Emicizumab Promotes Factor Xa Generation on Endothelial Cells
CONCLUSIONS: Emicizumab promotes FXa generation on the surface of endothelial cells, which is markedly enhanced in the presence of FEIBA®. These findings demonstrate a potential mechanism for the thrombotic complications seen with the combined use of emicizumab and FEIBA®.PMID:38460838 | DOI:10.1016/j.jtha.2024.02.017 (Source: Thrombosis and Haemostasis)
Source: Thrombosis and Haemostasis - March 9, 2024 Category: Hematology Authors: Ammon M Fager Patrick Ellsworth Nigel S Key Dougald M Monroe Maureane Hoffman Source Type: research
Using reference equations to standardise incremental shuttle walk test performance in children and young people with chronic conditions and facilitate the evaluation of exercise capacity and disease severity
Conclusions
ISWT performance may be useful in a clinic to identify those with worsening disease, but only when performance is standardised against a healthy reference population. The development of validated global reference equations is necessary for more robust assessment. (Source: BMJ Open)
Source: BMJ Open - March 8, 2024 Category: General Medicine Authors: Filipow, N., Bladen, M., Raywood, E., Robinson, E., Chugh, D., Douglas, H., Thorpe, N., O'Connor, R., Murray, N., Main, E. Tags: Open access, Paediatrics Source Type: research
Next-generation strategies to improve safety and efficacy of adeno-associated virus-based gene therapy for hemophilia: lessons from clinical trials in other gene therapies
Haematologica. 2024 Mar 7. doi: 10.3324/haematol.2023.284622. Online ahead of print.ABSTRACTThree major directions for the global progress of adeno-associated virus (AAV) vectors for gene therapies (GT) are analyzed: a) engineering vectors to increase transgene expression; b) aligning interests of the health system with costs and challenges for pharmaceutical industry; c) refining patient eligibility criteria, and endpoints definition. Currently employed AAV vectors may cause toxicity and adverse events. Furthermore, studies in animals do not fully predict risks and clinical benefits of AAV-based GT, and animal models refl...
Source: Haematologica - March 7, 2024 Category: Hematology Authors: Giovanni Di Minno Wolfgang Miesbach Giancarlo Castaman Flora Peyvandi Source Type: research
Hemophilia and hepatology, back to the future
Rev Esp Enferm Dig. 2024 Mar 7;116. doi: 10.17235/reed.2024.10105/2023. Online ahead of print.ABSTRACTno abstract.PMID:38450508 | DOI:10.17235/reed.2024.10105/2023 (Source: Revista Espanola de Enfermedades Digestivas)
Source: Revista Espanola de Enfermedades Digestivas - March 7, 2024 Category: Gastroenterology Authors: Antonio Olveira V íctor Jiménez Source Type: research
Next-generation strategies to improve safety and efficacy of adeno-associated virus-based gene therapy for hemophilia: lessons from clinical trials in other gene therapies
Haematologica. 2024 Mar 7. doi: 10.3324/haematol.2023.284622. Online ahead of print.ABSTRACTThree major directions for the global progress of adeno-associated virus (AAV) vectors for gene therapies (GT) are analyzed: a) engineering vectors to increase transgene expression; b) aligning interests of the health system with costs and challenges for pharmaceutical industry; c) refining patient eligibility criteria, and endpoints definition. Currently employed AAV vectors may cause toxicity and adverse events. Furthermore, studies in animals do not fully predict risks and clinical benefits of AAV-based GT, and animal models refl...
Source: Haematologica - March 7, 2024 Category: Hematology Authors: Giovanni Di Minno Wolfgang Miesbach Giancarlo Castaman Flora Peyvandi Source Type: research
Mental health in persons with von Willebrand disease in the United States - A large national database study
CONCLUSIONS: Our study shows that mental health disorders in PwvWD are a significant health burden, and that burden is increased with documented bleeding symptoms. It is important that primary care physicians and hematologists caring for this population recognize this increased risk, and appropriately screen and refer to mental health professionals.PMID:38453024 | DOI:10.1016/j.jtha.2024.02.015 (Source: Thrombosis and Haemostasis)
Source: Thrombosis and Haemostasis - March 7, 2024 Category: Hematology Authors: Andrew Tran Emily Waller Joana M Mack Shelley E Crary Divyaswathi Citla-Sridhar Source Type: research
Comprehensive Domain-Specific Analysis and IgG Profiling of anti-FVIII Antibodies using a bead-based multiplex immunoassay
CONCLUSION: LumiTope assay provides a sensitive and specific method for detection but also domain specification of anti-FVIII-antibodies. Implementation of bead-based assays could improve antibody detection, profiling, comparability of results, and can complement NBA.PMID:38453023 | DOI:10.1016/j.jtha.2024.02.016 (Source: Thrombosis and Haemostasis)
Source: Thrombosis and Haemostasis - March 7, 2024 Category: Hematology Authors: Behnaz Pezeshkpoor Ann-Cristin Berkemeier Kerstin Herbst Thilo Albert Jens M üller Johannes Oldenburg Source Type: research
Hemophilia and hepatology, back to the future
Rev Esp Enferm Dig. 2024 Mar 7;116. doi: 10.17235/reed.2024.10105/2023. Online ahead of print.ABSTRACTno abstract.PMID:38450508 | DOI:10.17235/reed.2024.10105/2023 (Source: Revista Espanola de Enfermedades Digestivas)
Source: Revista Espanola de Enfermedades Digestivas - March 7, 2024 Category: Gastroenterology Authors: Antonio Olveira V íctor Jiménez Source Type: research
Next-generation strategies to improve safety and efficacy of adeno-associated virus-based gene therapy for hemophilia: lessons from clinical trials in other gene therapies
Haematologica. 2024 Mar 7. doi: 10.3324/haematol.2023.284622. Online ahead of print.ABSTRACTThree major directions for the global progress of adeno-associated virus (AAV) vectors for gene therapies (GT) are analyzed: a) engineering vectors to increase transgene expression; b) aligning interests of the health system with costs and challenges for pharmaceutical industry; c) refining patient eligibility criteria, and endpoints definition. Currently employed AAV vectors may cause toxicity and adverse events. Furthermore, studies in animals do not fully predict risks and clinical benefits of AAV-based GT, and animal models refl...
Source: Haematologica - March 7, 2024 Category: Hematology Authors: Giovanni Di Minno Wolfgang Miesbach Giancarlo Castaman Flora Peyvandi Source Type: research
Mental health in persons with von Willebrand disease in the United States - A large national database study
CONCLUSIONS: Our study shows that mental health disorders in PwvWD are a significant health burden, and that burden is increased with documented bleeding symptoms. It is important that primary care physicians and hematologists caring for this population recognize this increased risk, and appropriately screen and refer to mental health professionals.PMID:38453024 | DOI:10.1016/j.jtha.2024.02.015 (Source: Thrombosis and Haemostasis)
Source: Thrombosis and Haemostasis - March 7, 2024 Category: Hematology Authors: Andrew Tran Emily Waller Joana M Mack Shelley E Crary Divyaswathi Citla-Sridhar Source Type: research
Comprehensive Domain-Specific Analysis and IgG Profiling of anti-FVIII Antibodies using a bead-based multiplex immunoassay
CONCLUSION: LumiTope assay provides a sensitive and specific method for detection but also domain specification of anti-FVIII-antibodies. Implementation of bead-based assays could improve antibody detection, profiling, comparability of results, and can complement NBA.PMID:38453023 | DOI:10.1016/j.jtha.2024.02.016 (Source: Thrombosis and Haemostasis)
Source: Thrombosis and Haemostasis - March 7, 2024 Category: Hematology Authors: Behnaz Pezeshkpoor Ann-Cristin Berkemeier Kerstin Herbst Thilo Albert Jens M üller Johannes Oldenburg Source Type: research
Hemophilia and hepatology, back to the future
Rev Esp Enferm Dig. 2024 Mar 7;116. doi: 10.17235/reed.2024.10105/2023. Online ahead of print.ABSTRACTno abstract.PMID:38450508 | DOI:10.17235/reed.2024.10105/2023 (Source: Revista Espanola de Enfermedades Digestivas)
Source: Revista Espanola de Enfermedades Digestivas - March 7, 2024 Category: Gastroenterology Authors: Antonio Olveira V íctor Jiménez Source Type: research
Next-generation strategies to improve safety and efficacy of adeno-associated virus-based gene therapy for hemophilia: lessons from clinical trials in other gene therapies
Haematologica. 2024 Mar 7. doi: 10.3324/haematol.2023.284622. Online ahead of print.ABSTRACTThree major directions for the global progress of adeno-associated virus (AAV) vectors for gene therapies (GT) are analyzed: a) engineering vectors to increase transgene expression; b) aligning interests of the health system with costs and challenges for pharmaceutical industry; c) refining patient eligibility criteria, and endpoints definition. Currently employed AAV vectors may cause toxicity and adverse events. Furthermore, studies in animals do not fully predict risks and clinical benefits of AAV-based GT, and animal models refl...
Source: Haematologica - March 7, 2024 Category: Hematology Authors: Giovanni Di Minno Wolfgang Miesbach Giancarlo Castaman Flora Peyvandi Source Type: research
Hemophilia and hepatology, back to the future
Rev Esp Enferm Dig. 2024 Mar 7;116. doi: 10.17235/reed.2024.10105/2023. Online ahead of print.ABSTRACTno abstract.PMID:38450508 | DOI:10.17235/reed.2024.10105/2023 (Source: Revista Espanola de Enfermedades Digestivas)
Source: Revista Espanola de Enfermedades Digestivas - March 7, 2024 Category: Gastroenterology Authors: Antonio Olveira V íctor Jiménez Source Type: research
Next-generation strategies to improve safety and efficacy of adeno-associated virus-based gene therapy for hemophilia: lessons from clinical trials in other gene therapies
Haematologica. 2024 Mar 7. doi: 10.3324/haematol.2023.284622. Online ahead of print.ABSTRACTThree major directions for the global progress of adeno-associated virus (AAV) vectors for gene therapies (GT) are analyzed: a) engineering vectors to increase transgene expression; b) aligning interests of the health system with costs and challenges for pharmaceutical industry; c) refining patient eligibility criteria, and endpoints definition. Currently employed AAV vectors may cause toxicity and adverse events. Furthermore, studies in animals do not fully predict risks and clinical benefits of AAV-based GT, and animal models refl...
Source: Haematologica - March 7, 2024 Category: Hematology Authors: Giovanni Di Minno Wolfgang Miesbach Giancarlo Castaman Flora Peyvandi Source Type: research
