Haemophilia: Current Treatment and Clinical Outcomes, Challenges and Opportunities: 2nd Edition
A special issue of Journal of Clinical Medicine (ISSN 2077-0383). This special issue belongs to the section "Hematology".
Deadline for manuscript submissions: 31 July 2025 | Viewed by 1
Special Issue Editor
Interests: hemophilia A and B; Von Willebrand disease; rare coagulation disorders; antipholipid antibodies (lupus anticoagulant, anticardiolipin antibodies); deep vein thrombosis and pulmonary embolism; venous thromboembolism; arterial thrombosis
Special Issues, Collections and Topics in MDPI journals
Special Issue Information
Dear Colleagues,
We invite you to contribute to the Special Issue entitled “Haemophilia: Current Treatment and Clinical Outcomes, Challenges and Opportunities: 2nd Edition”. This is the Special Issue’s second volume; we published nine papers in the first volume. For more details, please visit https://www.mdpi.com/journal/jcm/special_issues/D7TL8NGGX0.
In recent years, several new drug treatments have been developed for treating haemophilia patients. Recombinant clotting factor concentrates FVIII/FIX extended half-life, and new haemostasis agents administered subcutaneously (weekly to monthly) are available. These innovative approaches have the potential to enhance the standard of care by decreasing infusion frequency to increase compliance, promote prophylaxis, offer alternatives to inhibitor patients, and ease the administration route.
Prophylaxis is the gold-standard treatment in haemophilia to prevent bleeding and recurrent haemarthrosis, which can progress to an haemophilic arthropathy, yet numerous issues remain with regard to new drugs. Although EHL products are promising, the optimal strategy for the treatment of bleeds between prophylactic doses and dosing regimens needs to be individualized according to patient pharmacokinetics, accounting for age and physical activity. Emicizumab appears able to improve haemostasis in haemophilia patients, probably including those with inhibitors; however, they cannot currently prevent all bleeding. This context poses the following questions: What is the best treatment for reducing the risk of severe bleeding in newborns, such as intracranial haemorrhage? Could emicizumab and EHL equally protect joints from developing haemophilic arthropathy? What is the best product for active patients? This Special Issue, entitled “Haemophilia: Current Treatment and Clinical Outcomes, Challenges and Opportunities”, will provide an overview of current and developing drugs for haemophilia treatments, providing some answers to the many questions and concerns that exist.
Prof. Dr. Ezio Zanon
Guest Editor
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Keywords
- haemophilia A and B
- FVIII EHL, FIX EHL
- prophylaxis
- emicizumab
- health-related quality of life
- population pharmacokinetics
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