Special Issue "Haemophilia: Current Treatment and Clinical Outcomes, Challenges and Opportunities"
Deadline for manuscript submissions: 31 December 2023 | Viewed by 3350
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
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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 in order 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 hemarthrosis, which progresses towards an inevitable haemophilic arthropathy, yet numerous unanswered issues remain that concern new drugs. Although EHL products are promising, the optimal strategy for the treatment of bleeds between prophylactic doses and dosing regimens will likely have to be individualized according to patient pharmacokinetics, accounting for age and physical activity. Emicizumab appears to be able to improve haemostasis in haemophilia patients, probably including those with inhibitors; however, they do not currently appear to be able to prevent all bleeding. What is the best treatment to reduce the risk of severe bleeding for newborns, such as intracranial haemorrhage? Could emicizumab and EHL equally protect joints from developing haemophilic arthropathy? What is the best product for active patients? There is much more to cover. This Special Issue, “Haemophilia: Current Treatment and Challenges”, aims to provide an overview of current and developing drugs for haemophilia treatments, endeavouring to give some answers to the many questions and concerns that exist.
Prof. Dr. Ezio Zanon
Manuscript Submission Information
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- haemophilia A and B
- FVIII EHL, FIX EHL
- health-related quality of life
- population pharmacokinetics