ArticleResearch and practice in thrombosis and haemostasis2024
Emicizumab is well tolerated and effective in people with congenital hemophilia A regardless of age, severity of disease, or inhibitor status: a scoping review.
Article in Research and practice in thrombosis and haemostasis, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 26 papers.
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
26 citing papers in PubMed.
- Discovery and development of a bispecific antibody for the treatment of hemophilia.Proceedings of the National Academy of Sciences of the United States of America · 2026Article
- Safety and real-world effectiveness of eptacog beta with emicizumab prophylaxis: an interim analysis of the ATHN 16 study.Blood vessels, thrombosis & hemostasis · 2026Article
- Plasma emicizumab concentrations and bleeding rates in children and adults with severe hemophilia A.Research and practice in thrombosis and haemostasis · 2026Observational
- Maternal-fetal transfer of emicizumab in a pregnant mouse model.Blood advances · 2026Article
- Next-generation FVIIIa-mimetic bispecific antibody NXT007: evaluation in preclinical models of hemostasis and thrombosis.Blood advances · 2026Article
- Emicizumab prophylaxis in a preterm infant with severe hemophilia A: a case report on the feasibility of early use.Research and practice in thrombosis and haemostasis · 2026Article
- Race, Ethnicity, and Hemophilia: A Scoping Review.Journal of racial and ethnic health disparities · 2026Review
- Factor VIIIResearch and practice in thrombosis and haemostasis · 2026Article
- Paediatric Subanalysis of TSUBASA, Assessing Physical Activity, Bleeding, Quality of Life and Safety in People with Haemophilia A Receiving Emicizumab.TH open : companion journal to thrombosis and haemostasis · 2026Article
- Anti-emicizumab antibodies and their relevance in clinical practice.Research and practice in thrombosis and haemostasis · 2026Article
- The Mirage of Factor Equivalence: Examining the Complexities of Non-Factor Therapies in Haemophilia.Haemophilia : the official journal of the World Federation of Hemophilia · 2025Review
- NXT007 enhancesResearch and practice in thrombosis and haemostasis · 2025Article
- More real-world evidence on the impact of emicizumab.Research and practice in thrombosis and haemostasis · 2025Article
- Normalization in hemophilia: conceptual foundations and clinical implications.Research and practice in thrombosis and haemostasis · 2025Review
- FRONTIER1 multiple ascending dose extension: a safety, tolerability, pharmacokinetics, and pharmacodynamics study of Mim8 in people with hemophilia A.Research and practice in thrombosis and haemostasis · 2025Article
- Curative Therapies for Hemophilias and Hemoglobinopathies in Adults: Immune, Gene, and Stem Cell Approaches in a Global Context.Biomedicines · 2025Review
- Article
- Assessment of Clinical Characteristics and Sociocontextual Factors on Medication Adherence in Children and Adolescents With Hemophilia.Pediatric blood & cancer · 2025Article
- TSUBASA Study: Evaluating Association of Physical Activity and Bleeding Events in People With Haemophilia A Without Factor VIII Inhibitors Receiving Emicizumab.Haemophilia : the official journal of the World Federation of Hemophilia · 2025Observational
- Cautious use of activated prothrombin complex concentrate in patients receiving emicizumab: new evidence and clinical considerations.Research and practice in thrombosis and haemostasis · 2025Article
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.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Background: With the treatment landscape continually evolving, it is vital that the hemophilia community have an overview of all published data for approved therapies, such as emicizumab, to support shared decision making. Objectives: To bring together the clinical and real-world data for emicizumab use in people with congenital hemophilia A, regardless of age, disease severity, or factor VIII inhibitor status. Key focus areas were safety, efficacy, and quality of life (QoL). Methods: This scoping review used citation databases (PubMed, Embase, and the Cochrane Library) and manual searches of abstract books. Publications reporting original data for emicizumab in people with hemophilia A, published in English after December 2014, and reporting select endpoints were included. This narrative synthesis focused on zero bleeds, treated annualized bleeding rate (ABR), adverse events, and QoL measures. Results: Overall, 97 publications were included (cut-off: August 9, 2022). Treated ABR remained low (calculated mean and median treated ABRs ranged between 0.7-1.3 and 0.0-1.4, respectively), and the median percentage of people with zero treated bleeds was 66.7%. The proportion of people experiencing treatment-related adverse events ranged from 0.0% to 60.0%; most were injection-site reactions. Across 37 publications reporting on safety and enrolling >2300 individuals, 11 thrombotic events and 4 thrombotic microangiopathies were reported. Data from well-established tools show QoL benefits with emicizumab. Conclusion: This scoping review consolidates the global published experience for emicizumab in people with hemophilia A and supports the fact that emicizumab has an acceptable safety profile, is effective and efficacious in bleed prevention, and is associated with improvements in QoL.
Indexed as
Identifiers
What OpenQuestion holds
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.