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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.
Insights
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.
Abstract:
The hallmark of haemophilia A (HA) therapy is prophylaxis, aimed at spontaneous bleeding prevention. Emicizumab provides a viable alternative to intravenous factor replacement therapy. However, data on its use in infants are limited. This single-centre open arm prospective study reports on emicizumab prophylaxis in infants. We included severe HA patients under 1 year who started emicizumab prophylaxis since 2018, with longitudinal follow-up. The study collected data on demographics, clinical and laboratory variables, the occurrence of bleeding events, surgeries and treatment outcomes. Of the 27 enrolled infants, whose median age at prophylaxis initiation was 7 months, 24 primarily choose to start emicizumab therapy (3/27 switched from FVIII prophylaxis due to development of FVIII inhibitors). The median age for prophylaxis initiation decreased to 3 months in 2023. Following emicizumab initiation, the median calculated ABR decreased, and no intracranial haemorrhages were observed. Thrombin generation showed a significant improvement in peak height and endogenous thrombin potential at steady state after a loading period. Our study highlights a shift towards early prophylaxis in the era of non-replacement therapies. It underscores the need for continuous evaluation and refinement of treatment approaches, emphasizing personalized care and diligent monitoring in the evolving field of paediatric haemophilia care.
