ArticleBlood2020
A hemophilia A mouse model for the in vivo assessment of emicizumab function.
Article in Blood, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 36 papers, 1 of them a synthesis that pooled it.
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Who cites it
36 citing papers in PubMed, 1 synthesis or guideline pooled it, 60 citations in OpenAlex.
- Pharmacokinetics and Associated Efficacy of Emicizumab in Humans: A Systematic Review.Clinical pharmacokinetics · 2021Pooled it
- A novel humanized FIX/FX hemophilia A mouse model for preclinical evaluation of FVIIIa-mimetic bispecific antibodies.Blood advances · 2026Article
- Of mice and FVIIIa mimetics: new hemophilia A models for in vivo evaluation.Blood advances · 2026Article
- Plasma emicizumab concentrations and bleeding rates in children and adults with severe hemophilia A.Research and practice in thrombosis and haemostasis · 2026Observational
- A miniaturized chemiluminescent thrombin generation assay to assess the effect of APCC in persons with hemophilia A with inhibitors: an observational study.Research and practice in thrombosis and haemostasis · 2026Observational
- Development of a novel hemophilia A mouse model to study emicizumab and factor VIII inhibitor risk.Blood advances · 2026Article
- Next-generation FVIIIa-mimetic bispecific antibody NXT007: evaluation in preclinical models of hemostasis and thrombosis.Blood advances · 2026Article
- Factor VIIIResearch and practice in thrombosis and haemostasis · 2026Article
- Monochorionic diamniotic twin brothers with severe hemophilia A: a case report.Frontiers in pediatrics · 2026Article
- Augmented emicizumab-driven coagulation potential in hemophilia A state byResearch and practice in thrombosis and haemostasis · 2026Article
- Characterization of a factor VIII/immunoglobulin heavy chain μ double-knockout mouse model of hemophilia A for long-term exposure to factor VIII proteins.Research and practice in thrombosis and haemostasis · 2026Article
- Estimating the Factor VIII-Equivalent Activity of Emicizumab Using Global Assays of Haemostasis.Haemophilia : the official journal of the World Federation of Hemophilia · 2025Article
- Consistent clinical factor VIII equivalency is unlikely for non-factor therapies in hemophilic mice.Haematologica · 2025Article
- Physical Activity Awareness and Understanding of Treatment Protection Among People With Haemophilia and Their Caregivers in Central Europe.Haemophilia : the official journal of the World Federation of Hemophilia · 2025Article
- Thrombin Generation Assay to Support Hematologists in the Era of New Hemophilia Therapies.International journal of laboratory hematology · 2025Review
- Development of a novel and viable knock-in factor V deficiency murine model: Utility for an ultra-rare disease.PloS one · 2025Article
- Ex vivo evaluation of the effect of plasma-derived factor VIII/von Willebrand factor in patients with severe hemophilia A on emicizumab prophylaxis.Clinical and experimental medicine · 2024Article
- Roctavian gene therapy for hemophilia A.Blood advances · 2024Review
- Enhanced procoagulant activity of select hemophilia B causing factor IX variants with emicizumab.Blood · 2024Article
- Pre-clinical evaluation of an enhanced-function factor VIII variant for durable hemophilia A gene therapy in male mice.Nature communications · 2024Article
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Authors and funding
10 authors at 2 institutions in 2 countries.
Funding
No grant is acknowledged in the PubMed record.
Abstract
The bispecific antibody emicizumab is increasingly used for hemophilia A treatment. However, its specificity for human factors IX and X (FIX and FX) has limited its in vivo functional analysis to primate models of acquired hemophilia. Here, we describe a novel mouse model that allows emicizumab function to be examined. Briefly, FVIII-deficient mice received IV emicizumab 24 hours before tail-clip bleeding was performed. A second infusion with human FIX and FX, administered 5 minutes before bleeding, generated consistent levels of emicizumab (0.7-19 mg/dL for 0.5-10 mg/kg doses) and of both FIX and FX (85 and 101 U/dL, respectively, after dosing at 100 U/kg). Plasma from these mice display FVIII-like activity in assays (diluted activated partial thromboplastin time and thrombin generation), similar to human samples containing emicizumab. Emicizumab doses of 1.5 mg/kg and higher significantly reduced blood loss in a tail-clip-bleeding model using FVIII-deficient mice. However, reduction was incomplete compared with mice treated with human FVIII concentrate, and no difference in efficacy between doses was observed. From this model, we deducted FVIII-like activity from emicizumab that corresponded to a dose of 4.5 U of FVIII per kilogram (ie, 9.0 U/dL). Interestingly, combined with a low FVIII dose (5 U/kg), emicizumab provided enough additive activity to allow complete bleeding arrest. This model could be useful for further in vivo analysis of emicizumab.
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Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.