Trial reportThrombosis and haemostasis2021
Pharmacokinetics and Pharmacodynamics of Emicizumab in Persons with Hemophilia A with Factor VIII Inhibitors: HAVEN 1 Study.
Trial report in Thrombosis and haemostasis, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT02622321 (A Randomized, Multicenter, Open-Label, Phase III Clinical Trial to Evaluate the Efficacy, Safety, and Pharmacokinetics of Prophylactic Emicizumab Versus no Prophylaxis in Hemophilia A Patients With Inhibitors), which is not on this map. Cited by 46 papers, 5 of them syntheses that pooled it.
What it found
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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.
A Randomized, Multicenter, Open-Label, Phase III Clinical Trial to Evaluate the Efficacy, Safety, and Pharmacokinetics of Prophylactic Emicizumab Versus no Prophylaxis in Hemophilia A Patients With Inhibitors
Who cites it
46 citing papers in PubMed, 5 syntheses or guidelines pooled it, 94 citations in OpenAlex.
- Non-clotting factor therapies for preventing bleeds in people with congenital hemophilia A or B.The Cochrane database of systematic reviews · 2024Pooled it
- Pharmacokinetics and Associated Efficacy of Emicizumab in Humans: A Systematic Review.Clinical pharmacokinetics · 2021Pooled it
- Factor VIII replacement prophylaxis in patients with hemophilia A transitioning to adults: a systematic literature review.Orphanet journal of rare diseases · 2021Pooled it
- Bispecific Antibodies: From Research to Clinical Application.Frontiers in immunology · 2021Pooled it
- Outcomes of Emicizumab in Acquired Hemophilia Patients: A Systematic Review.Clinical and applied thrombosis/hemostasis : official journal of the International Academy of Clinical and Applied Thrombosis/HemostasisPooled it
- Trial
- Emicizumab dose up-titration in case of suboptimal bleeding control in people with haemophilia A.Haemophilia : the official journal of the World Federation of Hemophilia · 2023Trial
- [Evaluation of the efficacy and safety of reduced-dose emicizumab in infants with severe hemophilia A: a single-center real-world study].Zhongguo dang dai er ke za zhi = Chinese journal of contemporary pediatrics · 2026Article
- A Case of Acquired Hemophilia A Following Robot-Assisted Minimally Invasive Esophagectomy.Surgical case reports · 2026Article
- Massive Intra-Abdominal Pseudotumor in a Patient With Hemophilia A Treated With Emicizumab: A Case Report.Clinical case reports · 2026Article
- Factor VIIIResearch and practice in thrombosis and haemostasis · 2026Article
- Emicizumab, the factor VIII mimetic bispecific monoclonal antibody: effects on thrombin generation and thromboelastometry.Research and practice in thrombosis and haemostasis · 2025Article
- From pharmacokinetic-guided FVIII prophylaxis to low-dose Emicizumab prophylaxis: a pilot study revealed a flush of hope.Annals of hematology · 2025Article
- Mathematical analysis of emicizumab: affinity-driven complex formation and lipid-surface reactions.Journal of thrombosis and haemostasis : JTH · 2025Article
- Estimating the Factor VIII-Equivalent Activity of Emicizumab Using Global Assays of Haemostasis.Haemophilia : the official journal of the World Federation of Hemophilia · 2025Article
- TSUBASA study: evaluation of the quality and content of daily life of people with hemophilia A without factor VIII inhibitors on prophylactic treatment with emicizumab.Research and practice in thrombosis and haemostasis · 2025Article
- Thrombin Generation Assay to Support Hematologists in the Era of New Hemophilia Therapies.International journal of laboratory hematology · 2025Review
- Predicting Inhibitor Development in Hemophilia 'A' using Machine Learning: A Comprehensive Approach to Data Preprocessing, Balancing, and Biomarker Identification Using AI on the CHAMP Dataset.Current pharmaceutical biotechnology · 2025Article
- No correlation between thrombin generation and emicizumab levels: implications for monitoring emicizumab therapy.Research and practice in thrombosis and haemostasis · 2025Article
- Real-world use of emicizumab in Chinese children with hemophilia A: Retrospective data from a comprehensive care center.Pediatric investigation · 2024Article
Corrections and comments
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Authors and funding
9 authors at 4 institutions in 3 countries.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Emicizumab, a bispecific monoclonal antibody, bridges activated factor IX (FIXa) and FX, replacing the function of missing FVIIIa to restore effective hemostasis in persons with hemophilia A (PwHA). Here we assess pharmacokinetic (PK) and pharmacodynamic (PD) biomarkers in PwHA with FVIII inhibitors in the Phase III HAVEN 1 study (NCT02622321). Blood samples from 112 PwHA receiving 1.5 mg/kg once-weekly subcutaneous emicizumab were analyzed at central laboratories. Emicizumab concentrations for PK analysis were measured via validated immunoassay. PD effects were assessed using FVIII chromogenic activity assay containing human factors (Hyphen Biophen FVIII:C), and by FXIa-triggered thrombin generation (TG). Activated partial thromboplastin time (aPTT), prothrombin time (PT), antigen levels of FIX and FX, fibrinogen, D-dimer, and prothrombin fragment 1.2 (PF1.2) levels were determined. Emicizumab trough concentrations ≥ 50 µg/mL were maintained throughout the study. FVIII-like activity and TG (peak height) correlated with emicizumab concentrations and remained above 20 U/dL and 100 nM, respectively, with a weekly maintenance dose, theoretically converting persons with severe hemophilia A to a mild disease phenotype. aPTT was normalized at subtherapeutic concentrations of emicizumab. Plasma concentrations of target antigens FIX and FX were not significantly affected by emicizumab treatment; nor were fibrinogen, PT (international normalized ratio), D-dimer, or PF1.2. The PK profile of once-weekly emicizumab in HAVEN 1 provides sustained therapeutic plasma levels, consistent with population PK models. Both the PK profile and the PD and safety biomarkers are consistent with the established efficacy of emicizumab prophylaxis in PwHA with FVIII inhibitors.
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