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| http://dx.doi.org/10.1182/bloodadvances.2022008989 | DOI Listing |
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Murine vs. Human Osteoblast Responses to Coagulation and Inflammatory Factors: Reconsidering the Use of Animal Models in Hemophilia a Research.
Biomedicines
November 2024
Department of Pediatrics I, Medical University Innsbruck, 6020 Innsbruck, Austria.
: Hemophilia A is associated with frequent bleeding episodes, joint damage, and reduced bone mineral density (BMD). The role of coagulation factors and inflammatory cytokines on bone metabolism, particularly on osteoblast function, is of increasing interest. However, significant inter-species differences in bone remodeling raise concerns about the translatability of findings from murine models to human systems.
View Article and Find Full Text PDFReplacement therapy in pregnant women with von Willebrand disease during delivery: Factor levels and pharmacokinetics.
Hemasphere
January 2025
Department of Pediatric Hematology and Oncology Erasmus MC Sophia Children's Hospital, University Medical Center Rotterdam Rotterdam The Netherlands.
Limited data are available on VWF activity (VWF:Act) and factor VIII (FVIII:C) levels during delivery after VWF/FVIII concentrate administration in women with von Willebrand disease (VWD). We aimed to evaluate treatment with a specific VWF/FVIII concentrate on factor levels in women with VWD during delivery and the postpartum period. A retrospective single-center study was conducted between January 1, 2008, and August 1, 2022.
View Article and Find Full Text PDFA Prospective, Noninterventional Study to Evaluate the Impact of Emicizumab in the Management of Hemophilia A.
Cureus
December 2024
Department of Medicine, Assam Medical College and Hospital, Dibrugarh, IND.
Background and objective Hemophilia A (HA) is a genetic bleeding disorder caused by a lack of factor VIII (FVIII) and is associated with frequent bleeding and joint damage. Traditional intravenous treatments for this condition are cumbersome and can lead to complications. Emicizumab, a bispecific monoclonal antibody, offers a promising subcutaneous alternative with potential safety and efficacy-related benefits.
View Article and Find Full Text PDFLow-Dose Emicizumab Versus Low-/Intermediate-Dose Factor VIII Secondary Prophylaxis for Noninhibitor Haemophilia A Patients With Severe Bleeding Phenotype.
Haemophilia
December 2024
Division of Pediatric Hematology and Oncology, Department of Pediatrics, Faculty of Medicine, Chulalongkorn University and King Chulalongkorn Memorial Hospital, Bangkok, Thailand.
Background: Subcutaneous emicizumab, a factor VIII (FVIII)-mimicking bispecific monoclonal antibody, can effectively prevent bleeds in haemophilia A (HA) patients with/without inhibitors; however, its standard-dose regimens are financially burdensome. Low-dose emicizumab prophylaxis may alternatively be applied to noninhibitor HA patients in resource-limited settings.
Methods: During 2023, Thai patients with noninhibitor severe HA or moderate HA with severe bleeding phenotype (historical annualized bleeding rate [ABR] >5 bleeds/year before regular FVIII prophylaxis) who received low-/intermediate-dose FVIII secondary prophylaxis ≥8 months were enrolled.
Real-world use of emicizumab in Chinese children with hemophilia A: Retrospective data from a comprehensive care center.
Pediatr Investig
December 2024
Hematology Department, Hemophilia Comprehensive Care Center, Hematology Center, Beijing Key Laboratory of Pediatric Hematology-Oncology, Key Laboratory of Major Diseases in Children, National Center for Children's Health National Key Discipline of Pediatrics (Capital Medical University), Ministry of Education, Beijing Children's Hospital, Capital Medical University Beijing China.
Importance: Emicizumab (EMI) is efficacious and safe for hemophilia A (HA) prophylaxis. However, its high cost poses a challenge in China.
Objective: To explore the possibility of using reduced-dosage EMI in Chinese HA children.
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