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Nonneutralizing antibodies in Nordic persons with moderate hemophilia A and B (the MoHem study).
Res Pract Thromb Haemost
November 2024
Department of Haematology, Oslo University Hospital, Oslo, Norway.
Background: The impact of nonneutralizing antibodies (NNAs) in moderate hemophilia is elusive.
Objectives: To explore the presence of NNAs in Nordic persons with moderate hemophilia A (MHA) and B (MHB) in relation to treatment modality, clinical outcome, history of inhibitor, and the corresponding factor VIII (FVIII)/factor IX (FIX) gene mutation.
Methods: A cross-sectional multicenter study covering persons with MHA and MHB in Sweden, Finland, and Norway.
An assessment of burden associated with problem joints in children and adults with moderate or severe haemophilia A: analysis of the CHESS-Paediatrics and CHESS II cross-sectional studies.
Orphanet J Rare Dis
January 2025
Department of Physical and Rehabilitation Medicine, La Paz University Hospital (IdiPaz), Madrid, Spain.
Background: Clinical research has offered many definitions and fragmented perspectives of joint morbidity in haemophilia. As joint damage, pain and mobility impairment can be present without clinical record of persistent bleeding, a person-centric joint morbidity characterisation remained a priority for the haemophilia community, giving rise to the 'problem joint' concept. As diagnosing and managing joint morbidity is critical, the aim of this study was to analyse the holistic burden of problem joints in people with moderate or severe haemophilia A (HA).
View Article and Find Full Text PDFImpact of intermediate-dose tertiary prophylaxis on quality of life and psychological aspects of adult patients with severe/moderate hemophilia A.
Hematology
December 2025
Department of Hematology, The Third People's Hospital of Chengdu, The Affiliated Hospital of Southwest Jiaotong University, Chengdu Second Clinical College of Chongqing Medical University, Chengdu, People's Republic of China.
Objectives: Whether intermediate-dose tertiary prophylaxis can improve quality of life and psychological health in adults with severe/moderate hemophilia A has not been determined. This research aims to explore the impact of intermediate-dose tertiary prophylaxis with recombinant human FVIII (rhFVIII) on quality of life, anxiety and depression in such individuals transitioned from on-demand treatment.
Methods: This retrospective analysis collected data from July 2019 to July 2022.
Tolerance to Factor VIII in the Era of Non-Factor Therapies: Immunological Perspectives and a Systematic Review of the Literature.
J Thromb Haemost
January 2025
Amsterdam UMC location University of Amsterdam, Department of Pediatric Hematology, Meibergdreef 9, Amsterdam, The Netherlands. Electronic address:
Persons with hemophilia A (PWHA) lack clotting factor VIII (FVIII) due to a genetic mutation in the F8 gene. The administration of FVIII concentrate leads to the development of neutralizing anti-FVIII antibodies (inhibitors) in about 30% of children with severe hemophilia A. The other 70% of children do not mount a detectable antibody response, suggesting that they may have developed tolerance towards FVIII.
View Article and Find Full Text PDFSafety and efficacy of valoctocogene roxaparvovec with prophylactic glucocorticoids: 1-year results from the phase 3b, single-arm, open-label GENEr8-3 study.
J Thromb Haemost
January 2025
BioMarin Pharmaceutical Inc., Novato, CA, USA.
Background: Valoctocogene roxaparvovec, an adeno-associated virus vector that transfers a human factor VIII (FVIII) coding sequence to hepatocytes, provides bleeding protection for people with severe hemophilia A (HA).
Objective: Determine the efficacy and safety of valoctocogene roxaparvovec with concomitant prophylactic glucocorticoids in the open-label, single-arm, phase 3b GENEr8-3 trial.
Methods: Participants with severe HA who were using HA prophylaxis received one 6x10 vg/kg infusion of valoctocogene roxaparvovec concomitantly with daily prophylactic glucocorticoids (40 mg prednisolone equivalent/d weeks 0‒8; taper to 5 mg/d weeks 9‒19).
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