Rare Coagulation Factor Deficiencies (Factors VII, X, V, and II)

Batsuli, Glaivy; Kouides, Peter A.

doi:10.1016/j.hoc.2021.07.010

Cited by 17 publications

(11 citation statements)

References 63 publications

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“…Congenital coagulopathies, which are derived from a hereditary alteration in the genes coding for different coagulation proteins, may be autosomal or sex linked, dominant or recessive, and monogenic or polygenic [ 230 , 231 , 232 ]. Different coagulopathies have been described, including von Willebrand’s disease (deficiency of vWF), hemophilia A (deficiency of FVIII), hemophilia B (deficiency of FIX), FXI deficiency, and several alterations in other clotting factors, such as deficiencies of fibrinogen, prothrombin, FV, FVII, and FX, among others [ 233 ].…”

Section: Dysregulation Of Homeostasismentioning

confidence: 99%

The Vascular Endothelium and Coagulation: Homeostasis, Disease, and Treatment, with a Focus on the Von Willebrand Factor and Factors VIII and V

Pablo-Moreno

Serrano

Revuelta

et al. 2022

IJMS

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The vascular endothelium has several important functions, including hemostasis. The homeostasis of hemostasis is based on a fine balance between procoagulant and anticoagulant proteins and between fibrinolytic and antifibrinolytic ones. Coagulopathies are characterized by a mutation-induced alteration of the function of certain coagulation factors or by a disturbed balance between the mechanisms responsible for regulating coagulation. Homeostatic therapies consist in replacement and nonreplacement treatments or in the administration of antifibrinolytic agents. Rebalancing products reestablish hemostasis by inhibiting natural anticoagulant pathways. These agents include monoclonal antibodies, such as concizumab and marstacimab, which target the tissue factor pathway inhibitor; interfering RNA therapies, such as fitusiran, which targets antithrombin III; and protease inhibitors, such as serpinPC, which targets active protein C. In cases of thrombophilia (deficiency of protein C, protein S, or factor V Leiden), treatment may consist in direct oral anticoagulants, replacement therapy (plasma or recombinant ADAMTS13) in cases of a congenital deficiency of ADAMTS13, or immunomodulators (prednisone) if the thrombophilia is autoimmune. Monoclonal-antibody-based anti-vWF immunotherapy (caplacizumab) is used in the context of severe thrombophilia, regardless of the cause of the disorder. In cases of disseminated intravascular coagulation, the treatment of choice consists in administration of antifibrinolytics, all-trans-retinoic acid, and recombinant soluble human thrombomodulin.

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Section: Dysregulation Of Homeostasismentioning

confidence: 99%

The Vascular Endothelium and Coagulation: Homeostasis, Disease, and Treatment, with a Focus on the Von Willebrand Factor and Factors VIII and V

Pablo-Moreno

Serrano

Revuelta

et al. 2022

IJMS

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“…The prothrombin complex formed by coagulation factors V and X is the key factor driving thrombin generation (Camire, 2021;Lam and Moosavi, 2022). Tissue factor (TF) is released when the blood vessel is damaged and collagen fibers in the subintima are exposed, which is initiated when TF binds to FVII, activates FVII to factor VIIa (FVIIa), and forms a TF-FVIIa complex (Batsuli and Kouides, 2021). Once factor X is activated to FXa by the TF-FVIIa…”

Section: Physiological Hemostasismentioning

confidence: 99%

“…Frontiers in Materials frontiersin.org complex, the cascade continues along a common pathway to convert fibrinogen to fibrin. Among them, TF is the initiation of the extrinsic coagulation pathway and FVII is the initiation of the intrinsic coagulation pathway (Batsuli and Kouides, 2021). FXa generated by the extrinsic and intrinsic coagulation pathways can form FXa-FVa-Ca2+-phospholipid complexes, namely, prothrombinase complexes, with FVa on the surface of phospholipid membranes in the presence of Ca2+.…”

Section: Physiological Hemostasismentioning

confidence: 99%

Application of chitosan-based materials in surgical or postoperative hemostasis

et al. 2022

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Uncontrolled bleeding from trauma or surgery remains an important factor affecting the survival and prognosis of surgical patients. Failure to timeously stop bleeding will not only prolong the operative time but also threaten the patient’s life. Timely hemostasis after bleeding has become the most concerned event for surgeons. At present, the most commonly used hemostasis methods in the operating room include ligation of blood vessels, electrocautery, and gauze compression to stop bleeding. However, These hemostatic methods do great harm to surrounding tissues while achieving hemostasis. Based on tissue engineering repair strategies, the use of natural polymer materials as hemostatic agents has achieved clinical success. Gelatin sponge and cellulose gauze have been used clinically with good results. However, gelatin sponges are very expensive and place enormous financial pressure on patients. Therefore, there is an urgent need for new hemostatic materials for surgical hemostasis. Chitosan is a natural polysaccharide with biocompatibility and biodegradability, which plays an important role in tissue engineering and regenerative medicine. Chitosan gauze has been proven to have good hemostatic effects. The positive charge on the surface of chitosan can adsorb red blood cells and platelets at the bleeding site to form platelet thrombosis. However, chitosan is not easily soluble in water and has poor adsorption, which makes it a weak local hemostatic agent. Therefore, it is important to improve chitosan-based hemostatic material such that it l has an excellent hemostatic effect. In this review, we introduce the physiological coagulation process and discuss the physicochemical properties of chitosan and its role in hemostasis. Furthermore, we discuss the advantages and disadvantages of chitosan-based hemostatic materials. Finally, we summarize and discuss chitosan-based hemostatic materials.

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“…Rare and ultra-rare inherited BDs which cause significant morbidity remain difficult to diagnose, and are largely underdiagnosed globally despite technical advances. 20,21 Evidence-based guidelines for the treatment of most inherited BDs are largely absent, due to the lack of supporting aggregate data. 22 Sexism has been an issue in BD for centuries.…”

Section: Introductionmentioning

confidence: 99%

“…Von Willebrand disease (VWD), the most common inherited BD, inherited equally by men and women, is clinically complex and technically challenging to diagnose, 19 yet far fewer resources have been devoted to it than to haemophilia. Rare and ultra‐rare inherited BDs which cause significant morbidity remain difficult to diagnose, and are largely underdiagnosed globally despite technical advances 20,21 . Evidence‐based guidelines for the treatment of most inherited BDs are largely absent, due to the lack of supporting aggregate data 22 .…”

Section: Introductionmentioning

confidence: 99%

Building the blueprint: Formulating a community‐generated national plan for future research in inherited bleeding disorders

et al. 2022

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Introduction Decades of inherited bleeding disorders (BD) research transformed severe haemophilia from a childhood killer to a disorder managed across a full lifespan for many in economically developed countries. Health equity, a life unimpaired by disease complications, however, remains unimaginable for most people with an inherited BD (PWIBD). Aim The National Hemophilia Foundation (NHF) and American Thrombosis and Hemostasis Network (ATHN) undertook the development of a community‐driven United States (US) National Blueprint for Inherited Bleeding Disorders Research to transform the experience of all PWIBD and those who care for them. Methods Extensive community consultations were conducted to identify the issues most important to PWIBD and those who love and care for them. Expert multidisciplinary teams distilled these key areas of need into prioritised research questions, and identified the resources and infrastructure required to pursue them. A summit was held to gather feedback and inform the detailed blueprint. Results Community‐prioritised research areas fell into three broad categories: issues common across inherited BDs, those specific to individual disorders, and issues of infrastructure and capacity. NHF State of the Science Research Summit discussions of the research questions derived from the community priorities by six working groups provided important input for the drafting of the research blueprint for the coming decades. Conclusion The inherited BD community came together to develop the US National Blueprint for Inherited Bleeding Disorders Research dedicated to transforming the lives of all PWIBD including innovating solutions for the rarest disorders and under‐represented populations.

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Rare Coagulation Factor Deficiencies (Factors VII, X, V, and II)

Cited by 17 publications

References 63 publications

The Vascular Endothelium and Coagulation: Homeostasis, Disease, and Treatment, with a Focus on the Von Willebrand Factor and Factors VIII and V

The Vascular Endothelium and Coagulation: Homeostasis, Disease, and Treatment, with a Focus on the Von Willebrand Factor and Factors VIII and V

Application of chitosan-based materials in surgical or postoperative hemostasis

Building the blueprint: Formulating a community‐generated national plan for future research in inherited bleeding disorders

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