دورية أكاديمية
Plasma-derived FVIII/VWF complex shows higher protection against inhibitors than isolated FVIII after infusion in haemophilic patients: A translational study.
العنوان: | Plasma-derived FVIII/VWF complex shows higher protection against inhibitors than isolated FVIII after infusion in haemophilic patients: A translational study. |
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المؤلفون: | Bravo MI; Bioscience Research Group, Grifols, Barcelona, Spain., Pérez A; Bioscience Research Group, Grifols, Barcelona, Spain., Raventós A; Bioscience Research Group, Grifols, Barcelona, Spain., Grancha S; Bioscience Research Group, Grifols, Barcelona, Spain., Jorquera JI; Bioscience Research Group, Grifols, Barcelona, Spain., Butta NV; Thrombosis and Haemostasis Unit - IdiPAZ, University Hospital La Paz, Madrid, Spain., Álvarez-Román MT; Thrombosis and Haemostasis Unit - IdiPAZ, University Hospital La Paz, Madrid, Spain., Costa M; Bioscience Research Group, Grifols, Barcelona, Spain., Willis T; Bioscience Research Group, Grifols, Raleigh, North Carolina, USA., Jiménez-Yuste V; Thrombosis and Haemostasis Unit - IdiPAZ, University Hospital La Paz, Madrid, Spain.; Medicine Department, Autonoma University of Madrid, Madrid, Spain. |
المصدر: | Haemophilia : the official journal of the World Federation of Hemophilia [Haemophilia] 2022 Sep; Vol. 28 (5), pp. 737-744. Date of Electronic Publication: 2022 Jun 02. |
نوع المنشور: | Journal Article |
اللغة: | English |
بيانات الدورية: | Publisher: Blackwell Science Country of Publication: England NLM ID: 9442916 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1365-2516 (Electronic) Linking ISSN: 13518216 NLM ISO Abbreviation: Haemophilia Subsets: MEDLINE |
أسماء مطبوعة: | Original Publication: Osney Mead, Oxford, UK : Blackwell Science, [1994- |
مواضيع طبية MeSH: | Factor VIII*/administration & dosage , Factor VIII*/immunology , Factor VIII*/isolation & purification , Hemophilia A*/therapy , von Willebrand Factor*/administration & dosage , von Willebrand Factor*/isolation & purification, Animals ; Disease Models, Animal ; Immunoglobulin G/immunology ; Mice ; Recombinant Proteins/administration & dosage |
مستخلص: | Introduction: Presence of von Willebrand factor (VWF) in FVIII concentrates offers protection against neutralizing inhibitors in haemophilia A (HA). Whether this protection is more evident in plasma-derived (pd) FVIII/VWF or recombinant (r) FVIII concentrates remains controversial. Aim: We investigated the protection exerted by VWF against FVIII inhibitors in an in vivo mouse model of HA exposed to pdFVIII/VWF or to various rFVIII concentrates. Methods: Haemophilia A mice received the different FVIII concentrates after administration of vehicle or an inhibitory IgG purified from a commercial pool of HA plasma with inhibitors and FVIII:C recoveries were measured. Furthermore, using a novel clinically oriented ex vivo approach, Bethesda inhibitory activities (BU) of a commercial pool of HA plasma with inhibitors were assessed using normal plasma, or plasma from severe HA patients, without inhibitors, after treatment with the same concentrates. Results: in vivo studies showed that pdFVIII/VWF offers markedly higher protection against inhibitors when compared with any of the FVIII products without VWF. More importantly, in the ex vivo studies, plasma from patients treated with pdFVIII/VWF showed higher protection against inhibitors (P values ranging .05-.001) in comparison with that observed in plasma from patients who received FVIII products without VWF, regardless of the type of product evaluated. Conclusion: Data indicate that FVIII+VWF complexes assembled in the circulation after rFVIII infusion are not equivalent to the naturally formed complex in pdFVIII/VWF. Therefore, rFVIII infused into HA patients with inhibitors would be less protected by VWF than the FVIII in pdFVIII/VWF concentrates. (© 2022 The Authors. Haemophilia published by John Wiley & Sons Ltd.) |
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معلومات مُعتمدة: | Grifols |
فهرسة مساهمة: | Keywords: FVIII inhibitors; FVIIInull E16 Knockout mice; haemophilia A patients; haemophilia A treatment; plasma-derived FVIII; recombinant FVIII; von Willebrand factor |
المشرفين على المادة: | 0 (Immunoglobulin G) 0 (Recombinant Proteins) 0 (von Willebrand Factor) 9001-27-8 (Factor VIII) |
تواريخ الأحداث: | Date Created: 20220602 Date Completed: 20220913 Latest Revision: 20221015 |
رمز التحديث: | 20221213 |
مُعرف محوري في PubMed: | PMC9545517 |
DOI: | 10.1111/hae.14589 |
PMID: | 35654086 |
قاعدة البيانات: | MEDLINE |
تدمد: | 1365-2516 |
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DOI: | 10.1111/hae.14589 |