Trial reportHaemophilia : the official journal of the World Federation of Hemophilia2019
A multicentre, open-label study of emicizumab given every 2 or 4 weeks in children with severe haemophilia A without inhibitors.
Trial report in Haemophilia : the official journal of the World Federation of Hemophilia, 2019. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 67 papers, 3 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
67 citing papers in PubMed, 3 syntheses or guidelines pooled it, 150 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
- Outcomes of Emicizumab Treatment for Haemophilia A Paediatric Patients: A Systematic Review With Meta-Analysis.Haemophilia : the official journal of the World Federation of HemophiliaPooled it
- Coagulation potential of concomitant factor VIII administration in people with hemophilia A receiving emicizumab prophylaxis (CAGUYAMA study): a multicenter, open-label, nonrandomized clinical trial.Research and practice in thrombosis and haemostasis · 2026Trial
- 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
- Long-term safety and efficacy of the anti-tissue factor pathway inhibitor marstacimab in participants with severe haemophilia: Phase II study results.British journal of haematology · 2023Trial
- Trial
- Trial
- Trial
- Long-term safety and efficacy of emicizumab for up to 5.8 years and patients' perceptions of symptoms and daily life: A phase 1/2 study in patients with severe haemophilia A.Haemophilia : the official journal of the World Federation of Hemophilia · 2021Trial
- Evaluation of the Pharmacokinetics, Pharmacodynamics, and Safety of a Single Dose of Emicizumab in Healthy Chinese Subjects.Clinical pharmacology in drug development · 2021Trial
- A multicentre, open-label study of emicizumab given every 2 or 4 weeks in children with severe haemophilia A without inhibitors.Haemophilia : the official journal of the World Federation of Hemophilia · 2019Trial
- A novel humanized FIX/FX hemophilia A mouse model for preclinical evaluation of FVIIIa-mimetic bispecific antibodies.Blood advances · 2026Article
- Emicizumab prophylaxis in children with hemophilia A with factor VIII inhibitors in China (HAVEN 5).Chinese medical journal · 2026Article
- Maternal-fetal transfer of emicizumab in a pregnant mouse model.Blood advances · 2026Article
- Myeloid Cell Function and Cytokine Profiles in Paediatric Haemophilia A: Insights From FVIII and Emicizumab Prophylaxis.Journal of cellular and molecular medicine · 2026Article
- Transition to Emicizumab from standard-dose FVIII prophylaxis in young children with hemophilia A: a real-world study.Thrombosis journal · 2026Article
- Emicizumab prophylaxis beyond clinical trials: a multicenter, prospective real-world study of pediatric hemophilia patients with and without inhibitors.European journal of pediatrics · 2026Article
- Next-generation FVIIIa-mimetic bispecific antibody NXT007: evaluation in preclinical models of hemostasis and thrombosis.Blood advances · 2026Article
7 more citing papers are in PubMed but not listed here.
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
8 authors at 4 institutions in 1 country.
Funding
Abstract
introductionEmicizumab is a recombinant humanized bispecific monoclonal antibody mimicking the cofactor function of activated factor VIII.
aimIn this multicentre, open-label study (HOHOEMI), we evaluated the efficacy, safety and pharmacokinetics of emicizumab in Japanese paediatric patients aged <12 years with severe haemophilia A without factor VIII (FVIII) inhibitors.
methodsEmicizumab was administered subcutaneously, with four loading doses of 3 mg/kg every week followed by maintenance doses of 3 mg/kg every 2 weeks (Q2W) or 6 mg/kg every 4 weeks (Q4W) in 6 and 7 patients, respectively.
resultsAll patients completed at least 24 weeks of treatment. Baseline ages ranged from 4 months to 10 years, and all patients had been treated with FVIII prophylaxis prior to enrolment except a 4-month-old patient untreated with FVIII previously. In the respective Q2W and Q4W cohorts, 2/6 and 5/7 patients experienced no treated bleeding events, and annualized bleeding rates for treated bleeding events were 1.3 (95% confidence interval [CI], 0.6-2.9) and 0.7 (95% CI, 0.2-2.6). All caregivers preferred emicizumab to the patient's previous treatment. Only one related adverse event (injection site reaction) was observed. There were no thromboembolic events or thrombotic microangiopathy. Individual trough plasma concentrations of emicizumab were within the variability observed in preceding adult/adolescent studies. All patients tested negative for anti-emicizumab antibodies.
conclusionsEmicizumab administered Q2W or Q4W was efficacious and safe in paediatric patients with severe haemophilia A without inhibitors. This study was registered at http://www.clinicaltrials.jp (JapicCTI-173710).
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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.