement products and non-factor alternatives for hemophilia therapy
• Novel approaches in immune tolerance induction in children with inhibitors
• Real-time data on outcomes and quality of life of children and adolescents with hemophilia
• Advances in the application of curative therapies for hemophilia
• Novel approaches for the management of children and adolescents with moderate or mild hemophilia
• Diagnosis and management of hemophilia in girls
• Advances in the management of hemophilia in low- and middle-income countries (LMIC)
Hemophilia A and B are the most common severe bleeding disorders which are due to partial or total deficiency of coagulation factor VIII or factor IX, 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).
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. Also, 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.
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.
The goal of this Research Topic is to highlight the progress made in the past decade in the field of hemophilia in children. We aim to cover a wide range of topics, including the latest advancements in understanding hemostasis mechanisms, novel and emerging therapeutic options, and state-of-the-art approaches for monitoring children with hemophilia.
We encourage authors to submit their high-quality original basic, translational, or clinical research on this topic. Authoritative reviews are welcomed. Interesting case reports or case series covering novel methodologies, therapeutic advances, or any significant clinical observations will also be considered.
The following topics would be of special interest:
• Modern laboratory assessment of hemophilia in children in an era of extended half-life products and non-factor products
• Applications of pharmacokinetics to tailor treatment regimens in children and adolescents with hemophilia
• Novel and emerging replacement products and non-factor alternatives for hemophilia therapy
• Novel approaches in immune tolerance induction in children with inhibitors
• Real-time data on outcomes and quality of life of children and adolescents with hemophilia
• Advances in the application of curative therapies for hemophilia
• Novel approaches for the management of children and adolescents with moderate or mild hemophilia
• Diagnosis and management of hemophilia in girls
• Advances in the management of hemophilia in low- and middle-income countries (LMIC)
