ArticleBlood2020
BIVV001, a new class of factor VIII replacement for hemophilia A that is independent of von Willebrand factor in primates and mice.
Article in Blood, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 50 papers, 2 of them syntheses that pooled it.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
50 citing papers in PubMed, 2 syntheses or guidelines pooled it, 104 citations in OpenAlex.
- Efanesoctocog Alfa Versus Emicizumab in Adolescent and Adult Patients With Haemophilia A Without Inhibitors.Advances in therapy · 2025Pooled it
- Efanesoctocog Alfa versus Standard and Extended Half-Life Factor VIII Prophylaxis in Adolescent and Adult Patients with Haemophilia A without Inhibitors.Advances in therapy · 2025Pooled it
- Efanesoctocog Alfa Population Pharmacokinetics and Repeated Time-To-Event Analysis of Bleeds in Adults, Adolescents, and Children with Severe Hemophilia A.Journal of clinical pharmacology · 2025Trial
- Perioperative Management With Efanesoctocog Alfa in Patients With Haemophilia A in the Phase 3 XTEND-1 and XTEND-Kids Studies.Haemophilia : the official journal of the World Federation of Hemophilia · 2025Trial
- Treatment of Bleeding Episodes With Efanesoctocog Alfa in Previously Treated Patients With Severe Hemophilia A in the Phase 3 XTEND-1 Study.American journal of hematology · 2025Trial
- A Psychometric Analysis of the Haemophilia Quality of Life Questionnaire for Adults Physical Health Subscale and Total Scores: Data From the Phase 3 XTEND-1 Trial in Patients With Severe Haemophilia A.Haemophilia : the official journal of the World Federation of HemophiliaTrial
- Treatment satisfaction and patient preference for efanesoctocog alfa over previous hemophilia A treatment: Results from the XTEND-1 phase 3 clinical trial.Current therapeutic research, clinical and experimental · 2026Article
- Repeated short-interval administration of efanesoctocog alfa is not associated with increased global coagulation potential in hemophilia A mice.International journal of hematology · 2026Article
- Experiences with Efanesoctocog Alfa: Exit Interviews with Caregivers of Previously Treated XTEND-Kids Phase III Trial Patients with Haemophilia A.The patient · 2026Article
- Cost comparison of efanesoctocog alfa vs conventional factor VIII therapies for major surgeries in patients with severe hemophilia A.Blood vessels, thrombosis & hemostasis · 2026Article
- A revised classification of FVIII concentrates: rationale and novel metrics.Blood advances · 2026Article
- Article
- Cost Effectiveness of Efanesoctocog Alfa Versus Factor VIII Extended Half-Life in Adolescent and Adult Patients with Hemophilia A in the USA.PharmacoEconomics · 2026Article
- Molecular engineering approaches to half-life extension of therapeutic biomolecules.Frontiers in pharmacology · 2026Review
- Individual Comparative PK Evaluation of Single-dose Octocog Alfa, Rurioctocog Alfa Pegol, and Efanesoctocog Alfa in Adults with Severe Hemophilia A.TH open : companion journal to thrombosis and haemostasis · 2026Article
- Outcomes in participants switching from FVIII replacement therapy to efanesoctocog alfa prophylaxis in XTEND-1: a post hoc analysis.Therapeutic advances in hematology · 2026Article
- How multispecific molecules are transforming pharmacotherapy.Nature reviews. Drug discovery · 2025Review
- Factor IXa and factor X influence factor VIIIa stability and inactivation mechanisms in vitro and in vivo.Blood · 2025Article
- Regulation of immune responses to therapeutic factor VIII by transplacental delivery of Fc-fused immunodominant factor VIII domains or peptides.Haematologica · 2025Article
- Turning up the heat: optimizing Bethesda assays for efanesoctocog alfa monitoring.Research and practice in thrombosis and haemostasis · 2025Article
Corrections and comments
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Authors and funding
30 authors at 3 institutions in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Factor VIII (FVIII) replacement products enable comprehensive care in hemophilia A. Treatment goals in severe hemophilia A are expanding beyond low annualized bleed rates to include long-term outcomes associated with high sustained FVIII levels. Endogenous von Willebrand factor (VWF) stabilizes and protects FVIII from degradation and clearance, but it also subjects FVIII to a half-life ceiling of ∼15 to 19 hours. Increasing recombinant FVIII (rFVIII) half-life further is ultimately dependent upon uncoupling rFVIII from endogenous VWF. We have developed a new class of FVIII replacement, rFVIIIFc-VWF-XTEN (BIVV001), that is physically decoupled from endogenous VWF and has enhanced pharmacokinetic properties compared with all previous FVIII products. BIVV001 was bioengineered as a unique fusion protein consisting of a VWF-D'D3 domain fused to rFVIII via immunoglobulin-G1 Fc domains and 2 XTEN polypeptides (Amunix Pharmaceuticals, Inc, Mountain View, CA). Plasma FVIII half-life after BIVV001 administration in mice and monkeys was 25 to 31 hours and 33 to 34 hours, respectively, representing a three- to fourfold increase in FVIII half-life. Our results showed that multifaceted protein engineering, far beyond a few amino acid substitutions, could significantly improve rFVIII pharmacokinetic properties while maintaining hemostatic function. BIVV001 is the first rFVIII with the potential to significantly change the treatment paradigm for severe hemophilia A by providing optimal protection against all bleed types, with less frequent doses. The protein engineering methods described herein can also be applied to other complex proteins.
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Registered trials
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.