SynthesisHaemophilia : the official journal of the World Federation of Hemophilia
Outcomes of Emicizumab Treatment for Haemophilia A Paediatric Patients: A Systematic Review With Meta-Analysis.
Synthesis in Haemophilia : the official journal of the World Federation of Hemophilia. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 2 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.
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Who cites it
2 citing papers in PubMed.
- When and How to Start Prophylaxis in Children with Hemophilia.Transfusion medicine and hemotherapy : offizielles Organ der Deutschen Gesellschaft fur Transfusionsmedizin und Immunhamatologie · 2026Review
- The Global Gap in the Hemophilia Paradigm Shift: Disparities in Research, Care, and Musculoskeletal Health.Hematology reports · 2026Review
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Authors and funding
7 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
introductionHaemophilia A in paediatric patients presents a lifelong risk of spontaneous and trauma-induced haemorrhage, leading to progressive joint damage, disability and impaired quality of life. Emicizumab, a bispecific monoclonal antibody administered subcutaneously, offers sustained haemostatic protection and has shown promising outcomes in children.
aimTo systematically evaluate and quantitatively synthesise the efficacy, safety and immunogenicity outcomes of emicizumab prophylaxis in paediatric patients with haemophilia A.
methodsThis systematic review and meta-analysis was conducted according to PRISMA 2020 guidelines and registered in PROSPERO (CRD420251145633). Eligible studies reported quantitative outcomes for children with haemophilia A receiving emicizumab. Random-effects models were used to pool median annualised bleeding rates (ABR) and prevalence of joint bleeding, intracranial haemorrhage (ICH), inhibitor development and anti-drug antibodies (ADA).
resultsEighteen studies comprising 720 paediatric patients were included. The pooled median ABR was 0.50 bleeds/year (95% CI: 0.00-1.11), and no cases of ICH were reported across all studies. The pooled prevalence of joint bleeds was 5.4% (95% CI: 1.41-10.96), reflecting effective musculoskeletal protection. Inhibitor development occurred in less than 0.01% of patients (nine cases), and ADA were reported in five cases without loss of clinical efficacy. No significant differences were observed in subgroup analyses by study design or geographic region.
conclusionsEmicizumab prophylaxis provides robust and consistent bleed prevention with an excellent safety and immunogenicity profile in children with haemophilia A. The near-zero ABR and absence of intracranial haemorrhage highlight its potential to transform long-term outcomes and prevent haemophilic arthropathy.
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