Hemophilia A

surgery

Minor Surgeries Safe for Hemophilia With or Without Emicizumab, Study Says

Researchers discovered that minor surgeries can be safely performed in patients with hemophilia A receiving emicizumab prophyalxis, with or without factor VIII (FVIII) inhibitors, as published in Haemophilia. Since patients with hemophilia A are at a greater risk of prolonged bleeding, the management of surgery is an important aspect of hemophilia care. However, minor surgical…

hepatocytes

Gene Therapy Sustains Factor VIII and Hepatic Expressions in Hemophilia Study

A single infusion of valoctocogene roxaparvovec in adult patients with hemophilia A resulted in persistent expression of a modified human clotting factor VIII without long-term hepatic issues, according to study analyses published in Nature Medicine. Histopathology of liver biopsy samples taken from 5 patients who had received infusions of valoctocogene roxaparvovec revealed no architectural distortion,…

Exercisers

Newly Identified Risk Factors for Bleeding in Hemophilia

Researchers revealed several factors, including plasma factor levels, bleed history, and physical activity, as risk factors for bleeding in people with hemophilia, as published in the Journal of Thrombosis Haemostasis. They found no clear risk assessment model (RAM) but noted that additional factors, such as obesity and antithrombotic treatment, should also be investigated. “The identification…

platelet plasma

Emicizumab Benefits Neonate Hemophilia Model

Initiating treatment with emicizumab in the neonatal period may benefit patients with hemophilia A, according to a new study published in Pediatric Blood & Cancer. The experimental study conducted by Takeyama and colleagues analyzed plasma from 21 healthy neonates and created a hemophilia model by adding an anti-factor (F) VIII A2 monoclonal antibody (mAb) to…

Defenestrated Endothelium Affects Gene Transfer in Hemophilia

Researchers discovered that defenestrated endothelium can cause a delay in liver-directed gene transfer in patients with hemophilia A, according to a new study published in Blood Advances. Hemophilia A, if left untreated, can cause spontaneous bleeding into the joints, eventually leading to severe arthropathy. Medical researchers are increasingly looking at gene therapy as a potential…

blood clot

Anti-APC Monoclonal Antibody Improves Clotting in Hemophilia Mouse Model

Researchers demonstrated improved clotting in hemophilia A and B mice expressing human activated protein C (APC) after the administration of human APC mAb 1573 (HAPC1573), as published in Blood Advances. HAPC1573 is a monoclonal antibody that selectively blocks human protein C anticoagulant activity. The study was undertaken in an effort to overcome the limitations of…

clot

FDA Grants Fast Track Designation to ASC618 for Hemophilia A

The second-generation gene therapy for hemophilia A called ASC618 has received Fast Track Designation from the US Food and Drug Administration (FDA), as published in a press release. It also received a positive opinion for Orphan Medical Product Designation from the European Medical Agency’s (EMA) Committee for Orphan Medical Products. “This brings us one step…

chemical sampler

Computational Methods Analyze Effects of Variants in Hemophilia A

Researchers assessed the manner in which physicochemical properties affect severity and protein functionality in 5 human factor VIII (FVIII) variants that cause hemophilia A and published their results in Computer Methods and Programs in Biomedicine. They employed the Delphi web server tool to determine that a range of measures regarding the nature and position of…

Coagulation factor VIII

Tissue Factor Pathway Inhibitor as a Target to Manage Hemophilia Bleeding

Tissue factor pathway inhibitor (TFPI) could be a novel target for the treatment of hemophilia, according to a new study published in the Journal of Thrombosis and Haemostasis. The inhibition of TFPI can increase coagulation independently of clotting factors FVIII and FIX which could make it a treatment for both hemophilia A and hemophilia B.…

blood vessels

Gene Therapy Reduces Bleeding and FVIII Usage in Hemophilia Phase 3 Trial

A single infusion of valoctocogene roxaparvovec gene therapy greatly increased factor VIII (FVIII) production and reduced the annualized bleeding rate and use of concentrated FVIII in patients with hemophilia A, according to a 1-year analysis of study data published in the New England Journal of Medicine. The results showed that participants had increased mean FVIII…

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