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Emicizumab prophylaxis in infants: Single-centre experience.
Sarina Levy-Mendelovich1,2,3, Noa Greenberg-Kushnir4, Ivan Budnik5
1National Haemophilia Center and Thrombosis & Hemostasis Institute, Sheba Medical Center, Ramat Gan, Israel.
British Journal of Haematology
|January 24, 2024
Summary
Emicizumab prophylaxis is effective in preventing bleeds in infants with severe haemophilia A (HA). This study shows a trend towards earlier prophylaxis initiation and improved outcomes in young children with HA.
Area of Science:
- Hematology
- Pediatric Medicine
- Pharmacology
Background:
- Haemophilia A (HA) management traditionally relies on factor replacement therapy.
- Prophylaxis is the standard of care for preventing spontaneous bleeding in HA.
- Limited data exist on emicizumab use in infants with HA.
Purpose of the Study:
- To evaluate the safety and efficacy of emicizumab prophylaxis in infants with severe HA.
- To assess the impact of emicizumab on bleeding events and thrombin generation in this population.
- To identify trends in prophylaxis initiation age in the era of non-replacement therapies.
Main Methods:
- Prospective, single-centre, open-arm study of infants (<1 year) with severe HA starting emicizumab.
- Longitudinal data collection on demographics, clinical variables, bleeding events, surgeries, and treatment outcomes.
- Analysis of annualized bleeding rates (ABR) and thrombin generation parameters.
Main Results:
- 27 infants enrolled; median age at initiation 7 months (decreasing to 3 months by 2023).
- Significant reduction in median calculated ABR and no intracranial hemorrhages observed post-initiation.
- Improved thrombin generation (peak height, endogenous thrombin potential) after loading dose.
Conclusions:
- Emicizumab is a viable prophylactic option for infants with severe HA, demonstrating reduced bleeding and improved hemostasis.
- A shift towards earlier prophylaxis initiation in infants with HA is evident.
- Continuous monitoring and personalized care are crucial for optimizing treatment in pediatric HA.
