Trial reportHaemophilia : the official journal of the World Federation of Hemophilia2023
Emicizumab dose up-titration in case of suboptimal bleeding control in people with haemophilia A.
Trial report in Haemophilia : the official journal of the World Federation of Hemophilia, 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 11 papers.
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.
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Who cites it
11 citing papers in PubMed, 15 citations in OpenAlex.
- 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
- Haemophilia Prophylaxis in the Age of Innovation: Exploring Opportunities for Personalized Treatment.Haemophilia : the official journal of the World Federation of Hemophilia · 2025Review
- Thrombin Generation Assay to Support Hematologists in the Era of New Hemophilia Therapies.International journal of laboratory hematology · 2025Review
- No correlation between thrombin generation and emicizumab levels: implications for monitoring emicizumab therapy.Research and practice in thrombosis and haemostasis · 2025Article
- Long-term outcomes with emicizumab in hemophilia A without inhibitors: results from the HAVEN 3 and 4 studies.Research and practice in thrombosis and haemostasis · 2024Article
- Pharmacokinetics and coagulation biomarkers in children and adults with hemophilia A receiving emicizumab prophylaxis every 1, 2, or 4 weeks.Research and practice in thrombosis and haemostasis · 2024Article
- Emicizumab prophylaxis for people with hemophilia A: Waste estimation and the Brazilian perspective.Saudi pharmaceutical journal : SPJ : the official publication of the Saudi Pharmaceutical Society · 2023Article
- Serious bleeds in pediatric persons with hemophilia A on emicizumab prophylaxis.Research and practice in thrombosis and haemostasis · 2023Article
- Management and Outcomes of Invasive Procedures in Individuals with Hemophilia A on Emicizumab Prophylaxis: A Single Center Experience.Hematology reports · 2023Article
- The Evaluation of Clot Waveform Analyses for Assessing Hypercoagulability in Patients Treated with Factor VIII Concentrate.Journal of clinical medicine · 2023Article
- Thrombin Generation in Pediatric Haemophilia A Patients on Extended Half-Life FVIII versus Non-FVIII Therapies.Haemophilia : the official journal of the World Federation of HemophiliaArticle
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
12 authors at 9 institutions in 5 countries.
Funding
No grant is acknowledged in the PubMed record.
Abstract
introductionEmicizumab promotes effective haemostasis in people with haemophilia A (PwHA). It is indicated for routine prophylaxis of bleeding episodes in PwHA with or without factor (F)VIII inhibitors.
aimTo investigate the effect of emicizumab dose up-titration in PwHA with suboptimal bleeding control.
methodsData from seven completed or ongoing phase III studies were pooled. Pharmacokinetics, pharmacodynamics and bleeding events were evaluated before and after dose up-titration. Adverse events (AEs) were compared between PwHA with and without dose up-titration.
resultsOf 675 PwHA evaluable for the analysis, 24 (3.6%) had their maintenance dose up-titrated to 3 mg/kg once weekly (QW). Two participants had neutralising antibodies (nAbs) associated with decreased emicizumab exposure, and dose increase did not compensate for the effect of nAbs. In the other 22 participants, mean emicizumab steady-state trough concentrations increased from 44.0 to 86.2 μg/mL after up-titration. The median (interquartile range [IQR]) efficacy period prior to up-titration was 24.6 (24.0-32.0) weeks. The model-based annualised bleed rate for 'treated bleeds' and 'all bleeds' decreased by 70.2% and 72.9%, respectively, after a median (IQR) follow-up of 97.1 (48.4-123.3) weeks in the up-titration period. Incidences of injection-site reactions and serious AEs were higher in PwHA with up-titration; however, this was already observed in these participants before the dose up-titration. Overall, the safety profile appeared similar between PwHA with and without up-titration.
conclusionThe dose up-titration to 3 mg/kg QW was well tolerated. Bleed control improved in most participants whose bleeding tendency was inadequately controlled during clinical trials.
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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.