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Outcomes of Emicizumab Treatment for Haemophilia A Paediatric Patients: A Systematic Review With Meta-Analysis

Konstantina Bolou1, George Triantafyllou2, Athina Dettoraki3

  • 1School of Medicine, Faculty of Health Sciences, National and Kapodistrian University of Athens, Athens, Greece.

Haemophilia : the Official Journal of the World Federation of Hemophilia
|December 13, 2025
PubMed

Insights

Emicizumab prophylaxis significantly reduces bleeding in children with haemophilia A, showing a near-zero annualised bleeding rate and no intracranial haemorrhage cases. This treatment offers excellent safety and immunogenicity, potentially transforming long-term outcomes.

Area of Science:

  • Paediatric Haematology
  • Pharmacology
  • Clinical Medicine

Background:

  • Haemophilia A in children poses lifelong bleeding risks, joint damage, and reduced quality of life.
  • Emicizumab, a subcutaneous bispecific monoclonal antibody, provides sustained protection against haemorrhage.
  • Promising outcomes have been observed with emicizumab in paediatric populations.

Purpose of the Study:

  • To systematically evaluate and quantitatively synthesize the efficacy, safety, and immunogenicity of emicizumab prophylaxis.
  • To assess emicizumab's impact on bleeding rates, joint health, and adverse events in children with haemophilia A.

Main Methods:

  • Systematic review and meta-analysis adhering to PRISMA 2020 guidelines.
  • Inclusion of 18 studies with 720 paediatric patients receiving emicizumab.
  • Random-effects models used to pool outcomes including annualised bleeding rates (ABR), joint bleeds, intracranial haemorrhage (ICH), inhibitor development, and anti-drug antibodies (ADA).

Main Results:

  • Pooled median ABR was 0.50 bleeds/year; no ICH cases were reported.
  • Pooled prevalence of joint bleeds was 5.4%, indicating effective musculoskeletal protection.
  • Inhibitor development (<0.01%) and ADA (5 cases) occurred without loss of clinical efficacy.

Conclusions:

  • Emicizumab prophylaxis demonstrates robust and consistent bleed prevention in paediatric haemophilia A.
  • The treatment profile is characterized by excellent safety and immunogenicity.
  • Near-zero ABR and absence of ICH suggest emicizumab can transform long-term outcomes and prevent arthropathy.
Abstract

Keywords:
emicizumabevidence‐based medicinehaemophilia Ainfantmeta‐analysispaediatric

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