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Browsing by Author "Pierce, Glenn F."
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Item First conditional marketing authorization approval in the European Union for hemophilia "A" gene therapy(Elsevier, 2022) VandenDriessche, Thierry; Pipe, Steven W.; Pierce, Glenn F.; Kaczmarek, Radoslaw; Pediatrics, School of MedicineItem Liver Gene Therapy: Reliable and Durable?(Elsevier, 2019-11) Herzog, Roland W.; Pierce, Glenn F.; Pediatrics, School of MedicineItem Management of COVID-19-associated coagulopathy in persons with haemophilia(Wiley, 2021) Pipe, Steven W.; Kaczmarek, Radoslaw; Srivastava, Alok; Pierce, Glenn F.; Makris, Mike; Hermans, Cedric; Pediatrics, School of MedicineIntroduction: The SARS-CoV-2 coronavirus-induced infection (COVID-19) can be associated with a coagulopathy mainly responsible for pulmonary microvasculature thrombosis and systemic thromboembolic manifestations. The pathophysiology and management of the COVID-19 coagulopathy are likely more complex in patients with inherited bleeding diseases such as haemophilia. These individuals might indeed present with both bleeding and thrombotic complications and require simultaneous antithrombotic and haemostatic treatments. Objective: We propose practical guidance for the diagnosis and management of COVID-19 coagulopathy in persons with haemophilia. Results: Continuation of regular haemostatic treatment is recommended for ambulatory patients. For patients requiring hospital admission and on replacement therapy with factors VIII or IX concentrates, prophylaxis with concentrates should be intensified according to the risk of bleeding complications and associated with prophylactic doses of LMWH. For patients on nonreplacement therapy, emicizumab should be continued and possibly combined with factor VIII and prophylactic doses of LMWH depending on the risk of bleeding and thrombosis. Dose escalation of LMWH tailored to the risk of thrombosis can be employed but not supported by evidence. Conclusions: These practical recommendations are based on the current literature on COVID-19 with its impact on haemostasis, indications and modalities for thromboprophylaxis mainly in nonhaemophilic patients and how that is likely to affect persons with haemophilia in different circumstances. They will need to be tailored to each patient's clinical status and validated in future studies.Item Two gene therapies for hemophilia available: Now what?(Elsevier, 2023) Pierce, Glenn F.; Herzog, Roland W.; Pediatrics, School of Medicine