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A global comparative field study to evaluate the factor VIII activity of efanesoctocog alfa by one-stage clotting and chromogenic substrate assays at clinical haemostasis laboratories.

التفاصيل البيبلوغرافية
العنوان: A global comparative field study to evaluate the factor VIII activity of efanesoctocog alfa by one-stage clotting and chromogenic substrate assays at clinical haemostasis laboratories.
المؤلفون: Pipe S; Departments of Pediatrics and Pathology, University of Michigan, Ann Arbor, Michigan, USA., Sadeghi-Khomami A; Precision BioLogic, Dartmouth, Nova Scotia, Canada., Konkle BA; Washington Center for Bleeding Disorders, Seattle, Washington, USA.; Division of Hematology, University of Washington, Seattle, Washington, USA., Kitchen S; Department of Coagulation, Royal Hallamshire Hospital, Sheffield Teaching Hospitals NHS Foundation Trust, Sheffield, UK., Negrier C; University Lyon 1, Lyon, France., Liu M; Sanofi, Bridgewater, New Jersey, USA., Santagostino E; Sobi, Stockholm, Sweden., Willemze A; Sanofi, Amsterdam, Netherlands., Abad-Franch L; Sobi, Stockholm, Sweden., Knobe K; Sanofi, Chilly-Mazarin, France., Seth Chhabra E; Sanofi, Cambridge, Massachusetts, USA.
المصدر: Haemophilia : the official journal of the World Federation of Hemophilia [Haemophilia] 2024 Jan; Vol. 30 (1), pp. 214-223. Date of Electronic Publication: 2023 Oct 30.
نوع المنشور: Comparative Study; 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: Hemophilia A*/drug therapy , Hemostatics*/therapeutic use , Sleep Apnea, Obstructive*/drug therapy, Humans ; Actins ; Blood Coagulation Tests/methods ; Chromogenic Compounds/therapeutic use ; Factor VIII/therapeutic use ; Hemostasis ; Indicators and Reagents ; Laboratories ; Reproducibility of Results
مستخلص: Introduction: Structural and chemical modifications of factor VIII (FVIII) products may influence their behaviour in FVIII activity assays. Hence, it is important to assess the performance of FVIII products in these assays. Efanesoctocog alfa is a new class of FVIII replacement therapy designed to provide both high sustained factor activity levels and prolonged plasma half-life.
Aim: Evaluate the accuracy of measuring efanesoctocog alfa FVIII activity in one-stage clotting assays (OSAs) and chromogenic substrate assays (CSAs).
Methods: Human plasma with no detectable FVIII activity was spiked with efanesoctocog alfa or a full-length recombinant FVIII product comparator, octocog alfa, at nominal concentrations of 0.80 IU/mL, 0.20 IU/mL, or 0.05 IU/mL, based on labelled potency. Clinical haemostasis laboratories (N = 35) tested blinded samples using in-house assays. Data from 51 OSAs (14 activated partial thromboplastin time [aPTT] reagents) and 42 CSAs (eight kits) were analyzed.
Results: Efanesoctocog alfa activity was reliably (±25% of nominal activity) measured across all concentrations using OSAs with Actin FSL and multiple other aPTT reagents. Under- and overestimation of FVIII activity occurred with some reagents. No specific trend was observed for any class of aPTT activators. A two- to three-fold overestimation was consistently observed using CSAs and the OSA with Actin FS as the aPTT reagent across evaluated concentrations.
Conclusion: Under- or overestimation occurred with some specific OSAs and most CSAs, which has been previously observed with other modified FVIII replacement products. Efanesoctocog alfa FVIII activity was measured with acceptable accuracy and reliability using several OSA methods and commercial plasma standards.
(© 2023 Sanofi and The Authors. Haemophilia published by John Wiley & Sons Ltd.)
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فهرسة مساهمة: Keywords: FVIII replacement therapy; Haemophilia A; aPTT; assay; chromogenic; coagulation; von Willebrand factor
المشرفين على المادة: 0 (Actins)
0 (Chromogenic Compounds)
839MOZ74GK (F8 protein, human)
9001-27-8 (Factor VIII)
0 (Hemostatics)
0 (Indicators and Reagents)
تواريخ الأحداث: Date Created: 20231030 Date Completed: 20240116 Latest Revision: 20240116
رمز التحديث: 20240116
DOI: 10.1111/hae.14831
PMID: 37902390
قاعدة البيانات: MEDLINE
الوصف
تدمد:1365-2516
DOI:10.1111/hae.14831